A Peer Support Service Integrated Into the 988 Lifeline

A peer support option should be integrated into the 988 Suicide and Crisis Lifeline so that 988 service users can choose to connect with specialists based on a shared lived experience. As people and communities become more siloed, the risk of mortality and morbidity increases. Social connectedness is a critical protective factor. A peer support service would allow individuals to receive support built on a lived experience that is common to both the service user and the specialist. It should be free and easily accessible through phone call and text messaging. This service is especially timely, following the 2020 rollout of the 988 Suicide and Crisis Lifeline, as well as recent peer support initiatives at the federal and state levels.

While the efficacy of peer support is most known with mental illness, it has successfully helped a range of individuals including cancer patients, people experiencing homelessness, racial minorities, veterans, and formerly incarcerated people. The peer support service should provide support for all kinds of lived experience, including experiences with: disability resulting from poor physical or mental health, substance use, suicidal ideation, veteran-connected disability, financial insecurity, homelessness, domestic violence and family court, nonnuclear family structures or living alone, former incarceration, and belonging to racial or ethnic minority groups. The service should be a preventative intervention as well as complimentary assistance for those in recovery or treatment.

Challenge and Opportunity

The United States faces an “epidemic of loneliness and isolation.” While individuals from all backgrounds are afflicted, the most vulnerable and underserved members of society have suffered the most. A range of challenging circumstances cause this suffering, including poor physical or mental health, nontraditional living conditions, and historic inequality. Not having anyone who shares one’s lived experience is isolating. These challenging life circumstances take an emotional toll, and they become risk factors for a slew of physical and mental health conditions that increase morbidity and decrease life expectancy. 

In addition to the social costs, these outcomes have an increasingly devastating economic impact. Health care costs are projected to account for 20% of the U.S. economy by 2031. Psychiatric hospitalizations are steadily increasing, and a shortage of psychiatric inpatient beds is rampant.

Social connectedness alleviates physical and mental burdens. Peer support offers a cost-effective intervention for prevention and recovery. It reduces spending on both physical and mental illnesses, and it reduces psychiatric hospitalizations, saving an average of $4.76 for every $1 spent on peer support. In a New York City-based peer support program, service users saved about $2,000 per month in Medicaid costs and had 2.9 fewer hospitalizations per year. 

Telephone-based peer support has life-saving outcomes, too. Over telephone, peer support led to a 15% increase in women’s mammography screening rates, with the highest increase among women of low-to-middle income. Telephone-based peer support increased breastfeeding rates by 14% and reduced breastfeeding dissatisfaction by 10% among first-time mothers, and it led to a 10% change in diet among patients with heart disease. While peer support would not solve national crises of homelessness or rising healthcare costs, it would ease them by fostering community empowerment and self-reliance and reducing federal intervention.

In 2023, SAMHSA rolled out “National Model Standards for Peer Support Certification”. This guide provides recommendations for how each state can integrate its own “peer mental health workforce across all elements of the healthcare system.” In its current form, SAMHSA’s strategy targets lived experiences with substance use and mental health. A broader scope would assist and empower more underserved members of the community. Following the momentum of SAMHSA’s initiative, now is the most optimal time to integrate a peer support service into the 988 Lifeline.

Peer support exists in the U.S., but services are spread thin across private and nonprofit sectors. The Restoring Hope for Mental Health and Well-Being Act of 2022 authorized $1.7 trillion in funding until FY2027 for various health initiatives, including peer support mental health services. This funding relies on organizations and institutions to independently implement peer support services based on community needs. However, a fragmented system can result in underuse, limited accessibility, and a varied quality of service, with pockets of the United States lacking any service at all. It also poses privacy concerns about how individuals’ data are stored and used, as well as cybersecurity vulnerabilities with smaller organizations that may lack a robust security infrastructure.

A peer support service that is integrated into the 988 Lifeline would ensure that all Americans have equal access to a high-quality, confidential peer support network. The standardization of the 988 Lifeline is a prime example of successful implementation. Its transition from a 10-digit number to a three-digit dial led to a 33% average increase in in-state call volume over four months. Standardization shifted funding from primarily private and nonprofit initiatives and donations to stable public sector support. As a result, call pickup rates rose from 70% to 93%, and wait times dropped from 2 minutes 20 seconds to just 35 seconds.

The 988 Lifeline also adheres to privacy and confidentiality protocols in line with the Health Insurance Portability and Accountability Act (HIPAA) Security Rule. Under these protocols, the 988 Lifeline retains minimal information about callers and texters, and this information stays private and securely stored. A peer support service with similar safeguards would help both service users and peer support workers (PSWs) feel safe when sharing sensitive information.

Peer support for mental health has gained traction recently. The National Alliance for Mental Illness (NAMI) offers peer-to-peer courses, although these are currently limited in their duration and available locations. Last year, Congress expressed an interest in peer support mental health services: a “Supporting All Students Act” was proposed in the Senate for “peer and school-based mental health support.” This endeavor especially targets the escalating suicide crisis among youths in the form of a peer-to-peer suicide prevention. The Wisconsin Department of Public Instruction also recently introduced Peer-to-Peer Suicide Prevention Grants. The federal and state-level governments clearly recognize the value of peer support work. Still, they underutilize its potential.

Loneliness and isolation are at an all-time high in the United States. However, they stem from a multitude of causes. An empathic connection with a peer who has lived the same experience has immense potential for healing and recovery in both the PSW and the service user. The 988 Lifeline should include an integrated peer support service for both mental health and a broad range of lived experiences. While there exist some peer support services for conditions not related to mental health, these are not standardized or easily accessible to the entire American population. Many peer-operated warmlines (i.e., support lines) exist. However, they are spread across dozens of phone numbers and websites with varied and limited sources of funding. A single peer support service integrated into the 988 Lifeline would make peer support universally available. This service would address the emotional toll that accompanies a wide range of challenging life circumstances.

In short, a peer support service integrated into the 988 Lifeline would make the efforts of existing peer support organizations even more effective as it would:

Action Plan

The peer support service should begin by covering only a few kinds of lived experiences. After this implementation, the service should be expanded to cover a broader range of experiences.

Recommendation 1. Create a Peer Support Task Force (PSTF).

A PSTF should be established within SAMHSA. The secretary of the U.S. Department of Health and Human Services (HHS) should work with SAMHSA’s assistant secretary for mental health and substance use to establish this temporary task force.

The PSTF should lead the implementation of the peer support service, acting as an interagency task force that coordinates with partners across the public, private, and nonprofit sectors. This partnership will ensure that different lived experiences are accounted for and that existing resources are used effectively. The PSTF should collaborate with federal agencies, including the Department of Veterans Affairs (VA) and the Indian Health Service (IHS), as well as with advocacy organizations like NAMI and the American Cancer Society that champion the needs of people with specific lived experiences.

The PSTF should be charged with the following recommendations to start.

Recommendation 2. Integrate a peer support option into the 988 Lifeline.

Under the authority of the PSTF, integrating a peer support option into the 988 Lifeline could bypass additional Congressional action.

In the first pass, the integrated peer support option should cover only a few kinds of lived experiences (e.g., suicide and behavioral crises, veteran-connected disability).

  1. Before scaling the peer support service across the United States, the PSTF should implement a pilot program to test and refine protocols. The PSTF should select 988 Lifeline centers in states that already have a strong peer support program, and it should pilot peer support call and text services. The pilot program should select a few peer support needs to test first (e.g., suicide and behavioral crises, veteran-connected disability), as a narrow scope will be easier to assess for the first pass.
  2. The PSTF should then incorporate feedback from this pilot program as it scales up and integrates a peer support option into the nationwide 988 Lifeline.
  3. The PSTF should coordinate with the Federal Communications Commission (FCC) to add a caller menu option for peer support into the 988 Lifeline. (E.g., “Press 4 for peer support.”)
  4. The PSTF should coordinate with the FCC and U.S. Digital Service to implement a telephone triage, so the service user can submit a request for a specific kind of peer support and then be routed to a PSW on shift who has a lived experience that best matches the submitted request.
  5. The PSTF should facilitate the 988 Lifeline’s partnership with existing local and national peer support organizations and warmlines. Partnering with these existing organizations would bolster the 988 Lifeline’s capacity to provide peer support. Furthermore, states that already have a strong peer support certification in place could easily integrate their trained PSWs into the 988 Lifeline services using their existing infrastructure and expertise.
  6. The PSTF should coordinate with the FCC to incorporate a peer support text messaging option within the 988 Lifeline’s text and chat services. (For example, service users could text “PEER” to 9-8-8 and be routed to a PSW on shift.)
  7. The PSTF should implement a public campaign to the general public clarifying that the 988 Lifeline will remain as a suicide and crisis hotline, but it will also provide access to broader peer support services. The available peer support services should be clearly outlined on the 988 Lifeline website.

PSWs should work in call centers alongside 988 Lifeline phone and text specialists. 

Recommendation 3. Develop an action plan to fund and sustain the integration of the peer support service.

The peer support service would need funding to become integrated into the 988 Lifeline. The government has recently approved funds for mental health initiatives, such as with the Restoring Hope for Mental Health and Well-Being Act of 2022. In 2023, HHS announced an additional $200 million in funding for the 988 Lifeline, but overall, HHS has granted almost $1.5 billion in total toward the 988 Lifeline. Similar avenues of funding should help jumpstart the integration of the peer support service.

For continued maintenance, fees for the peer support service should apply in a similar fashion to 9-1-1. That is, service users should not pay each time they access the 988 Lifeline or its integrated peer support service. Some U.S. states have already passed 9-8-8 implementation legislation that allows a monthly flat fee to be collected through telephone and wireless service providers, as permitted in the “National Suicide Hotline Designation Act of 2020”. The remaining states should be encouraged to pass similar legislation. The fee should remain in an account that is spent only for the maintenance of the 988 Lifeline and peer support services. If a fee is collected, then the FCC should provide an annual report on these fees and their usage.

Funding for the peer support service’s integration into the 988 Lifeline would entail the following:

Recommendation 4. Establish state-level standardized peer support training and certification. 

PSWs should learn skills that are commonly taught to 988 Lifeline specialists, including active listening and recovery-oriented language. They should learn how to share their own stories, navigate challenging conversations, maintain boundaries, and self-care. Their training should be standardized at the state level, and it should be based on the “National Model Standards for Peer Support Certification” established by SAMHSA. 

While most U.S. states have some version of peer support certification for mental health and/or substance use recovery, the PSTF should work with advocacy organizations to ensure that the state-level standardized training accounts for the peer support needs and demographics of each state. The peer support training should address the needs of people with a diverse range of lived experiences. Peer support needs can also be studied using caller outcome data that are recorded by 988 Lifeline specialists.

The PSTF should recruit trainers who will train and mentor the PSWs. To start, the PSTF should liaise within SAMHSA and with existing peer support organizations to coordinate this recruitment.

Recommendation 5. Establish a nation-wide system for the recruitment and training of PSWs.

Recruit individuals who would like to use their own lived experience to help other community members. Recruitment should occur through hospitals, clinics, as well as peer-run and community-based organizations to maximize recruitment pathways. 

Current 988 Lifeline phone and text specialists could also be good candidates for the peer support service. Many are motivated by their own lived experiences and are already trained to handle calls across multiple helplines. 988 Lifeline specialists are instructed to focus on the service user seeking help and not share about themselves. However, specialists’ own lived experiences––if they are comfortable sharing them––represent untapped potential.

Once recruited, individuals who are training to become PSWs should attend live training sessions online with a peer cohort.

Recommendation 6. Expand the peer support service to cover a diverse range of lived experiences.

After successfully establishing the peer support service, it should be expanded upon to provide support for a broad and diverse range of lived experiences. The available peer support services should continue to be updated and outlined on the 988 Lifeline website.

Conclusion

A peer support service should be integrated into the 988 Lifeline. A service that caters to all kinds of lived experience paves way for a more empowered and self-reliant community. It fosters a societal mindset of helping each other based on shared lived experience in an empathic and healthy way. Peer support empowers both the PSW and the service user. While the service user finds empathy and understanding, the PSW finds a renewed sense of purpose and confidence. In this way, peer support would alleviate loneliness and isolation that result from a variety of causes while instilling longer-term resilience into the community.

This action-ready policy memo is part of Day One 2025 — our effort to bring forward bold policy ideas, grounded in science and evidence, that can tackle the country’s biggest challenges and bring us closer to the prosperous, equitable and safe future that we all hope for whoever takes office in 2025 and beyond.

Frequently Asked Questions
What is a service user?

A service user refers to any individual who uses the peer support service for assistance.

What if peer support workers (PSWs) become distressed or emotionally fatigued?

Peer support work can be empowering and healing for those in recovery. However, as with any emotionally challenging work, PSWs benefit from ongoing support and supervision. Some interactions can be draining, especially if they hit too close to home. It is common for 988 Lifeline call centers to have a trained staff member, such as a psychologist or therapist, on call for such situations. A PSW could use this resource to debrief or process as needed. Additionally, during recruitment, PSW candidates should be screened to ensure they are comfortable speaking about their own lived experience and helping others who are going through the same experience.

How would PSWs be compensated?

Most PSWs would be compensated in the same way as their 988 Lifeline call and text specialist counterparts. They would be compensated with rigorous ongoing training, support, and resources for recovery and self-care in return for their time. They would also benefit from a social support system of fellow PSWs. Some individuals who may be ideal candidates for peer support work may struggle to find employment and health insurance. They will be less likely to volunteer if they do not have a living wage. To maximize the range of lived experiences available, certain individuals should be eligible for financial compensation.

For the peer support phone and text service, how is a caller or texter connected with a PSW who has the same lived experience?

A service user’s phone call or text should be routed to a 988 Lifeline peer support operator or telephone triage, where the service user is asked to say aloud (or type) what kind of support they are searching for (e.g., “I want to speak with someone who has been in prison.”) Then, the service user’s call (or text) would be routed to a PSW (under an alias) anywhere in the United States who 1) is currently on shift and 2) has a lived experience that is closest to the service user’s request. Each PSW would have associated keywords, such as “former incarceration,” “PTSD,” or “living alone,” which means that they are trained to connect with any service user about those lived experiences. The PSW would follow the service user’s lead in the conversation, and the PSW could share parts of their own lived experience when appropriate.

Why should the peer support service include a text messaging option?

Text messaging can be more accessible than a phone line for youths and people with disabilities. The 988 Lifeline includes a text messaging option for this same reason.

Why should we integrate another option into the 988 Lifeline when some states still struggle to find sustainable ways to fund and staff the 988 Lifeline as it currently is?

It is true that both the regular 988 Lifeline and peer support service would provide resources and emotional support. By incorporating peer support into the 988 Lifeline, existing local and national peer support organizations would be eligible to partner with the 988 Lifeline and bolster its capacity to provide peer support. This endeavor will help with some staffing concerns. Furthermore, the peer support service would cover a diverse range of lived experiences extending beyond mental health. Therefore, more individuals may be motivated to join the 988 Lifeline staff to share their unique lived experience and help others who feel the same way.


Integrating a versatile peer support service into the 988 Lifeline transforms the latter into a service that every single American can use. (Every American has some unique lived experience.) The service’s versatility may help incentivize the remaining states to pass legislation to collect a 988 Lifeline fee through telephone and wireless service providers.


Finally, peer support is a cost-effective, preventative intervention. It should help remove the burden on other federal services, thereby reducing overall spending in time.

What happens next?

After implementing the action plan outlined in this memo, a subsequent memo should outline how to integrate peer support work into the community in person and on a large scale. Incorporating peer support into the 988 Lifeline would show its effectiveness to the public, policymakers, and healthcare professionals. This credibility would bolster endeavors to integrate peer support work into community settings throughout the country (e.g., behavioral health centers, hospitals and emergency rooms, and community clinics). These endeavors could also lead to professionalizing peer support, so PSWs can be reimbursed through Medicaid programs and health insurance.

Clearing the Path for New Uses for Generic Drugs

The labeling-only 505(b)(2) NDA pathway for non-manufacturers to seek FDA approval

Repurposing generic drugs as new treatments for life-threatening diseases is an exciting yet largely overlooked opportunity due to a lack of market-driven incentives. The low profit margins for generic drugs mean that pharmaceutical companies rarely invest in research, regulatory efforts, and marketing for new uses. Nonprofit organizations and other non-commercial non-manufacturers are increasing efforts to repurpose widely available generic drugs and rapidly expand affordable treatment options for patients. However, these non-manufacturers find it difficult to obtain regulatory approval in the U.S. They face significant challenges in using the existing approval pathways, specifically in: 1) providing the FDA with required chemistry, manufacturing, and controls (CMC) data, 2) providing the FDA with product samples, and 3) conducting post-marketing surveillance. Without a straightforward path for approval and updating drug labeling, non-manufacturers have relied on off-label use of repurposed drugs to drive uptake. This practice results in outdated labeling for generics and hinders widespread clinical adoption, limiting patient access to these potentially life-saving treatments. 

To encourage greater adoption of generic drugs in clinical practice – that is, to encourage the repurposing of these drugs – the FDA should implement a dedicated regulatory pathway for non-manufacturers to seek approval of new indications for repurposed generic drugs. A potential solution is an extension of the existing 505(b)(2) new drug application (NDA) approval pathway. This extension, the “labeling-only” 505(b)(2) NDA, would be a dedicated pathway for non-manufacturers to seek FDA approval of new indications for well-established small molecule drugs when multiple generic products are already available. The labeling-only 505(b)(2) pathway would be applicable for repurposing drugs for any disease. Creating a regulatory pathway for non-manufacturers would unlock access to innovative therapies and enable the public to benefit from the enormous potential of low-cost generic drugs.

Challenge and Opportunity

The opportunity for generic drug repurposing

On-patent, branded drugs are often unaffordable for Americans. Due to the high cost of care, 42% of patients in the U.S. exhaust their life savings within two years of a cancer diagnosis. Generic drug repurposing – the process of finding new uses for FDA-approved generic drugs – is a major opportunity to quickly create low-cost and accessible treatment options for many diseases. In oncology, hundreds of generic drugs approved for non-cancer uses have been tested as cancer treatments in published preclinical and clinical studies. 

The untapped potential for generic drug repurposing in cancer and other diseases is not being realized because of the lack of market incentives. Pharmaceutical companies are primarily focused on de novo drug development to create new molecular and chemical entities. Typically, pharmaceutical companies will invest in repurposing only when the drugs are protected by patents or statutory market exclusivities, or when modification to the drugs can create new patent protection or exclusivities (e.g., through new formulations, dosage forms, or routes of administration). Once patents and exclusivities expire, the introduction of generic drugs creates competition in the marketplace. Generics can be up to 80-85% less expensive than their branded counterparts, driving down overall drug prices. The steep decline in prices means that pharmaceutical companies have little motivation to invest in research and marketing for new uses of off-patent drugs, and this loss of interest often starts in the years preceding generic entry.

In theory, pharmaceutical companies could repurpose generics without changing the drugs and apply for method-of-use patents, which should provide exclusivity for new indications and the potential for higher pricing. However, due to substitution of generic drugs at the pharmacy level, method-of-use patents are of little to no practical value when there are already therapeutically equivalent products on the market. Pharmacists can dispense a generic version instead of the patent-protected drug product, even if the substituted generic does not have the specific indication in its labeling. Currently, nearly all U.S. states permit substitution by the pharmacy, and over a third have regulations that require generic substitution when available.

Nonprofits like Reboot Rx and other non-commercial non-manufacturers are therefore stepping in to advance the use of repurposed generic drugs across many diseases. Non-manufacturers, which do not manufacture or distribute drugs, aim to ensure there is substantial evidence for new indications of generic drugs and then advocate for their clinical use. Regulatory approval would accelerate adoption. However, even with substantial evidence to support regulatory review, non-manufacturers find it difficult or impossible to seek approval for new indications of generic drugs. There is no straightforward pathway to do so within the current U.S. regulatory framework without offering a specific, manufactured version of the drug. This challenge is not unique to the U.S.; recent efforts in the European Union (EU) have sought to address the regulatory gap. In the 2023 EU reform of pharmaceutical legislation, Article 48 is currently under review by the European Parliament as a potential solution to allow nonprofit entities to spearhead submissions for the approval of new indications for authorized medicinal products with the European Medicines Agency. To maximize the patient impact of generic drugs in America, non-manufacturers should be able to drive updates to FDA drug labeling, enabling widespread clinical adoption of repurposed drugs in a formal, predictable, and systematic manner.

The importance of FDA approval

Drugs that are FDA-approved can be prescribed for any indication not listed on the product labeling, often referred to as “off-label use”. Since non-manufacturers face significant challenges pursuing regulatory approval for new indications, they often must rely on advocating for off-label use of repurposed drugs.

While off-label use is widely accepted and helpful for specific circumstances, there are significant advantages to having FDA approval of new drug indications included in labeling. FDA drug labeling is intended to contain up-to-date information about drug products and ensures that the necessary conditions of use (including dosing, warnings, and precautions) are communicated for the specific indications. It is the primary authoritative source for making informed treatment decisions and is heavily valued by the medical community. Approval may increase the likelihood of uptake by clinical guidelines, pathways systems, and healthcare payers. Indications with FDA approval may generate greater awareness of the treatment options, leading to a broader and more rapid impact on clinical practice. 

Clinical practice guidelines are often the leading authority for prescribers and patients regarding off-label use. In oncology, for example, the National Comprehensive Cancer Network (NCCN) Guidelines are commonly used guidelines that include many off-label uses. However, guidelines do not exist for every disease and medical specialty, which can make it more difficult to gain acceptance for off-label uses. The Centers for Medicare and Medicaid Services (CMS) policy routinely covers off-label drug uses if they are listed in certain compendia. The NCCN Compendia, which is based on the NCCN Guidelines, is the only accepted compendium that is disease-specific.

Off-label use requires more effort from individual prescribers and patients to independently evaluate new drug data, thereby slowing uptake of the treatments. This can be especially difficult for community-based physicians, who need to remain up-to-date on new treatment options across many diseases. Off-label prescribing can also introduce medico-legal risks, such as malpractice. These burdens and risks limit off-label prescribing, even when there is supportive evidence for the new uses.

As new uses for generic drugs are discovered, it is crucial to update the labeling to ensure alignment with current clinical practice. Outdated generic drug labeling means that prescribers and patients may not have access to all the necessary information to understand the full risk-benefit profile. Americans deserve to have access to all effective treatment options – especially low-cost and widely available generic drugs that could help mitigate the financial toxicity and health inequities faced by many patients. For the public benefit, the FDA should support approaches that remove regulatory barriers for non-manufacturers and modernize drug labeling.

Existing pathways for manufacturers to obtain FDA approval 

The current FDA approval system is based on the idea that sponsors have discrete physical drug products. Traditionally, sponsors seeking FDA approval are pharmaceutical companies or drug manufacturers that intend to produce (or contract for production), distribute, and sell the finished drug product. For the purposes of FDA regulation, “drug” refers to a substance intended for use in the treatment or prevention of disease; “drug product” is the final dosage form that contains a drug substance and inactive ingredients made and sold by a specific manufacturer. One drug can be present on the market in multiple drug products. In the current regulatory framework, drug products are approved through one of the following:

Manufacturers can add new indications to their approved labeling without modifying the drug product through existing pathways. With supportive clinical evidence for the new indication, the NDA holder can file a supplemental NDA (sNDA), while an ANDA holder may submit a 505(b)(2) NDA as a supplement to their existing ANDA. As previously discussed, the drug product will likely be subject to pharmacy-level substitution with any available therapeutically equivalent generic. The marketing exclusivities that sponsors may receive from the FDA do not protect against this substitution. Therefore, these pathways are rarely, if ever, used by pharmaceutical companies when there are already multiple generic manufacturers of the product. 

Challenges for non-manufacturers in using existing pathways

Since manufacturers are not incentivized to seek regulatory approval for new indications, labeling changes are more likely to happen if driven by non-manufacturers. Yet non-manufacturers face significant challenges in utilizing the existing regulatory pathways. Sponsors must submit the following information for all NDAs for the FDA’s review: 1) clinical and nonclinical data on the safety and effectiveness of the drug for the proposed indication; 2) the proposed labeling; and 3) chemistry, manufacturing, and controls (CMC) data describing the methods of manufacturing and the controls to maintain the drug product’s quality. 

To submit and maintain NDAs, non-manufacturer sponsors would need to address the following challenges: 

  1. Providing the FDA with required CMC data. NDA sponsors must provide CMC data for FDA review. Non-manufacturers would not produce physical drug products, and therefore they would not have information on the manufacturing process. 
  2. Providing the FDA with product samples. If requested, NDA sponsors must have the drug products and other samples (e.g., drug substances or reference standards) available to support the FDA review process and must make available for inspection the facilities where the drug substances and drug products are manufactured. Non-manufacturers would not have physical drug products to provide as samples, the capabilities to produce them, or access to the facilities where they are made. 
  3. Conducting post-marketing surveillance. Post-marketing responsibilities to maintain an NDA include conducting annual safety reporting and maintaining a toll-free number for the public to call with questions or concerns. Non-manufacturers, such as small nonprofits, may not have the bandwidth or resources to meet these requirements.

Within the current statutory framework, a non-manufacturer could sponsor a 505(b)(2) NDA to obtain approval of a new indication by partnering with a current manufacturer of the drug – either an NDA or ANDA holder. The manufacturer would help meet the technical requirements of the 505(b)(2) application that the non-manufacturer could not fulfill independently. Through this partnership, the non-manufacturer would acquire the CMC data and physical drug product samples from the manufacturer and rely on the manufacturer’s facilities to fulfill FDA inspection and quality requirements.

Once approved, the 505(b)(2) NDA would create a new drug product with indication-specific labeling, even though the product would be identical to an existing product under a previous NDA or ANDA. The 505(b)(2) NDA would then be tied to the specific manufacturer due to the use of their CMC data, and that manufacturer would be responsible for producing and distributing the drug product for the new indication.

As a practical matter, this pathway is rarely attainable. Manufacturers of marketed drug products, particularly generic drug manufacturers, lack the incentives needed to partner with non-manufacturers. Manufacturers may not want to provide their CMC data or samples because it may prompt FDA inspection of their facilities, require an update to their CMC information, or open the door to product liability risks. The existing incentive structure strongly discourages generic drug manufacturers from expending any additional resources on researching new uses or making any changes to their product labeling that would deviate from the original RLD product. 

Plan of Action 

To modernize drug labeling and enhance clinical adoption of generic drugs, the FDA should implement a dedicated regulatory pathway for non-manufacturers to seek approval of new indications for repurposed generic drugs. Ultimately, such a pathway would enable drug repurposing and be a crucial step toward equitable healthcare access for Americans. We propose a potential solution – a “labeling-only” 505(b)(2) NDA – as an extension of the existing 505(b)(2) approval pathway. 

Overview of the proposed labeling-only 505(b)(2) NDA pathway 

The labeling-only 505(b)(2) NDA would enable non-manufacturers to reference CMC information from previous FDA determinations and, when necessary, provide the FDA with samples of commercially available drug products. Through this approach, the new indication would not be tied to a specific drug product made by one manufacturer. There is no inherent necessity for a new indication of a generic drug to be exclusively linked to a single manufacturer or drug product when the FDA has already approved multiple therapeutically equivalent generic drugs. Any of these interchangeable drug products would be considered equally safe and effective for the new indication, and patients could receive any of these drug products due to pharmacy-level substitution. 

We describe non-manufacturer repurposing sponsors as entities that intend to submit or reference clinical data through a labeling-only 505(b)(2) NDA. This pathway is designed to expand the FDA-approved labeling of generic drugs for new indications, including those that may already be considered the standard of care. Non-manufacturers do not have the means to independently produce or distribute drug products. Instead, they intend to show that there is substantial evidence to support the new use through FDA approval, and then advocate for the indication in clinical practice. This evidence may be based on their research or research performed by other entities, including clinical trials and real-world data analyses.

The labeling-only 505(b)(2) NDA pathway helps address the three major challenges non-manufacturers face in pursuing regulatory approval. Through this pathway, non-manufacturers would be able to: 

  1. Reference the FDA’s previous determinations on CMC data. Currently, a 505(b)(2) NDA can reference the FDA’s previous determinations of safety and effectiveness for an approved drug product. For eligible generic drugs, the labeling-only 505(b)(2) NDA would build on this practice by allowing non-manufacturer sponsors to reference the FDA’s previous determinations on any NDA or ANDA that the manufacturing process and CMC data are adequate to meet regulatory standards.
  2. Provide the FDA with product samples using commercially available drug product samples. Currently, it is up to the discretion of the FDA whether or not to request samples in the review of an application. With the labeling-only 505(b)(2) NDA, non-manufacturers would provide the FDA with samples of commercially available products from generic manufacturers. Given that the FDA would have already evaluated the products and their bioequivalence to the RLD during the previous reviews, it is not expected that the FDA would need to re-examine the product at the level of requesting samples, except potentially to examine the packaging and physical presentation of the product for compatibility with the new indication and conditions of use. The facilities where the drugs are made would remain available for inspection, under the same terms and conditions as the existing, approved marketing applications. 
  3. Manage post-marketing responsibilities. Since most post-marketing surveillance and adverse event reporting are drug product-specific, these obligations would continue to be the responsibility of the manufacturer of the physical drug product dispensed. With the labeling-only 505(b)(2) NDA, the non-manufacturer would not have product-specific obligations because they are not putting a new product into the marketplace. However, we anticipate the non-manufacturer would be responsible for the repurposed indication on their labeling, including but not limited to post-marketing surveillance as well as indication-specific adverse event reporting and reasonable follow-up.

Under the labeling-only 505(b)(2) NDA, the non-manufacturer sponsor would not introduce a new physical drug product into the market. The new labeling created by the approval would not expressly be associated with one specific product. The non-manufacturer’s labeling would refer to the drug by its established generic name. In that way, the non-manufacturer sponsor’s approval and labeling could be applicable to all equivalent versions of the drug product and would be available for patients to receive from their pharmacy in the same way that generic drugs are typically dispensed at the pharmacy. That is, with the benefit of pharmacy-level substitution, patients could receive any available, therapeutically equivalent drug products from any current manufacturer. 

Eligibility criteria

We envision the users of this pathway to be non-manufacturers that conduct drug repurposing research for the public benefit, including organizations like nonprofits and patient advocacy groups. The FDA should implement and enforce additional guardrails on eligibility to ensure that sponsors operate in good faith and cannot otherwise meet traditional NDA requirements. This process may include pre-submission meetings and reviews. The labeling-only 505(b)(2) NDA should be held to the FDA’s standard level of rigor and scrutiny of safety and effectiveness for the proposed indication during the review process.

The labeling-only 505(b)(2) would only be suitable for well-established, commercially available small molecule generic drugs, which can be identified as: 

  1. Drugs with a U.S. Pharmacopeia and National Formulary (USP-NF) monograph. The USP-NF monograph system ensures the uniformity of available products on the market by setting a consensus minimum standard of identity, strength, quality, and purity among all marketed versions of a drug. It is expressly recognized in the Federal Food, Drug, and Cosmetics Act (FDCA). The USP-NF strives to have substance and product monographs for all FDA-approved drugs. USP-NF monographs for generics are commonly available because the drugs have been on the market for a long time and are typically produced by multiple manufacturers. Drug products in the U.S. market must conform to the standards in the USP-NF, when available, to avoid possible charges of adulteration and misbranding. 

By statute and regulation, the FDA already allows for NDAs and ANDAs to reference the USP-NF to satisfy some CMC requirements, such as for specifications of the drug substance. As an illustration of the acceptance of the USP-NF, clinical trial protocols requiring the use of background therapy or supportive care, as well as trials testing medical devices requiring the use of a drug product, often will specify that any available version of the drug product meeting USP-NF standards can be used. We propose that products without USP-NF monographs, including certain newer drugs and drugs with especially complicated manufacturing processes that are not conducive to standardization, would not be eligible for the labeling-only 505(b)(2) pathway.

  1. Drugs with multiple A-rated, therapeutically equivalent products in the FDA Orange Book. The FDA does not regulate which specific products are dispensed or substituted for a given drug prescription. The listing of therapeutic equivalents in the Orange Book facilitates the seamless replacement of drug products from different manufacturers in clinical practice. Therapeutically equivalent drug products: i) have demonstrated bioequivalence to the RLD; ii) have the same strength, dosage form, and route of administration as the RLD; and iii) are labeled for the same conditions of use as the RLD. Therapeutic equivalents that meet these criteria are designated “A-rated” in the Orange Book. A-rated drug products are substitutable for any other version of that A-rated drug product, including the RLD itself. 

Implementation 

The labeling-only 505(b)(2) NDA pathway could be implemented through an FDA guidance document interpreting the current statute and regulations or through legislation that clarifies the FDA’s existing authority. Guidance documents contain the FDA’s interpretation of a given policy on regulatory issues. The FDA’s Center for Drug Evaluation and Research (CDER) could issue new guidance that allows for interpretation of the existing statute, thereby officially authorizing previous FDA determinations of acceptable CMC data to be referenceable for eligible generic drugs and adjusting drug sample requirements. Alternatively, the labeling-only 505(b)(2) could be enacted by Congress through a statutory change by incorporation into FDCA, which is up for reauthorization through the Prescription Drug User Fee Act (PDUFA) in 2027, or other congressional acts as appropriate. FDA guidance would be a faster pathway to adoption, while statutory authorization would offer additional safeguards for the continuance of the pathway long term.

The labeling-only 505(b)(2) NDA pathway could be funded through user fees, which are established by PDUFA for the cost to file and maintain NDAs. However, many nonprofit sponsors would not be able to afford the same user fees as for-profit pharmaceutical manufacturers. Relevant statutes will likely need to be updated to create a different fee schema for non-manufacturers who use the labeling-only 505(b)(2) pathway. In a similar spirit to reducing barriers to maintaining up-to-date labeling, the 2017 PDUFA update waived the fee for submitting an sNDA, which is how an existing RLD holder would update their labeling with new indication information.

Conclusion

Patients need new and affordable treatment options for diseases that have a devastating societal impact, and repurposing generic drugs can help address this need. Nonprofits and other non-manufacturers are driving these efforts forward due to a lack of interest from pharmaceutical companies. As momentum gains for generic drug repurposing, the U.S. regulatory system needs a pathway for non-manufacturers to seek FDA approval of new indications for existing generic drugs. Our proposed labeling-only 505(b)(2) NDA would eliminate undue administrative burden, enabling non-manufacturers to pursue FDA approval of new indications. It would allow the FDA to provide the public with the most up-to-date drug labeling, improving the ability of patients and physicians to make informed treatment decisions. This dedicated pathway would increase the availability of effective treatment options while reducing costs for the American healthcare system.

This action-ready policy memo is part of Day One 2025 — our effort to bring forward bold policy ideas, grounded in science and evidence, that can tackle the country’s biggest challenges and bring us closer to the prosperous, equitable and safe future that we all hope for whoever takes office in 2025 and beyond.

Frequently Asked Questions
Could the FDA make labeling changes for repurposed generic drugs without the process being driven by non-manufacturer sponsors?

The FDA does not have sufficient bandwidth or resources to meet the opportunity we have with repurposed generic drugs. To be the primary driver of labeling changes for repurposed generic drugs, the FDA would need to identify repurposing opportunities and also thoroughly compile and evaluate the safety and effectiveness data for the new indications. Project Renewal in FDA’s Oncology Center of Excellence is working with RLD holders to update the labeling of certain older oncology drugs where the outdated labeling does not reflect their current clinical use. The initial focus of Project Renewal is limited, and newly repurposed treatments are not included within its scope. For newly repurposed treatments, the FDA could evaluate the drugs and post to the Federal Register reports on their safety and effectiveness that could be referenced by manufacturers. However, this approach is burdensome as it would require a significant commitment of FDA resources. By introducing a motivated, third-party non-manufacturer as the primary driver for labeling changes, non-manufacturers can contribute expertise and resources to enable faster data evaluation for more drugs.

If a repurposed indication for a generic drug was approved through the labeling-only 505(b)(2) NDA pathway, would current manufacturers be required to update their labeling?

The pre-existing NDA sponsor could update their labeling to add the new indication through an sNDA that references the labeling-only 505(b)(2) NDA. ANDA holders would then be legally required to match their labeling to that of the RLD. The FDA should determine whether all current manufacturers would be required to update their labeling following approval of the new indication, and if so, the appropriate process.

Could drug repurposing and expanding the market for generics cause an increase in drug prices?

Generic drugs play a vital role in the U.S. healthcare system by decreasing drug spending and increasing the accessibility of essential medicines. Generics account for 91% of prescriptions filled in the U.S. Expanding the market with generics for new indications could lead to short-term price increases above the inflation rate for off-patent branded and generic drugs in some unlikely circumstances. For example, if a new use for a generic drug substantially increases demand for the drug, there is a short-term risk that prices for the drug or potential substitutes could rise until manufacturers build more capacity to increase supply. To mitigate this risk, generic manufacturers could be notified about potential increases in demand so they can plan for increased production.

Would off-label uses of drugs still be covered by healthcare payers if there is a pathway to seek approval for those uses?

Generally, healthcare payers are not required to cover or reimburse for off-label uses of drugs. Unless a drug undergoes utilization management, payers cover most generic drugs for off-label uses because coverage is agnostic of indication. Clinical practice guidelines are highly influential in the widespread adoption of off-label treatments into the standard of care and are often referenced by payers making reimbursement decisions. In oncology, many off-label treatments are included in guidelines; only 62% of treatments in the NCCN are aligned with FDA-approved indications. For example, more than half of the NCCN recommendations for metastatic breast cancer are off-label treatments. Due to the breadth of off-label use, we anticipate payers would continue to cover repurposed generic drugs used off-label, even if there is a pathway available for non-manufacturers to pursue FDA approval.

Would a labeling-only 505(b)(2) NDA sponsor receive any marketing exclusivities for the new indications?

We do not envision any form of exclusivity being granted for indications pursued via a labeling-only 505(b)(2) NDA. Given that the non-manufacturer sponsor would rely on existing products produced by multiple generic manufacturers, there is no new product to grant exclusivity. Even if some form of exclusivity were given to the non-manufacturer, it would be insufficient to guarantee the use of any particular drug product over another due to pharmacy-level substitution.

An Innovation Agenda for Addiction: Breakthrough Medicines That Scale

The federal government should expand the FDA’s priority review voucher program (PRV) and provide market exclusivity advantages to encourage the development of medications for addiction. 

Taken together, substance use disorders (alcohol, cigarettes, and other drugs) cause more deaths in the U.S. every year than cancer or heart disease and cause devastating downstream social harms. Despite this, only 3% of eligible patients received substance use disorder (SUD) medication, a result of low uptake and efficacy of existing medications and a lack of options for patients addicted to stimulants. This is due to a near-total absence of pharmaceutical research and development activity. To make population level impact to reduce harms from opioids, methamphetamine, cocaine, alcohol, and cigarettes, we must address the broken market dynamics in addiction medicine. 

The PRV program should be expanded to cover opioid use disorder, alcohol use disorder, stimulant use disorder, and smoking. In addition, drugs that are approved for these SUD indications should have extended exclusivity and sponsors that develop these medications should receive vouchers to extend exclusivity for other medications.

Challenge and Opportunity 

Addiction policy efforts on both the left and the right have struggled. Despite substantial progress reducing smoking, 29 million Americans still smoke cigarettes and feel unable to quit and 480,000 Americans die each year from smoking. While overdose deaths from opioids, cocaine, and methamphetamine have fallen slightly from their peak in 2022, they are still near record highs, three  times higher than 20 years ago. Alcohol deaths per capita have doubled since 1999. 

Roughly 60% of all crimes and 65% of violent crimes are related to drugs or alcohol; and the opioid crisis alone costs the United States $1.5 trillion a year. Progress in reducing addiction is held back because people with a substance use disorder take medication. This low uptake has multiple causes: in opiate use disorder, uptake is persistently low despite recent relaxations of prescription rules, with patients reporting a variety of reasons for refusal; treatments for alcohol use disorder have modest effects; and there are no approved treatments for stimulant use disorder. Only three percent take SUD medications, as shown in figure 1 below [link to image]. In brief, only 2% of those suffering alcohol use disorder, 13% of those with opiate use disorder, 2% of smokers, and approximately 0% of illicit stimulant users are receiving medication, giving a weighted average of about 3%.

There has been rapid innovation in the illicit market as synthetic opioids and expanded meth production have lowered price and increased strength and availability. Meanwhile, there has been virtually no innovation in medicines to prevent and treat addiction. The last significant FDA approval for opioid use disorder was buprenorphine in 2002; progress since then has been minimal, with new formulations or dosing of old medications. For alcohol use disorder, the most recent was acamprosate in 2004 (and it is rarely prescribed due to limited efficacy and three times a day dosing).

None of the ten largest pharmaceutical companies have active addiction medicine programs or drug candidates, and the pharmaceutical industry as a whole has only pursued minimal drug development. According to the trade association BIO, “Venture investment into companies with novel addiction drug programs over the last 10 years is estimated at $130M, 270 times less than oncology.”

There are promising addiction drug candidates being studied by academics but without industry support they will never become medicines. If pharmaceutical companies spent just 10% of what they spend on obesity therapies, we might quickly make progress.

For example, GLP-1 medicines like Ozempic and Mounjaro have strong anti-addictive effects across substances. Randomized trials and real-world patient health record studies show dramatic drops in consumption of drugs and alcohol for patients taking a GLP-1. Many addiction scientists now consider these compounds to be the biggest breakthrough in decades. However, Novo Nordisk and Eli Lilly, who own the drugs currently in the market, do not plan to run phase 3 addiction trials on them, due to fear of adverse events in substance use disorder populations. The result is that a huge medical opportunity is stuck in limbo indefinitely. Fortunately, Lilly has recently signaled that they will run trials on related compounds, but remain years from approval.

Conversations with industry leaders make clear that large pharmas avoid SUD indications for several reasons. First, their upside appears limited, since current SUD medications have modest sales. Second, like other psychiatric disorders, the problem is challenging given the range and complexity of neurological targets and the logistical challenges of recruiting people with substance use disorder as participants. Finally, companies face downside reputational and regulatory risk if participants, who face high baseline rates of death from overdose regardless, were to die in trials. In the case of Ozempic and Mounjaro, sponsors face an obstacle some have termed the “problem of new uses” – clinical trials of an already lucrative drug for a new indication carry downside risk if new side effects or adverse events are reported. 

Image from Charting the fourth wave, based on CDC data

Plan of Action

Market Shaping Interventions

Recommendation 1. Expand the FDA priority review voucher (PRV) program to include addiction medicine indications.

The FDA priority review voucher (PRV) program incentivizes development of drugs for rare pediatric and infectious diseases by rewarding companies who get drugs approved with a transferable voucher that accelerates FDA approval. These vouchers are currently selling for an average of $100M. The PRV program doesn’t cost the government any money but it makes drug development in the designated categories much more lucrative. The PRV program has proven very successful, leading to a surge in approvals of medications.

As a neglected market with urgent unmet medical and public health needs, and which also promises to benefit the broader public by reducing the negative externalities of addiction, addiction medicine is a perfect fit for the PRV program. Doing so could transform the broken market dynamics of the field. The advantage of the PRV program is that it does not require substantial new congressional appropriations, though it will require Congress giving the FDA authority to expand the PRV program, as it has done previously to add other disease areas.

Recommendation 2. Extend exclusivity for addiction medicines and incentivize pursuit of new indications

Market exclusivity is a primary driver of pharmaceutical industry revenue. Extending exclusivities would have a very large effect on industry behavior and is needed to create sufficient incentives. The duration of exclusivity for alcohol use disorder, opioid use disorder, stimulant use disorder, and smoking cessation indications should be extended along with other incentives.  

For precedent, there are already a number of FDA programs that extend medication exclusivity, including ‘orphan drug exclusivity’ and the qualified infectious disease product (QIDP) program. Like rare diseases and antibiotics, addiction is a market that requires incentives to function effectively. In addition, successful treatments, given the negative externalities of addiction, have public benefit beyond the direct medical impact, and deserve additional public incentives.

Recommendation 3. Modernize FDA Standards of Efficacy for Substance Use Disorder Trials

A significant barrier to pharmaceutical innovation in SUDs is outdated or unpredictable efficacy standards sometimes set by the FDA for clinical trials. Efficacy expectations for substance use disorder indications are often rooted in abstinence-only and other binary measure orientations that the scientific and medical community has moved past when evaluating substance use disorder harms.

This article in the American Journal of Drug and Alcohol Abuse demonstrates that binary outcome measures like ‘number of heavy drinking days’ (NHDD) can underestimate the efficacy of treatments. This recent report from NIAAA on alcohol trial endpoints recommends a shift away from abstinence-based endpoints and towards more meaningful consumption-based endpoints. This approach should be adopted by the FDA for all SUD treatments, not just alcohol.

There are some indications that the FDA has begun modernizing their approach. This recent paper from NIH and FDA on smoking cessation therapies provides updated guidance that moves in the right direction.

More broadly, the FDA should work to adopt endpoints and standards of efficacy that mirror standards in other disease areas. This shift is best achieved through new guidance or statements issued by the FDA, which would offer positive assurance to pharmaceutical companies that they have achievable paths to approval. Predictability throughout the medication development life cycle is absolutely essential for companies considering investment.

Congress should include statements in upcoming appropriations and authorizations that state:

  1. The FDA should adopt non-binary standards of efficacy for addiction treatments that are aligned with standards for other common disorders and the FDA shall, within 12 months, report on the standards employed for substance use disorder relative to other prevalent chronic conditions and report steps to eliminate disparities in evidentiary standards and issue new guidance on the subject.
  2. The FDA should publish clear guidance on endpoints across SUDs to support planning among pharmaceutical companies considering work in this field.

Conclusion

Sustained focus and investment in diabetes and heart disease treatments has enabled medical breakthroughs. Addiction medicine, by contrast, has been largely stagnant for decades. Stimulating private-sector interest in addiction medicine through regulatory and exclusivity incentives, as well as modernized efficacy standards, is essential for disrupting the status quo. Breakthroughs in addiction medicine could save hundreds of thousands of lives in the US and provide long-term relief for one of our most intractable social problems. Given the negative externalities of addiction, this would also have enormous benefits for society at large, reducing crime and intergenerational trauma and saving money on social services and law enforcement.

This action-ready policy memo is part of Day One 2025 — our effort to bring forward bold policy ideas, grounded in science and evidence, that can tackle the country’s biggest challenges and bring us closer to the prosperous, equitable and safe future that we all hope for whoever takes office in 2025 and beyond.

Frequently Asked Questions
Why doesn’t the private sector target SUD? Why is government incentive necessary?

Per author conversations with industry leaders, private sector interest in SUD medication development is limited for the following reasons:



  • The upside of pursuing SUD indications appears limited, since current SUD medications, which are generally targeted for specific substances, have modest sales.

  • Even with preliminary evidence that GLP-1 drugs may be efficacious for some SUD indications (e.g, alcohol, opiates, and tobacco), companies are reluctant to pursue label expansion for SUD. As described previously, with already lucrative drugs, companies face a downside risk (termed the “problem of new uses”) from running large clinical trials, and possibly uncovering new side effects or incurring random adverse events which could harm reputation and existing markets.

  • In the specific case of SUD, this downside risk might be especially large, since people with substance use disorder have high baseline rates of overdose and death.


Moreover, there is an argument that a treatment for SUD is a public good, to the degree that it ameliorates the negative externalities of addiction – increasing the case for more public-sector incentives for SUD treatment. The end result is that medical treatments for SUD are stuck in an indefinite limbo, with private-sector interest in SUD, as documented previously, being very low.

Why are we optimistic about SUD medications?

The current lack of effective and widely used SUD medications is disheartening, but this is in the context of private sector disinterest and scant funding. Even modest successes in SUD treatment have the potential to kickstart an innovation loop, akin to the rush of biotech companies hastening to enter the obesity treatment field. Prior to the success of the GLP-1 drugs, obesity treatment had been moribund, and viewed pessimistically in light of drugs that had limited efficacy or had been withdrawn for side effects like suicidality or cardiovascular issues.


An SUD success like GLP-1 for obesity has the potential to kindle a similar rush of interest; the challenge is the initiation of that cascade. Given the very low levels of investment in SUD treatments, there is potential low-hanging fruit that, given sufficient funding, could be trialed and deployed.

What are the innovations in the illicit drug market?

There has been rapid innovation in the field of addiction, but it’s been happening on the wrong side: addiction-inducing technologies are becoming more powerful, while SUD treatments have largely stagnated. This innovation is most evident in synthetic opioids and methamphetamine.


Compared to heroin, fentanyl is about 25x stronger (on a per-weight basis), and hence, much easier to smuggle. As the Commission on Combating Synthetic Opioid Trafficking put it:


Single-digit metric tonnage of pure fentanyl is not a large amount and could easily fit into a shipping container or a truck trailer, which seriously challenges interdiction…Perhaps as much as 5 MT [metric tons] of pure fentanyl would be needed to satisfy the entire annual U.S. consumption for illegally supplied opioids.


Moreover, as a recent Scientific American article documented, innovations in fentanyl production, including the use of safer precursors and methods that don’t require sophisticated equipment, mean that fentanyl production is now decentralized, and resistant to attempts by law enforcement to shut it down.


As fentanyl has come to dominate the opioid supply over the past 10 years, overdose deaths have risen dramatically. New synthetic opioids and non-opioids like xylazine are also becoming common.


At the same time, due to advances in production techniques in Mexico, methamphetamine production has skyrocketed in recent decades while purity has improved. Worst of all, unlike heroin, fentanyl is easily combined with meth and cocaine in pills and powder.


The DEA has highlighted the presence of “super labs” in Mexico capable of producing hundreds of pounds of meth per batch.


Together, these three innovations (fentanyl, cheap meth, and new combinations) have led to a 400% increase in overdose deaths in the past 20 years. Without equally powerful innovations to reduce addiction rates, we will never make long-term and sustainable progress.

From Strategy to Impact: Establishing an AI Corps to Accelerate HHS Transformation

To unlock the full potential of artificial intelligence (AI) within the Department of Health and Human Services (HHS), an AI Corps should be established, embedding specialized AI experts within each of the department’s 10 agencies. HHS is uniquely positioned for—and urgently requires—this investment in AI expertise, as it plays a pivotal role in delivering efficient healthcare to millions of Americans. HHS’s responsibilities intersect with areas where AI has already shown great promise, including managing vast healthcare datasets, accelerating drug development, and combating healthcare fraud. 

Modeled after the success of the Department of Homeland Security (DHS)’s existing AI Corps, this program would recruit top-tier professionals with advanced expertise in AI, machine learning, data science, and data engineering to drive innovation within HHS. While current HHS initiatives like the AI Council and AI Community of Practice provide valuable strategic guidance, they fall short in delivering the on-the-ground expertise necessary for meaningful AI adoption across HHS agencies. The AI Corps would fill this gap, providing the hands-on, agency-level support necessary to move beyond strategy and into the impactful implementation intended by recent federal actions related to AI. 

This memo uses the Food and Drug Administration (FDA) as a case study to demonstrate how an AI Corps member could spearhead advancements within HHS’s agencies. However, the potential benefits extend across the department. For instance, at the Centers for Disease Control and Prevention (CDC), AI Corps experts could leverage machine learning for more precise outbreak modeling, enabling faster, more targeted public health responses. At the National Institutes of Health (NIH), they could accelerate biomedical research through AI-driven analysis of large-scale genomic and proteomic data. Similarly, at the Centers for Medicare and Medicaid Services (CMS), they could improve healthcare delivery by employing advanced algorithms for patient data analytics, predicting patient outcomes, and enhancing fraud detection mechanisms.

Challenge and Opportunity

AI is poised to revolutionize not only healthcare but also the broad spectrum of services under HHS, offering unprecedented opportunities to enhance patient outcomes, streamline administrative processes, improve public health surveillance, and advance biomedical research. Realizing these benefits and defending against potential harms demands the effective implementation and support of AI tools across HHS. The federal workforce, though committed and capable, currently lacks the specialized expertise needed to fully harness AI’s potential, risking a lag in AI adoption that could impede progress.

The public sector is responding well to this opportunity since it is well positioned to attract leading experts to help leverage new technologies. However, for federal agencies, attracting technical experts has been a perennial challenge, resulting in major setbacks in government tech projects: Of government software projects that cost more than $6 million, only 13% succeed. 

Without introducing a dedicated AI Corps, existing employees—many of whom lack specialized AI expertise—would be required to implement and manage complex AI tools alongside their regular duties. This could lead to the acquisition or development of AI solutions without proper evaluation of their suitability or effectiveness for specific use cases. Additionally, without the necessary expertise to oversee and monitor these systems, agencies may struggle to ensure they are functioning correctly and ethically. As a result, there could be significant inefficiencies, missed opportunities for impactful AI applications, and an increased reliance on external consultants who may not fully understand the unique challenges and needs of each agency. This scenario not only risks undermining the effectiveness of AI initiatives but also heightens the potential for errors, biases, and misuse of AI technologies, ultimately hindering HHS’s mission and objectives.

HHS’s AI Strategy recognizes the need for AI expertise in government; however, its focus has largely been on strategic oversight rather than the operational execution needed on the ground, with the planned establishment of an AI Council and AI Community of Practice prioritizing policy and coordination. While these entities are crucial, they do not address the immediate need for hands-on expertise within individual agencies. This leaves a critical gap in the hands-on expertise required to safely implement AI solutions at the agency level. HHS covers a wide breadth of functions, from administering national health insurance programs like Medicare and Medicaid to conducting advanced biomedical research at the NIH, with each agency facing distinct challenges where AI could provide transformative benefits. However, without dedicated support, AI adoption risks becoming fragmented, underutilized, or ineffective.

For example, at the CDC, AI could significantly improve infectious disease surveillance systems, enabling more timely interventions and enhancing the CDC’s overall preparedness for public health crises, moving beyond traditional methods that often rely on slower, manual analysis. Furthermore, the Administration for Children and Families (ACF) could leverage AI to better allocate resources, improve program outcomes, and support vulnerable populations more effectively. There are great opportunities to use machine learning algorithms to accelerate data processing and discovery in fields such as cancer genomics and personalized medicine. This could help researchers identify new biomarkers, optimize clinical trial designs, and push forward breakthroughs in medical research faster and more efficiently. However, without the right expertise, these game-changing opportunities could not only remain unrealized but also introduce significant risks. The potential for biased algorithms, privacy breaches, and misinterpretation of AI outputs poses serious concerns. Agency leaders may feel pressured to adopt technologies they don’t fully understand, leading to ineffective or even harmful implementations. Embedding AI experts within HHS agencies is essential to ensure that AI solutions are deployed responsibly, maximizing benefits while mitigating potential harms.

This gap presents an opportunity for the federal government to take decisive action. By recruiting and embedding top-tier AI professionals within each agency, HHS could ensure that AI is treated not as an ancillary task but as a core component of agency operations. These experts would bring the specialized knowledge necessary to integrate AI tools safely and effectively, optimize processes, and drive innovation within each agency.

DHS’s AI Corps, launched as part of the National AI Talent Surge, provides a strong precedent for recruiting AI specialists to advance departmental capabilities. For instance, AI Corps members have played a vital role in improving disaster response by using AI to quickly assess damage and allocate resources more effectively during crises. They have also enhanced cybersecurity efforts by using AI to detect vulnerabilities in critical U.S. government systems and networks. Building on these successes, a similar effort within HHS would ensure that AI adoption moves beyond a strategic objective to a practical implementation, with dedicated experts driving innovation across the department’s diverse functions.

Case Study: The Food and Drug Administration (FDA)

The FDA stands at the forefront of the biotechnology revolution, facing the dual challenges of rapid innovation and a massive influx of complex data. Advances in gene editing, personalized medicine, and AI-driven diagnostics promise to transform healthcare, but they also present significant regulatory hurdles. The current framework, though robust, struggles to keep pace with these innovations, risking delays in the approval and implementation of groundbreaking treatments.

This situation is reminiscent of the challenges faced in the 1980s and 1990s, when advances in pharmaceutical science outstripped the FDA’s capacity to review new drugs, leading to the so-called “drug lag.” The Prescription Drug User Fee Act of 1992 was a pivotal response, streamlining the drug review process by providing the FDA with additional resources. However, the continued reliance on scaling resources may not be sustainable as the complexity and volume of data increase.

The FDA has begun to address this new challenge. For example, the Center for Biologics Evaluation and Research has established committees like the Artificial Intelligence Coordinating Committee and the Regulatory Review AI Subcommittee. However, these efforts largely involve existing staff who must balance AI responsibilities with their regular duties, limiting the potential impact. Moreover, the focus has predominantly been on regulating AI rather than leveraging it to enhance regulatory processes.

Placing an AI expert from the HHS AI Corps within the FDA could fundamentally change this dynamic. By providing dedicated, expert support, the FDA could accelerate its regulatory review processes, ensuring timely and safe access to innovative treatments. The financial implications are significant: the value of accelerated drug approvals, as demonstrated by the worth of Priority Review Vouchers (acceleration of four months = ~$100 million), indicates that effective AI adoption could unlock billions of dollars in industry value while simultaneously improving public health outcomes.

Plan of Action

To address the challenges and seize the opportunities outlined earlier, the Office of the Chief Artificial Intelligence Officer (OCAIO) within HHS should establish an AI Corps composed of specialized experts in artificial intelligence, machine learning, data science, and data engineering. This initiative will be modeled after DHS’s successful AI Corps and tailored to the unique needs of HHS and its 10 agencies.

Recommendation 1. Establish an AI Corps within HHS.

Composition: The AI Corps would initially consist of 10 experts hired to temporary civil servant positions, with one member allocated to each of HHS’s 10 agencies, and each placement lasting one to two years. These experts will possess a range of technical skills—including AI, data science, data engineering, and cloud computing—tailored to each agency’s specific needs and technological maturity. This approach ensures that each agency has the appropriate expertise to effectively implement AI tools and methodologies, whether that involves building foundational data infrastructure or developing advanced AI applications.

Hiring authority: The DHS AI Corps utilized direct hiring authority, which was expanded by the Office of Personnel Management under the National AI Talent Surge. HHS’s AI Corps could adopt a similar approach. This authority would enable streamlined recruitment of individuals into specific AI roles, including positions in AI research, machine learning, and data science. This expedited process would allow HHS to quickly hire and onboard top-tier AI talent.

Oversight: The AI Corps would be overseen by the OCAIO, which would provide strategic direction and ensure alignment with HHS’s broader AI initiatives. The OCAIO would also be responsible for coordinating the activities of the AI Corps, setting performance goals, and evaluating outcomes.

Budget and Funding

Estimated cost: The AI Corps is projected to cost approximately $1.5 million per year, based on an average salary of $150,000 per corps member. This estimate includes salaries and operational costs such as training, travel for interagency collaboration, and participation in conferences. 

Funding source: Funding would be sourced from the existing HHS budget, specifically from allocations set aside for digital transformation and innovation. Given the relatively modest budget required, reallocation within these existing funds should be sufficient. 

Recruitment and Training

Selection process: AI Corps members would be recruited through a competitive process, targeting individuals with proven expertise in AI, data science, and related fields. 

Training: Upon selection, AI Corps members would undergo an intensive orientation and training program to familiarize them with the specific needs and challenges of HHS’s various agencies. This also includes training on federal regulations, ethics, and data governance to ensure that AI applications comply with existing laws and policies.

Agency Integration

Deployment: Each AI Corps member would be embedded within a specific HHS agency, where they would work closely with agency leadership and staff to identify opportunities for AI implementation. Their primary responsibility would be to develop and deploy AI tools that enhance the agency’s mission-critical processes. For example, an AI Corps member embedded at the CDC could focus on improving disease surveillance systems through AI-driven predictive analytics, while a member at the NIH could drive advancements in biomedical research by using machine learning algorithms to analyze complex genomic data.

Collaboration: To ensure cross-agency learning and collaboration, AI Corps members would convene regularly to share insights, challenges, and successes. These convenings would be aligned with the existing AI Community of Practice meetings, fostering a broader exchange of knowledge and best practices across the department.

Case Study: The FDA

AI Corps Integration at the FDA

Location: The AI Corps member assigned to the FDA would be based in the Office of Digital Transformation, reporting directly to the chief information officer. This strategic placement would enable the expert to work closely with the FDA’s leadership team, ensuring that AI initiatives are aligned with the agency’s overall digital strategy.

Key responsibilities

Process improvement: The AI Corps member would collaborate with FDA reviewers to identify opportunities for AI to streamline regulatory review processes. This might include developing AI tools to assist with data analysis, automate routine tasks, or enhance decision-making capabilities.

Opportunity scoping: The expert would engage with FDA staff to understand their workflows, challenges, and data needs. Based on these insights, the AI Corps member would scope and propose AI solutions tailored to the FDA’s specific requirements.

Pilot projects: The AI Corps member would lead pilot projects to test AI tools in real-world scenarios, gathering data and feedback to refine and scale successful initiatives across the agency.

Conclusion

Establishing an AI Corps within HHS is a critical step toward harnessing AI’s full potential to enhance outcomes and operational efficiency across federal health agencies. By embedding dedicated AI experts within each agency, HHS can accelerate the adoption of innovative AI solutions, address current implementation gaps, and proactively respond to the evolving demands of the health landscape.

While HHS may currently have less technological infrastructure compared to departments like the Department of Homeland Security, targeted investment in in-house expertise is key to bridging that gap. The proposed AI Corps not only empowers agencies like the FDA, CDC, NIH, and CMS to enhance their missions but also sets a precedent for effective AI integration across the federal government. Prompt action to establish the AI Corps will position HHS at the forefront of technological innovation, delivering tangible benefits to the American public and transforming the way it delivers services and fulfills its mission.

This action-ready policy memo is part of Day One 2025 — our effort to bring forward bold policy ideas, grounded in science and evidence, that can tackle the country’s biggest challenges and bring us closer to the prosperous, equitable and safe future that we all hope for whoever takes office in 2025 and beyond.

Frequently Asked Questions
How will the AI Corps avoid becoming just another bureaucratic layer?

The AI Corps is designed to be the opposite of bureaucracy—it’s about action, not administration. These experts will be embedded directly within agencies, working alongside existing teams to solve real-world problems, not adding paperwork. Their mission is to integrate AI into daily operations, making processes more efficient and outcomes more impactful. By focusing on tangible results and measurable improvements, the AI Corps will be judged by its ability to cut through red tape, not create it.

What if AI Corps members are too ahead of the curve for existing agency cultures?

Innovation can present challenges, but the AI Corps is designed to address them effectively. These experts will not only bring technical expertise but also serve as facilitators who can translate advanced AI capabilities into practical applications that align with existing agency cultures. A key part of their role will be to make AI more accessible and understandable, ensuring it is valuable to all levels of staff, from frontline workers to senior leadership. Their success will depend on their ability to seamlessly integrate advanced technology into the agency’s everyday operations.

Why focus on AI when there are so many other pressing health issues?

AI isn’t just another tool; it’s a force multiplier that can help solve those other pressing issues more effectively. Whether it’s accelerating drug approvals at the FDA or enhancing public health responses across HHS, AI has the potential to improve outcomes, save time, and reduce costs. By embedding AI experts within agencies, we’re not just addressing one problem—we’re empowering the entire department to tackle multiple challenges with greater efficiency and impact.

What’s in it for the AI experts? Why would top talent join the AI Corps?

For top AI talent, the AI Corps offers a unique opportunity to make a difference at a scale that few private-sector roles can match. It’s a chance to apply their skills to public service, tackling some of the nation’s most critical challenges in healthcare, regulation, and beyond. The AI Corps members will have the opportunity to shape the future of AI in government, leaving a legacy of innovation and impact. The allure of making a tangible difference in people’s lives can be a powerful motivator for the right kind of talent.

Why not outsource AI talent or rely on consultants instead of building in-house expertise?

While outsourcing AI talent or using consultants can offer short-term benefits, it often lacks the sustained engagement necessary for long-term success. Building in-house expertise through the AI Corps ensures that AI capabilities are deeply integrated into the agency’s operations and culture. A notable example illustrating the risks of overreliance on external contractors is the initial rollout of HealthCare.gov. The website faced significant technical issues at launch due to coordination challenges and insufficient in-house technical oversight, which hindered public access to essential healthcare services. In contrast, recent successful government initiatives—such as the efficient distribution of COVID-19 test kits and the timely processing of economic stimulus payments directly into bank accounts—demonstrate the positive impact of having the right technical experts within government agencies.

How will the AI Corps collaborate with existing IT and data teams within agencies?

Collaboration is crucial to the AI Corps’ success. Instead of working in isolation, AI Corps members will integrate with existing IT and data teams, bringing specialized AI knowledge that complements the teams’ expertise. This partnership approach ensures that AI initiatives are well-grounded in the agencies’ existing infrastructure and aligned with ongoing IT projects. The AI Corps will serve as a catalyst, amplifying the capabilities of existing teams rather than duplicating their efforts.

Could the AI Corps inadvertently lead to job displacement within agencies?

The AI Corps is focused on augmentation, not replacement. The primary goal is to empower existing staff with advanced tools and processes, enhancing their work rather than replacing them. AI Corps members will collaborate closely with agency employees to automate routine tasks and free up time for more meaningful activities. A 2021 study by the Boston Consulting Group found that 60% of employees view AI as a coworker rather than a replacement. This reflects the intent of the AI Corps—to build capacity within agencies and ensure that AI is a tool that amplifies human effort, fostering a more efficient and effective workforce.

What does success look like for the HHS AI Corps program after one or two years?

Success for the AI Corps program means that each HHS agency has made measurable progress toward integrating AI and related technologies, tailored to their specific needs and maturity levels. Within one to two years, agencies might have established robust data infrastructures, migrated platforms to the cloud, or developed pilot AI projects that address key challenges. Success also includes fostering a culture of innovation and experimentation, with AI Corps members identifying opportunities and creating proofs of concept in low-risk environments. By collaborating across agencies, these experts support each other and amplify the program’s impact. Ultimately, success is reflected in enhanced capabilities and efficiencies within agencies, setting a strong foundation for ongoing technological advancement aligned with each agency’s mission.

Creating an HHS Loan Program Office to Fill Critical Gaps in Life Science and Health Financing

We propose the establishment of a Department of Health and Human Services Loan Programs Office (HHS LPO) to fill critical and systematic gaps in financing that prevent innovative life-saving medicines and other critical health technologies from reaching patients, improving health outcomes, and bolstering our public health. To be effective, the HHS LPO requires an authority to issue or guarantee loans, up to $5 billion in total. Federally financed debt can help fill critical funding gaps and complement ongoing federal grants, contracts, reimbursement, and regulatory policies and catalyze private-sector investment in innovation.

Challenge and Opportunity

Despite recent advances in the biological understanding of human diseases and a rapidly advancing technological toolbox, commercialization of innovative life-saving medicines and critical health technologies face enormous headwinds. This is due in part to the difficulty in accessing sustained financing across the entire development lifecycle. Further, macroeconomic trends such as non-zero interest rates have substantially reduced deployed capital from venture capital and private equity, especially with longer investment horizons. 

The average new medicine requires 15 years and over $2 billion to go from the earliest stages of discovery to widespread clinical deployment. Over the last 20 years, the earliest and riskiest portions of the drug discovery process have shifted from the province of large pharmaceutical companies to a patchwork of researchers, entrepreneurs, venture capitalists, and supporting organizations. While this trend has enabled new entrants into the biotechnology landscape, it has also required startup companies to navigate labyrinthine processes of technical regulatory guidelines, obtaining long-term and risk-friendly financing, and predicting payor and provider willingness to ultimately adopt the product.

Additionally, there are major gaps in healthcare infrastructure such as lack of adequate drug manufacturing capacity, healthcare worker shortages, and declining rural hospitals. Limited investment is available for critical infrastructure to support telehealth, rural healthcare settings, biomanufacturing, and decentralized clinical trials, among others.

The challenges in health share some similarities to other highly regulated, capital-intensive industries, such as energy. The Department of Energy (DOE) Loan Program Office (LPO) was created in 2005 to offer loans and loan guarantees to support businesses in deploying innovative clean energy, advanced transportation, and tribal energy projects in the United States. LPO has closed more than $40 billion in deals to date. While agencies across HHS rely primarily on grants and contracts to deploy research and development (R&D) funding, capital-intensive projects are best deployed as loans, not only to appropriately balance risk between the government and lendees but also to provide better stewardship over taxpayer resources via mechanisms that create liquidity with lower budget impact. Moreover, private-sector financing rates are subject to market-based interest rates, which can have enormous impacts on available capital for R&D.

Plan of Action

There are many federal credit programs across multiple departments and agencies that provide a strong blueprint for the HHS LPO to follow. Examples include the aforementioned DOE Loan Programs Office, which provides capital to scale large-scale energy infrastructure projects using new technologies, and the Small Business Administration’s credit programs, which provide credit financing to small businesses via several loan and loan matching programs.

Proposed Actions

We propose the following three actions:

Scope

Similar to how DOE LPO services the priorities of the DOE, the HHS LPO would develop strategy priorities based on market gaps and public health gaps. It would also develop a rigorous diligence process to prioritize, solicit, assess, and manage potential deals, in alignment with the Federal Credit Reform Act and the associated policies set forth by the Office of Management and Budget and followed by all federal credit programs. It would also seek companion equity investors and creditors from the private sector to create leverage and would provide portfolio support via demand-alignment and -generation mechanisms (e.g., advance manufacturing commitments and advanced market commitments from insurers).

We envision several possible areas of focus for the HHS LPO:

  1. Providing loans or loan guarantees to amplify investment funds that use venture capital or other private investment tools, such as early-stage drug development or biomanufacturing capacity. While these funds may already exist, they are typically underpowered.
  2. Providing large-scale financing in partnership with private investors to fund major healthcare infrastructure gaps, such as rural hospitals, decentralized clinical trial capacity, telehealth services, and advanced biomanufacturing capacity.
  3. Providing financing to test new innovative finance models, e.g. portfolio-based R&D bonds, designed to attract additional capital into under-funded R&D and lower financial risks.

Conclusion

To address the challenges in bringing innovative life-saving medicines and critical health technologies to market, we need an HHS Loan Programs Office that would not only create liquidity by providing or guaranteeing critical financing for capital-intensive projects but address critical gaps in the innovation pipeline, including treatments for rare diseases, underserved communities, biomanufacturing, and healthcare infrastructure. Finally, it would be uniquely positioned to pilot innovative financing mechanisms in partnership with the private sector to better align private capital towards public health goals.

This action-ready policy memo is part of Day One 2025 — our effort to bring forward bold policy ideas, grounded in science and evidence, that can tackle the country’s biggest challenges and bring us closer to the prosperous, equitable and safe future that we all hope for whoever takes office in 2025 and beyond.

Frequently Asked Questions
What is the DOE Loan Programs Office, and how is it similar to the proposed HHS Loan Programs Office?

The DOE LPO, enabled via the Energy Policy Act of 2005, enables the Secretary of Energy to provide loan guarantees toward publicly or privately financed projects involving new and innovative energy technologies.


The DOE LPO provides a bridge to private financing and bankability for large-scale, high-impact clean energy and supply chain projects involving new and innovative technologies. It also expands manufacturing capacity and energy access within the United States. The DOE LPO has enabled companies involving energy and energy manufacturing technologies to achieve infrastructure-scale growth, including Tesla, an electric car manufacturer; Lithium Americas Corp., a company supplying lithium for batteries; and the Agua Caliente Solar Project, a solar power station sponsored by NRG Solar that was the largest in the world at its time of construction.


The HHS LPO would similarly augment, guarantee, or bridge to private financing for projects involving the development and deployment of new and innovative technologies in life sciences and healthcare. It would draw upon the structure and authority of the DOE LPO as its basis.

What potential use cases would the HHS LPO serve?

The HHS LPO could look to the DOE LPO for examples as to how to structure potential funds or use cases. The DOE LPO’s Title 17 Clean Energy Financing Program provides four eligible project categories: (1) projects deploying new or significantly improved technology; (2) projects manufacturing products representing new or significantly improved technologies; (3) projects receiving credit or financing from counterpart state-level institutions; and (4) projects involving existing infrastructure that also share benefits to customers or associated communities.


Drawing on these examples, the HHS LPO could support project categories such as (1) emerging health and life science technologies; (2) the commercialization and scaling access of novel technologies; and (3) expanding biomanufacturing capacity in the United States, particularly for novel platforms (e.g., cell and gene therapies).

How much would the HHS LPO cost?

The budget could be estimated via its authority to make or guarantee loans. Presently, the DOE LPO has over $400 billion in loan authority and is actively managing a portfolio of just over $30 billion. Given this benchmark and the size of the private market for early-stage healthcare venture capital valued at approximately $20 billion, we encourage the creation of an HHS LPO with $5 billion in loan-making authority. Using proportional volume to the $180 million sought by DOE LPO in FY2023, we estimate that an HHS LPO with $5 billion in loan-making authority would require a budget appropriation of $30 million.

What accountability or oversight measures are required to ensure proper operation and evaluate performance?

The HHS LPO would be subject to oversight by the HHS Inspector General, OMB, as well as the respective legislative bodies, the House of Representatives Energy and Commerce Committee and the Senate Health, Education, Labor and Pension Committee.


Like the DOE LPO, the HHS LPO would publish an Annual Portfolio Status Report detailing its new investments, existing portfolio, and other key financial and operational metrics.

What alternative options could serve the same purpose as the HHS LPO, and why is the HHS LPO preferable?

It is also possible for Congress to authorize existing funding agencies, such as BARDA, the Advanced Research Projects Agency for Health (ARPA-H), or the National Institutes for Health (NIH), with loan authority. However, due the highly specialized talent needed to effectively operate a complex loan financing operation, the program is significantly more likely to succeed if housed into a dedicated HHS LPO that would then work closely with the other health-focused funding agencies within HHS.


The other alternative is to expand the authority for other LPOs and financing agencies, such as the DOE LPO or the U.S. Development Finance Corporation, to focus on domestic health. However, that is likely to create conflicts of priority given their already large and diverse portfolios.

What are the next steps required to stand up the HHS LPO?

The project requires legislation similar to the Department of Energy’s Title 17 Clean Energy Financing Program, created via the Energy Policy Act of 2005 and subsequently expanded via the Infrastructure Investment and Jobs Act in 2021 and the Inflation Reduction Act in 2022.


This legislation would direct the HHS to establish an office, presumably a Loan Programs Office, to make loan guarantees to support new and innovative technologies in life sciences and healthcare. While the LPO could reside within an existing HHS division, the LPO would most ideally be established in a manner that enables it to serve projects across the full Department, including those from the National Institutes of Health, Food and Drug Administration, Biomedical Advanced Research and Development Authority, and the Centers for Medicare and Medicaid Services. As such, it would preferably not reside within any single one of these organizations. Like the DOE LPO, the HHS LPO would be led by a director, who would be directed to hire the necessary finance, technical, and operational experts for the function of the office.


Drawing on the Energy Policy Act of 2005 that created the DOE LPO, enabling legislation for the HHS Loan Programs office would direct the Secretary of HHS to make loan guarantees in consultation with the Secretary of Treasury toward projects involving new and innovative technologies in healthcare and life sciences. The enabling legislation would include several provisions:



  • Necessary included terms and conditions for loan guarantees created via the HHS LPO, including loan length, interest rates, and default provisions;

  • Allowance of fees to be captured via the HHS LPO to provide funding support for the program; and

  • A list of eligible project types for loan guarantees.

Who are potential supporters of this policy? Who are potential skeptics?

Supporters are likely to include companies developing and deploying life sciences and healthcare technologies, including early-stage biotechnology research companies, biomanufacturing companies, and healthcare technology companies. Similarly, patient advocates would be similarly supportive because of the LPO’s potential to bring new technologies to market and reduce the overall Weighted Average Cost of Capital (WACC) for biotechnology companies, potentially supporting expanded access.


Existing financiers of research in biomedical sciences technology may be neutral or ambivalent toward this policy. On one hand, it would provide expanded access to syndicated financing or loan guarantees that would compound the impact of each dollar invested. On the other hand, most financiers currently use equity financing, which enables the demand for a high rate of return via subsequent investment and operation. An LPO could provide financing that requires a lower rate of return, thereby diluting the impact of current financiers in the market.


Skeptics are likely to include groups opposing expansions of government spending, particularly involving higher-risk mechanisms like loan guarantees. The DOE LPO has drawn the attention of several such skeptics, oftentimes leading to increased levels of oversight from legislative stakeholders. The HHS LPO could expect similar opposition. Other skeptics may include companies with existing medicines and healthcare technologies, who may be worried about competitors introducing products with prices and access provisions that have been enabled via financing with lower WACC.

The Medicare Advance Healthcare Directive Enrollment (MAHDE) Initiative: Supporting Advance Care Planning for Older Medicare Beneficiaries

Taking time to plan and document a loved one’s preferences for medical treatment and end-of-life care helps respect and communicate their wishes to doctors while reducing unnecessary costs and anxiety. There is currently no federal policy requiring anyone, including Medicare beneficiaries, to complete an Advance Healthcare Directive (AHCD), which documents an individual’s preferences for medical treatment and end-of-life care. At least 40% of Medicare beneficiaries do not have a documented AHCD. In the absence of one, medical professionals may perform major and costly interventions unknowingly against a patient’s wishes. 

To address this gap, the Centers for Medicare and Medicaid Services (CMS) should launch the Medicare Advance Healthcare Directive Enrollment (MAHDE) Initiative to support all adults over age 65 who are enrolled in Medicare or Medicare Advantage plans to complete and annually renew, at no extra cost, an electronic AHCD made available and stored on Medicare.gov or an alternative secure digital platform. MAHDE would streamline the process and make it easier for Medicare enrollees to complete and store directives and for healthcare providers to access them when needed. CMS could also work with the National Committee for Quality Assurance (NCQA) to expand Advance Care Planning (ACP) Healthcare Effectiveness Data and Information Set (HEDIS) measures to include all Medicare Advantage plans caring for beneficiaries aged 65 and older. 

AHCDs save families unnecessary heartache and confusion at times of great pain and vulnerability. They also aim to improve healthcare decision-making, patient autonomy, and function as a long-term cost-saving strategy by limiting undesired medical interventions among older adults. 

Challenge and Opportunity

Advance healthcare directives document an individual’s preferences for medical treatment in medical emergencies or at the end of life. 

AHCDs typically include two parts: 

  1. Identifying a healthcare proxy or durable power of attorney, who will make decisions about an individual’s health when they are unable to.
  2. A living will, which describes the treatments an individual wants to receive in emergencies—such as CPR, breathing machines, and dialysis—as well as decisions on organ and tissue donation. 

Other documents complement AHCDs and help communicate treatment wishes during emergencies or at the end of life. These include do-not-resuscitate orders, do-not-hospitalize  orders, and Physician (or Medical) Orders for Life-Sustaining Treatment forms, along with similar portable medical order forms for seriously ill or frail individuals (e.g., Medical Orders for Scope of Treatment, Physician Orders for Scope of Treatment, and Transportable Physician Orders for Patient Preferences). These forms are all designed to honor an individual’s healthcare preferences in a future medical emergency.

With the U.S. aging population projected to reach more than 20% of the total population by 2030, addressing end-of-life care challenges is increasingly urgent. As people age, their healthcare needs become more complex and expensive. Notably, 25% of all Medicare spending goes toward treating people in the last 12 months of their life. However, despite commonly receiving more aggressive treatments, many older adults prefer less intensive medical interventions and prioritize quality of life over prolonging life. This discrepancy between care received and a patient’s wishes is common, highlighting the need for clear and proactive communication and planning around medical preferences. Research shows patients with ACPs are less likely to receive unwanted and aggressive treatments in their last weeks of life, are more likely to enroll in hospice for comfort-focused care, and are less likely to die in hospitals or intensive care units.  

Established ACP Policies and Support Mechanisms

Historically, some federal policies have underscored the importance of patient decision-making rights and the role of AHCDs in helping patients receive their desired care. These policies reflect the ongoing effort to empower patients to make informed decisions about their healthcare, particularly in end-of-life situations. 

The Patient Self-Determination Act (PSDA), a federal law introduced in 1990 as a part of the Omnibus Budget Reconciliation Act, was created to ensure that patients are informed of their rights regarding medical care and their ability to make decisions about that care, especially in situations where they are no longer able to make decisions for themselves. 

The Act requires hospitals, skilled nursing facilities (SNFs), home health agencies, hospice programs, and health maintenance organizations to: 

  1. Inform patients of their rights to make decisions under state law about their medical care, including accepting or refusing treatment.
  2. Periodically inquire whether a patient has completed a legally valid AHCD and make note in their medical record.  
  3. Not discriminate against patients who do or do not have an advance directive. 
  4. Ensure AHCDs and other documented care wishes are carried out, as permitted by state law. 
  5. Provide education to staff, patients, and the community about AHCDs and the right to make their own medical decisions. 

It also directs the Secretary of Health and Human Services to research and assess the implementation of this law and its impact on health decision-making. Additionally, to encourage physicians and qualified health professionals to facilitate ACP conversations and complete AHCDs, CMS introduced and approved two new billing codes in 2016, allowing qualified health providers to bill CMS for advance care planning as a separate service regardless of diagnosis, place of service, or how often services are needed (Figure 1). These codes were expanded in 2017 with the temporary Healthcare Common Procedure Coding System code G0505, followed by CPT code 99483, to offer care planning and cognitive assessment services that include advance care planning for Medicare beneficiaries with cognitive impairment.

Figure 1. Two primary CPT codes and billing descriptors for advance care planning reimbursement. (Source: CMS Medicare Learning Network Fact Sheet)

Figure 1. Two primary CPT codes and billing descriptors for advance care planning reimbursement. (Source: CMS Medicare Learning Network Fact Sheet)

In 2022, ACP was introduced as one of four key components of the Care for Older Adults (COA) initiative within the Healthcare Effectiveness Data and Information Set measures. HEDIS is a proprietary set of clinical care performance measures developed by the National Committee for Quality Assurance (NCQA), a private, nonprofit accreditation organization that creates standardized measures to help health plans assess and report on the quality of care and services. HEDIS evaluates areas such as chronic disease management, preventive care, and care utilization, enabling reliable comparisons of health plan performance and identifying areas for improvement. It is reported that 235 million Americans are enrolled in plans that report HEDIS results, and reporting HEDIS measures is mandatory for Medicare Advantage plans.

The COA initiative includes the ACP measure as a reporting requirement for Medicare Special Needs Plans (SNPs), which are plans designed for individuals who have complex care needs, are eligible for Medicare and Medicaid, have disabling or chronic conditions, and/or live in an institution. The report includes the percentage of Medicare Advantage members within a specified population who participate in ACP discussions each year. This population currently includes:

HEDIS measures contribute to the overall STAR rating of Medicare Advantage and other health plans, which helps beneficiaries choose high-quality plans, enables highly rated plans to attract more members, and influences the funding and bonuses CMS provides to these plans.

Despite the PSDA, CMS provider reimbursement codes that incentivize physicians and qualified health professionals to facilitate advance care planning, and its recent inclusion in HEDIS measures for Medicare Special Needs Plans, there remain many barriers to completing AHCDs.

Barriers to AHCD Completion 

Although Medicare provides health and financial security to nearly all Americans aged 65 and older, completing a comprehensive AHCD is not universally expected within this population. Conversations about treatment decisions in future emergencies and end-of-life care are often avoided for various cultural, religious, financial, and mental health reasons. When they do happen, preferences are more often shared with loved ones but not documented or communicated to healthcare professionals. It is perhaps unsurprising, then, that only about half of Medicare beneficiaries have completed an AHCD. Studies show that of those who have, most do so in conjunction with estate planning, which may explain increasing cultural and socioeconomic disparities in the completion of AHCDs. 

For Medicare beneficiaries who wish to complete an AHCD with a physician or qualified health professional, Medicare only covers planning as part of the annual wellness visit. If ACP is provided outside of this visit, the beneficiary must meet the Medicare Part B deductible, which is $240 in 2024, before coverage begins. If the deductible has not been met through other Part B services (such as doctor visits, preventive care, mental health services, or outpatient procedures), the beneficiary is responsible for the deductible and a 20% coinsurance payment. Additionally, some states may require attorney services or notarization to legally validate an AHCD, which could incur extra costs.

These additional costs can make it challenging for many Medicare beneficiaries to complete an AHCD when they want to. Furthermore, depending on the complexity of their situation and readiness to make decisions, many patients may need more than one visit with their clinical provider to make decisions about critical illness and end of life care, creating more out-of-pocket expenses. 

AHCDs can also vary widely, are not uniform across states, and are often stored in paper formats that can be easily lost or damaged, or are embedded in bulky, multipage estate plans. Efforts to centralize AHCDs have been made through state-based AHCD registries, but their availability and management vary significantly and there is limited data on their use and effectiveness. Additionally, private ACP programs through initiatives like the Uniform Health-Care Decisions Act (UHCDA), the Five Wishes program, MyDirectives, and the U.S. Advance Care Plan Registry (USACPR), among others, have contributed to broadening ACP accessibility and awareness. However, data from private ACP programs are not widely published or have shown variable results. The UHCDA, first drafted and approved in 1993 by the Uniform Law Commission—a nonprofit organization focused on promoting consistency in state laws—was updated in 2023 and aims to address these variations in state AHCD policies, however with varying degrees of success. The Five Wishes program reports 40 million copies of their paper and digital advance directives in circulation nationwide, and their digital program recently launched a partnership with MyDirectives, a leader in digital advance care planning, to facilitate electronic access to legally recognized ACP documents. Unfortunately, data on completion and storage of these directives is not consistently reported across all users. 

Despite efforts by numerous organizations to improve ACP completion, access, and usability, the lack of updated federal policy supporting advance care planning makes it difficult for patients to complete them and healthcare providers to quickly locate and interpret them in critical situations. When AHCDs are not available, incomplete, or hard to find, medical professionals may be unaware of patients’ care preferences during urgent moments, leading to treatment decisions that may not align with the patients’ wishes.

Plan of Action

To support all Medicare beneficiaries aged 65 and older in documenting their end-of-life care preferences, encourage the completion of AHCDs, and improve accessibility of AHCDs for healthcare professionals, CMS should launch the Medicare Advance Healthcare Directive Enrollment Initiative to focus on the following four interventions.

Recommendation 1. Streamline the process of AHCD completion and electronic storage during open enrollment through Medicare.gov or an alternative CMS-approved secure ACP digital platform. 

To provide more clarity and support to fulfill patients’ wishes in their end-of-life care, CMS should empower all adults over the age of 65 enrolled in Medicare and Medicare Advantage plans to complete an electronic AHCD and renew it annually, at no extra cost, during Medicare’s designated open enrollment period. Though electronic completion is preferred, paper options will continue to be available and can be submitted for electronic upload and storage. 

Supporting the completion of an AHCD during open enrollment presents a strategic opportunity to integrate AHCD completion into overall discussions about healthcare options. New open enrollment tools can be made easily available on Medicare.gov or in partnership with an existing digital ACP platform such as the USACPR, the newly established Five Wishes and MyDirectives partnership, or a centralized repository of state registries, enabling beneficiaries to complete and safely store their directives electronically. User-friendly tools and resources should be tailored to guide beneficiaries through the process and should be age-appropriate and culturally sensitive. 

Building on this approach, some states are also taking steps to integrate electronic ACP completion and storage into healthcare enrollment processes. For example, in 2022, Maryland unanimously passed legislation that mandates payers to offer ACP options to all members during open enrollment and at regular intervals thereafter. It also requires payers to receive notifications on the completion and updates of ACP documents. Additionally, providers are required to use an electronic platform to create, upload, and store AHCDs.

An annual electronic renewal process during open enrollment would allow Medicare beneficiaries to review their own selections and make appropriate changes to ensure their choices are up to date. The annual review will also allow for educational opportunities around the risks and benefits of life-extending efforts through the secure Medicare enrollment portal and is a time interval that accounts for the abrupt changes in health status as individuals age. The electronic enhancements also provide a better fit with the modern technological healthcare landscape and can be completed in person or via a telehealth ACP visit with a physician or qualified health professional. Updates to AHCDs can also be made at any time outside of the open enrollment period. 

CMS could also work across state lines and in collaboration with private ACP organizations, the UHCDA, and state-appointed AHCD representatives to develop a universal template for advance directives that would be acceptable nationwide. Alternatively, Medicare.gov could provide tailored, state-specific electronic forms to meet state legal requirements, like the downloadable forms provided by organizations such as AARP, a nonprofit, nonpartisan organization for Americans over 50, and CaringInfo, a program of the National Hospice and Palliative Care Organization. Either approach would ensure AHCDs are legally compliant while centralizing access to the correct forms for easy completion and secure electronic storage.

Recommendation 2. Remove barriers to access advance care planning services. 

CMS should remove the deductible and 20% coinsurance when beneficiaries engage in voluntary ACP services with a physician or other qualified health professional outside of their yearly wellness visit. 

The current deductible and coinsurance requirements may discourage participants from completing their AHCDs with the guidance of a medical provider, as these costs can be prohibitive. This is similar to how higher cost-sharing and out-of-pocket health expenses often result in cost-related nonadherence, reducing healthcare engagement and prescription medication adherence. When individuals face higher out-of-pocket costs for care, they are more likely to delay treatments, avoid doctor visits, and fill fewer prescriptions, even if they have insurance coverage. Removing deductibles and coinsurance for ACP visits would allow individuals to complete or update their AHCDs as needed, without financial strain and with support from their clinical team, like preventive services.

Additionally, CMS could consider continued health provider education on facilitating ACPs and partnership with organizations like Institute of Healthcare Improvement’s The Conversation Project, which encourages open discussions about end-of-life care preferences. Partnering with Evolent (formerly Vital Decisions) could also support ongoing telehealth discussions between behavioral health specialists and older adults, focusing on late-life and end-of-life care preferences to encourage formal AHCD completion. Internal studies of the Evolent program, aimed at Medicare Advantage beneficiaries, demonstrated an average savings of $13,956 in the final six months of life and projected a potential Medicare spending reduction of up to $8.3 billion.

These enhancements recognize advance care planning as an ongoing process of discussion  and documentation that ensures a patient’s care and interventions reflect their values, beliefs, and preferences when unable to make decisions for themselves. It also emphasizes that goals of care are dynamic, and as they evolve, beneficiaries should feel supported and empowered to update their AHCDs affordably and with guidance from educational tools and trained professionals when needed.

Recommendation 3. Ensure electronic accessibility for healthcare providers.

CMS should also integrate the Medicare.gov AHCD storage system or a CMS-approved alternative with existing electronic health records (EHRs).

EHR systems in the United States currently lack full interoperability, meaning that when patients move through the continuum of care—from preventive services to medical treatment, rehabilitation, ongoing care maintenance—and between healthcare systems, their medical records, including AHCDs, may not transfer with them. This makes it challenging for healthcare providers to efficiently access these directives and deliver care that aligns with a patient’s wishes when the patient is incapacitated. To address this, CMS could encourage the integration of the Medicare.gov AHCD storage system or an alternative CMS-approved secure ACP digital platform to interface with all EHRs.

This storage platform could operate as an external add-on feature, allowing AHCDs to be accessible through any EHR, regardless of the healthcare system. Such external add-ons are typically third-party tools or modules that integrate with existing EHR systems to extend functionality, often addressing needs not covered by the core system. These add-ons are commonly used to connect EHRs with tools like clinical decision support systems, telehealth platforms, health information exchanges, and patient communication tools.

Such a universal, electronic system would prevent AHCDs from being misplaced, make them easily accessible across different states and health systems, and allow for easy updates. This would ensure that Medicare beneficiaries’ end-of-life care preferences are consistently honored, regardless of where they receive care. 

Recommendation 4. Provide financial incentives for AHCD completion.

CMS should offer financial incentives for completing an AHCD, including options like tax credits, reduced or waived copayments and deductibles, prescription rebates, or other health-related subsidies.

Medicare’s increasing monthly premiums and cost-sharing requirements are often a substantial burden, especially for beneficiaries on fixed incomes. Nearly one in four Medicare enrollees age 65 and older report difficulty affording premiums, and almost 40% of those with incomes below twice the federal poverty level struggle to cover these costs. Additional financial burdens arise from extended care beyond standard coverage limits.

For example, in 2024, Medicare requires beneficiaries to pay $408 per day for inpatient, rehabilitation, inpatient psychiatric, and long-term acute care between days 61–90 of a hospital stay, totaling $11,832. Beyond 90 days, beneficiaries incur $816 per day for up to 60 lifetime reserve days, amounting to $48,720. Once these lifetime reserve days are exhausted, patients bear all inpatient costs, and these reserve days are never replenished again once they are used. Although the average hospital length of stay is typically shorter, inpatient days under Medicare do not need to be consecutive. This means if a patient is discharged and readmitted within a 60-day period, these patient payment responsibilities still apply and will not reset until there has been at least a 60-day break in care. 

Medicare coverage for skilled nursing facilities is similarly limited: While Medicare fully covers the first 20 days when transferred from a qualified inpatient stay (at least three consecutive inpatient days, excluding the day of discharge), days 21–100 require a copayment of $204 per day, totaling $16,116. After 100 days, all SNF costs fall to the beneficiary. These costs are significant, and without out-of-pocket maximums, they can create financial hardship.

Some of these costs can be subsidized with Medicare Supplemental Insurance, or Medigap plans, but they come with additional premiums. By regularly educating patients and families of these costs—and offering tax credits, waived or reduced copayments and deductibles, prescription rebates, or account credits—CMS could provide substantial financial relief while encouraging the completion of AHCDs.

Encourage Expansion of the NCQA’s ACP HEDIS Measure

Finally, the MAHDE Initiative can be coupled with the expansion of the HEDIS measure to establish a comprehensive strategy for advancing proactive healthcare planning among Medicare beneficiaries. By encouraging both the accessibility and completion of AHCDs, while also integrating ACP as a quality measure for all Medicare Advantage enrollees aged 65 and older, CMS would embed ACP into standard patient care. This approach would incentivize health plans to prioritize ACP and help align patients’ care goals with the services they receive, fostering a more patient-centered, value-driven model of care within Medicare.

Figure 2. Four key features of the Medicare Advance Healthcare Directive Enrollment (MAHDE) Initiative. This initiative should also work in tandem with efforts to encourage the NCQA to expand ACP HEDIS measures to include all Medicare Advantage beneficiaries aged 65 and older. (Source: Dr. Tiffany Chioma Anaebere)

Figure 2. Four key features of the Medicare Advance Healthcare Directive Enrollment (MAHDE) Initiative. This initiative should also work in tandem with efforts to encourage the NCQA to expand ACP HEDIS measures to include all Medicare Advantage beneficiaries aged 65 and older. (Source: Dr. Tiffany Chioma Anaebere)

Conclusion

When patients and their families are clear on their goals of care, it is much less challenging for medical staff to navigate crises and stressful clinical situations. In unfortunate cases when these decisions have not been discussed and documented before a patient becomes incapacitated, doctors witness families struggle deeply with these choices, often leading to intense disagreements, conflict, and guilt. This uncertainty can also result in care that may not align with the patient’s goals. 

Physicians and other qualified health professionals should continue to be trained on best practices to facilitate ACP with patients, and more importantly, the system should be redesigned to support these conversations early and often for all older Americans. The MAHDE Initiative is feasible, empowers patients to engage in ACP, and will reduce medical costs nationwide by allowing patients to be educated about their options and choose the care they want in future emergencies and at the end of life. 

Starting an AHCD enrollment initiative with the Medicare population older than age 65 and achieving success in this group can pave the way for expanding ACP efforts to other high-need groups and, eventually, the general population. This approach fosters a healthcare environment where, as a nation, we become more comfortable discussing and managing healthcare decisions during emergencies and at the end of life.

This action-ready policy memo is part of Day One 2025 — our effort to bring forward bold policy ideas, grounded in science and evidence, that can tackle the country’s biggest challenges and bring us closer to the prosperous, equitable and safe future that we all hope for whoever takes office in 2025 and beyond.

Frequently Asked Questions
Will completing an Advance Healthcare Directive (AHCD) be required to receive Medicare benefits?

No. While the MAHDE Initiative will encourage all adults over age 65 who are enrolled in Medicare or Medicare Advantage plans to complete or renew an electronic AHCD annually through Medicare.gov or an alternative CMS-approved secure ACP digital platform, it will not be a requirement for receiving Medicare benefits or care.

How will Medicare beneficiaries complete an AHCD?

Working alongside state-specific submission guidelines, Medicare beneficiaries can securely complete their AHCD on their own or during a visit with a qualified medical provider or health professional, either in person or through telehealth.



  • Online submission: An accessible electronic version will be available on Medicare.gov or an alternative CMS-approved secure ACP digital platform, allowing individuals to complete and submit their AHCD online, with guidance from their care provider as needed.

  • Paper version: Alternatively, individuals can also choose to complete a paper version of the AHCD, which can then be submitted to Medicare or a CMS-approved alternative for well-digitized electronic upload, storage, and access by healthcare professionals on Medicare.gov.


Review, updates, or to or confirm “no change” to these directives can be made annually online or by resubmitting updated paper forms during open enrollment or anytime as desired. Flexible options aim to make the process of AHCD completion accessible and convenient for all Medicare beneficiaries.

Does the author endorse any of the products or organizations mentioned in this policy memorandum?

The author does not endorse specific products, individuals, or organizations. Any references are intended as examples or options for further exploration, not as endorsements or formal recommendations.

Driving Equitable Healthcare Innovations through an AI for Medicaid (AIM) Initiative

Artificial intelligence (AI) has transformative potential in the public health space – in an era when millions of Americans have limited access to high-quality healthcare services, AI-based tools and applications can enable remote diagnostics, drive efficiencies in implementation of public health interventions, and support clinical decision-making in low-resource settings. However, innovation driven primarily by the private sector today may be exacerbating existing disparities by training models on homogenous datasets and building tools that primarily benefit high socioeconomic status (SES) populations. 

To address this gap, the Center for Medicare and Medicaid Innovation (CMMI) should create an AI for Medicaid (AIM) Initiative to distribute competitive grants to state Medicaid programs (in partnership with the private sector) for pilot AI solutions that lower costs and improve care delivery for rural and low-income populations covered by Medicaid. 

Challenge & Opportunity

In 2022, the United States spent $4.5 trillion on healthcare, accounting for 17.3% of total GDP. Despite spending far more on healthcare per capita compared to other high-income countries, the United States has significantly worse outcomes, including lower life expectancy, higher death rates due to avoidable causes, and lesser access to healthcare services. Further, the 80 million low-income Americans reliant on state-administered Medicaid programs often have below-average health outcomes and the least access to healthcare services. 

AI has the potential to transform the healthcare system – but innovation solely driven by the private sector results in the exacerbation of the previously described inequities. Algorithms in general are often trained on datasets that do not represent the underlying population – in many cases, these training biases result in tools and models that perform poorly for racial minorities, people living with comorbidities, and people of low SES. For example, until January 2023, the model used to prioritize patients for kidney transplants systematically ranked Black patients lower than White patients – the race component was identified and removed due to advocacy efforts within the medical community. AI models, while significantly more powerful than traditional predictive algorithms, are also more difficult to understand and engineer, resulting in the likelihood of further perpetuating such biases. 

Additionally, startups innovating the digital health space today are not incentivized to develop solutions for marginalized populations. For example, in FY 2022, the top 10 startups focused on Medicaid received only $1.5B in private funding, while their Medicare Advantage (MA)-focused counterparts received over $20B. Medicaid’s lower margins are not attractive to investors, so digital health development targets populations that are already well-insured and have higher degrees of access to care.

The Federal Government is uniquely positioned to bridge the incentive gap between developers of AI-based tools in the private sector and American communities who would benefit most from said tools. Accordingly, the Center for Medicare and Medicaid Innovation (CMMI) should launch the AI for Medicaid (AIM) Initiative to incentivize and pilot novel AI healthcare tools and solutions targeting Medicaid recipients. Precedents in other countries demonstrate early success in state incentives unlocking health AI innovations – in 2023, the United Kingdom’s National Health Service (NHS) partnered with Deep Medical to pilot AI software that streamlines services by predicting and mitigating missed appointment risk. The successful pilot is now being adopted more broadly and is projected to save the NHS over $30M annually in the coming years. 

The AIM Initiative, guided by the structure of the former Medicaid Innovation Accelerator Program (IAP), President Biden’s executive order on integrating equity into AI development, and HHS’ Equity Plan (2022), will encourage the private sector to partner with State Medicaid programs on solutions that benefit rural and low-income Americans covered by Medicaid and drive efficiencies in the overall healthcare system. 

Plan of Action

CMMI will launch and operate the AIM Initiative within the Department of Health and Human Services (HHS). $20M of HHS’ annual budget request will be allocated towards the program. State Medicaid programs, in partnership with the private sector, will be invited to submit proposals for competitive grants. In addition to funding, CMMI will leverage the former structure of the Medicaid IAP program to provide state Medicaid agencies with technical assistance throughout their participation in the AIM Initiative. The programs ultimately selected for pilot funding will be monitored and evaluated for broader implementation in the future. 

Sample Detailed Timeline

Risks and Limitations

Conclusion

The AI for Medicaid Initiative is an important step in ensuring the promise of artificial intelligence in healthcare extends to all Americans. The initiative will enable the piloting of a range of solutions at a relatively low cost, engage with stakeholders across the public and private sectors, and position the United States as a leader in healthcare AI technologies. Leveraging state incentives to address a critical market failure in the digital health space can additionally unlock significant efficiencies within the Medicaid program and the broader healthcare system. The rural and low-income Americans reliant on Medicaid have too often been an afterthought in access to healthcare services and technologies – the AIM Initiative provides an opportunity to address this health equity gap.

This action-ready policy memo is part of Day One 2025 — our effort to bring forward bold policy ideas, grounded in science and evidence, that can tackle the country’s biggest challenges and bring us closer to the prosperous, equitable and safe future that we all hope for whoever takes office in 2025 and beyond.

Establish Data Standards To Protect Newborn DNA Privacy by Developing Data Storage Standards for Newborn Screening Samples

Newborn screening is performed on millions of babies in the U.S. every year to test for rare genetic diseases and, when necessary, allow for early treatment. While newborn screening is mandated by the federal government, each state runs its own screening program. Importantly, individual states manage how newborn screening data is stored and, potentially, accessed and used in the future. While such data is often used for quality assurance testing and clinical research, there have been  instances of law enforcement subpoenaing newborn screening data for use in criminal investigations. For example, New Jersey used newborn screening data to investigate a decades-old sexual assault This raises major concerns about overall transparency of data use and  privacy in the newborn screening process. 

The incoming administration should encourage states to develop data handling standards for newborn screening data. Specifically these standards should include how long data is stored and who can access it. This can be accomplished by directing the Health and Human Services’ (HHS) Federal Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) to provide recommendations that clearly communicate data use and privacy measures to state health departments. In addition, the incoming administration should also encourage development of increased educational materials for parents to explain these privacy concerns, and create funding opportunities to incentivize both of these measures.

Challenge and Opportunity

Newborn screening is a universal practice across the United States. Blood samples are taken from infants only a few days old to test for a variety of genetic diseases such as phenylketonuria, which can cause intellectual disability that can be prevented through changes in diet—if it is caught early enough. These blood samples can be used for both metabolic and genetic tests, depending on which disease is being tested for and how it is detected.  Phenylketonuria, for example, is detected by high levels of a molecule called phenylalanine in the blood, while spinal muscular atrophy (SMA) is detected by changes in the genetic sequence of the gene associated with SMA. Newborn screening is an essential practice that identifies a wide range of severe diseases before symptoms occur, and three babies out of every 1,000 are identified with a genetic condition. 

While newborn screening is required by the federal government, each state can determine which panel of diseases are tested. The Department of Health and Human Services established an Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), which regularly updates a Recommended Uniform Screening Panel (RUSP) with conditions. For example, SMA was approved to the RUSP in 2018, and all 50 states have now added SMA to their screening panels.  Much of the effort to both nominate conditions to the federal RUSP and to encourage individual states to adapt spinal muscular atrophy testing was led by patient advocacy groups such as CureSMA, and these sorts of groups play a significant role in the addition of future conditions. Similar efforts are underway for Krabbe disease, which was added to the RUSP in 2024  and is currently screened for in only twelve states, a number that may increase in the coming years as more states consider adding it to their panels.   State advisory boards will review new disease nominations and, along with their status on the RUSP, will often consider how prevalent a disease is, if there are treatments available for the disease, and cost-effectiveness of screening for this condition.  Regardless of which tests are performed, every state participates in newborn screening. Importantly, newborn screening does not require affirmative consent from parents—some states offer opt-out options, generally for religious reasons, but 98% of infants are screened. 

Mandatory newborn screening programs have led health departments across the country to obtain genetic data from nearly every child in the country for decades. With recent developments in genetic sequencing technologies, this means that, theoretically, this newborn screening data could be repurposed for other functions. In 2022, the New Jersey Office of the Public Defender filed a lawsuit against the New Jersey Department of Health for complying with a subpoena to provide newborn screening data to the police as part of a sexual assault investigation. Specifically, law enforcement subpoenaed the blood sample of a suspect’s child, which they used to perform new DNA analysis to match DNA crime scene evidence. The lawsuit reveals that the New Jersey Department of Health has retained newborn screening blood spots for over twenty years; that the data obtained from the subpoena was used to bring criminal charges for a crime committed in 1996; and that the Office of the Public Defender were not provided information about how many similar subpoenas have been complied with in the past. 

This case highlights the bigger issue of newborn screening data as the United States’ “hidden national DNA database.” Law enforcement has potential access to decades of samples that can be used for genetic analysis that were not intended for law enforcement use. Police in other states like California have also sought access to newborn screening databases for investigational purposes. In California, the state health department keeps samples indefinitely, and not only is this information not disclosed to parents, it no longer provides parents with an opt-out option. As law enforcement agencies across states begin to understand the magnitude of data that can be found in these databases, it is becoming clear that health department policies for regulating access to these data are lacking. 

Using genetic data in law enforcement has become increasingly common. The practice of “investigative genetic genealogy,” or IGG, has made national headlines in recent years, in which law enforcement can access genetic data from publicly available databases to use in criminal investigations. These databases are full of genetic data that consumers who participate in direct-to-consumer genetic testing, such as 23andMe, can use to voluntarily upload and share their data with more people. IGG presents its own privacy concerns, but it is important to recognize the voluntary nature of both (a) participating in direct-to-consumer testing and (b) uploading it to a third-party website. Newborn screening, on the other hand, is not an optional practice.  

Proponents of IGG argue that using genetic data is very effective at not only catching killers—and doing so quicker than without DNA data—but also exonerating innocents.  However, this fact does not outweigh the major issues of privacy, transparency, and the fact that this approach potentially violates the fourth amendment’s protections against unreasonable search and seizures—especially when it comes to incorporating newborn screening data into these approaches. A previous court case found a hospital in violation of the fourth amendment for providing law enforcement with warrantless drug screening results from pregnant women, even though the women were under the impression they were receiving diagnostic tests. The Supreme Court argued that the hospital’s actions break down public trust in the health system, as patients have a “reasonable expectation of privacy” regarding their test results. While cases of subpoenaing newborn screening data may not currently violate any legal procedure, allowing law enforcement access to these data for use in future investigations, particularly without informing the individuals or parents involved, may also erode trust in the health system. This may lead to parents—when given the option—to opting out of newborn screening programs more often, leading to an increase in genetic and metabolic disorders going undiagnosed in newborns and causing major health problems in the future. In addition, with many scientists advocating for adopting whole-genome sequencing of newborns—instead of simply sequencing a panel of genes that are commonly identified as disease-causing in newborns—the amount of potential available genetic data could be staggering.  As a result, the incoming administration needs to take action to address the lack of transparent policies regarding newborn screening data in order to maintain its success as a public health measure.

Plan of Action

Current genetic privacy legislation

The landscape of genetic privacy legislation is, currently, somewhat patchwork. At the federal level, the most relevant legislation includes (1) the Genetic Information Nondiscrimination Act (GINA), (2) the Affordable Care Act (ACA), and (3) the Health Insurance Portability and Accountability Act (HIPAA). GINA specifically prohibits genetic discrimination in health insurance and in the workplace. This means that health insurers cannot deny coverage based on genetic data, and employers cannot make hiring, firing, or promotion decisions based on genetic data. The ACA strengthens GINA’s stipulation against genetic discrimination in health insurance by mandating that any health insurance issuer must provide coverage to whomever applies, as well as including genetic information on the list of factors that cannot be considered when determining overage or premium costs. HIPAA additionally regulates genetic data gathered in a healthcare setting, which includes newborn screening data, but HIPAA-protected information can be shared at the request of a court order or subpoena. The FBI developed an interim policy regarding all types of forensic genetic genealogy—often used with direct-to-consumer genetic tests but could also be applicable to newborn screening—which states the criteria required for investigators to use this approach. Criteria includes the requirement that a case must be an unsolved violent crime. In addition, the interim policy states that investigative agencies must identify themselves as law enforcement—a previous case was solved by accessing genetic databases without disclosing this information to the database—and that any collected data must be destroyed upon conclusion of the case.

Additionally, many states have additional laws that strengthen genetic privacy regulation on top of federal regulations. Maryland  passed a bill that regulates the use of genetic data in criminal investigations—specifically, it requires that law enforcement obtains informed consent from non-suspects before using their DNA in investigations. Other recent state regulations that address law enforcement access to genetic data in one way or another include Montana, which requires government agencies to obtain a warrant to access genetic data, and Tennessee, which explicitly allows law enforcement to access genetic data as long as they obtain a warrant or subpoena. Importantly, many of these laws are geared more towards addressing genetic data from direct-to-consumer testing and do not directly apply to newborn screening. Like federal legislation, state genetic privacy legislation is largely lacking in policies to address the use of newborn screening by law enforcement. 

On top of legislation regarding genetic privacy, states all have their own respective policies regarding newborn screening that vary dramatically. For example, a court in Minnesota found that nonconsensual storage of newborn screening data for use outside of genetic screening purposes violates the state genetic privacy law stating that genetic information can only be distributed with an individual’s written consent, leading to Minnesota destroying its newborn screening samples. Other states have no legislation at all. Additionally, states can have laws addressing other, non-law enforcement uses of newborn screening data; another major use of newborn screening data is research. 

Policy Recommendations

The incoming administration should address the lack of transparency in newborn screening data management by implementing the following recommendations:

Direct the ACHDNC to develop national recommendations detailing standards for newborn genetic screening sample and data handling.

These standards should include:

Standards for what the data can be used for outside of newborn screening, and by whom. Newborn screening data is used in additional ways outside of law enforcement; it can also be used for quality assurance to help ensure tests are working properly, to help develop new tests, and in clinical trials. There are compelling arguments for these uses; for clinical research, for example, this data can contribute towards research studying the disease the child may have been diagnosed with. However, for the sake of transparency, policy should state specifically what newborn genetic data can and cannot be used for, and who is allowed access to the data under these circumstances. For instance, Michigan has a program called the Michigan BioTrust, which takes the leftover, de-identified newborn screening samples for use in research towards understanding disease. Parents can choose to opt in or out at the time of screening, and parents—as well as children, upon turning 18—can change their mind and have their data removed later if they so choose. Regardless of state decisions on whether law enforcement should be able to access their newborn screening data, clearly stating what the data can be used for overall is paramount for parents to understand what happens to their children’s samples.

The length of time that blood samples and genetic data can be stored in state databases, and when, if ever, the data will be destroyed. As detailed by the lawsuit, New Jersey had been storing samples for over twenty years, although parents were not actually aware of this fact until the lawsuit was filed; potentially in response to this lawsuit, starting in November 2024, New Jersey will be destroying blood spots older than 2 years. Similarly, Delaware stores blood spot samples for three years before destroying them. While there is no definitive answer to what the best timeline for saving samples is, establishing a transparent timeline for how long samples can be stored in each state will improve data handling transparency.

What say, if any, do parents have in what is done with their child’s samples and data. In Texas, after a lawsuit determining that storing newborn screening samples without consent was against the law, parents have the right to request their child’s samples be destroyed if they so choose. Developing policies that allow parents—or children themselves, once they become adults—to have a say in what happens to the samples after screening is completed would provide individuals control of their data without disincentivizing testing. 

Partner with state advisory boards to develop educational materials for parents detailing ACHDNC recommendations and state-specific policy.

While newborn screening is mandated, there is variable information available to parents regarding what is done with the data. For example, Michigan has an extensive Q&A page on their Department of Health website addressing many major newborn screening-related questions, including a section addressing what is done with samples after screening is complete. In contrast, West Virginia’s Q&A page does not address what happens to the samples after testing.  Not only would developing standard policies for data handling be beneficial, but improving the dissemination of such information to parents would increase overall transparency and improve trust in the system. The incoming administration should work closely with state advisory boards to improve the communication of newly-developed data handling standards to parents and other relevant parties.

Incentivize development of plans by providing grant opportunities to state health departments to support newborn screening programs.

Currently, newborn screening programs receive no direct federal funding; however, costs include operating costs, testing equipment, and personnel on top of the tests themselves. In general, newborn screening is paid for through a fee for the tests, which are often covered by the parents’ health insurance, or the State Children’s Health Insurance Program or Medicaid. However, grants such as the NBS Co-Propel have been awarded to states in the past for creating improvements in their newborn screening programs such as support for long-term follow up on patients that have positive test results returned to them. The Co-Propel grant was administered through the Maternal & Child Health Bureau (MCHB) of Health and Human Services; the incoming administration could recommend that MCHB initiates a new funding opportunity for states to either develop data storage standards and/or educational materials for families to encourage the adaptation of these standards.

Conclusion

Newborn genetic screening is an essential public health measure that saves thousands of lives each year by identifying diseases in newborns that can either be prevented early or treated immediately rather than waiting until severe symptom onset. However, with the advent of new genetic technologies and the burgeoning use of newborn genetic screening data in law enforcement investigations, major privacy and transparency issues are becoming known to parents, potentially putting trust in the newborn screening process at risk. This could reduce desire to participate in these programs, leading to an inability to quickly diagnose many preventable or treatable conditions. The incoming administration should work towards encouraging state health departments to develop clear and well-communicated data storage standards for newborn screening samples in order to combat these concerns moving forward.

This action-ready policy memo is part of Day One 2025 — our effort to bring forward bold policy ideas, grounded in science and evidence, that can tackle the country’s biggest challenges and bring us closer to the prosperous, equitable and safe future that we all hope for whoever takes office in 2025 and beyond.

Frequently Asked Questions
How does newborn screening actually work?

Newborn screening is performed by pricking a newborn’s heel to obtain a blood sample, or “blood spot,” within two days of being born. These blood spot samples are used for both metabolic tests and genetic tests. Metabolic tests measure different molecules in the blood that might signal a disease, such as high levels of an amino acid called phenylalanine, which in healthy amounts is used by our bodies to make proteins and in high amounts can cause phenylketonuria. Genetic tests are performed by sequencing a panel, or selection, of genes that are often associated with newborn screening diagnoses; often, genetic testing is performed after a positive hit on a metabolic test to both confirm and further clarify the diagnosis.

What does the Advisory Committee on Heritable Disorders in Newborns and Children do?

The role of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) is to communicate with the Secretary of the Department of Health and Human Services regarding newborn screening policies. This not only includes managing the Recommended Uniform Screening Panel, but also providing advice on grants and research projects related to newborn screening research, assistance with developing policies for state and local health departments for newborn screening implementation, and recommendations towards reducing child mortality from the diseases screened.

What is included in the Recommended Uniform Screening Panel?

The Recommended Uniform Screening Panel (RUSP) is the list of disorders recommended for newborn testing. As of July 2024, the RUSP contains 38 “core conditions,” which are conditions that states specifically test for, and 26 “secondary conditions,” which are conditions that physicians may identify incidentally while screening for core conditions. New conditions can be added, and conditions can be moved between categories if the advisory board chooses to do so. These conditions include metabolic disorders such as phenylketonuria, endocrine disorders such as thyroid disorders, hemoglobin disorders such as sickle cell anemia, and others such as cystic fibrosis.

Where can I learn more about genetic privacy laws by state?

The National Human Genome Research Institute has a searchable database that details the different state genetic privacy laws, including their legislative status and a summary of their intended purpose. These laws have many goals, including expanding protections against genetic discrimination, research subject protections, artificial intelligence, and more.

Advisory Committees for the 21st Century Recommendations Toolkit

From January 2024 to July 2024, the Federation of American Scientists interviewed 30 current and former Advisory Committee (AdComm) members. Based on these discussions, we were able to source potential policy recommendations for the executive level, for the executive level, that may assist with enhancing the FDA’s ability to obtain valuable advice for evidence-based decision-making. In this toolkit, we build off of those discussions by providing you with actionable policy reform recommendations. We hope that these recommendations catapult the Advisory Committee structure into one best suited to equip all AdComms with the necessary tools needed to continue providing the government with the best advice.

Download a PDF version of these recommendations on the left.

Recommendations and Best Practices for Actionable Policy Implementation

Voting for FDA Advisory Committees

Update Type: Process

Best Practices for Implementation 

The United States Food and Drug Administration can uphold their voting mechanism by updating their document entitled, “Guidance for FDA Advisory Committee members and FDA staff: Voting Procedures for Advisory Committee Meetings” to include language that clearly states a vote should be taken at all Advisory Committee meetings where a medical product is being reviewed. This guidance should also indicate that the absence of voting should only occur if an Advisory Committee meeting has been convened to discuss issues of policy. Further, this guidance should be considered a level 2 guidance as it falls into the category of addressing a “controversial issue”. To effectuate these changes, a notice of availability (NOA) may be submitted to the Federal Register for public input (public input is not a requirement before implementation). 

Potential Language to be Utilized for Guidance 

In an effort to continue to allow Advisory Committee members to provide unbiased, evidence-based feedback and uphold such an integral part of the Advisory Committee process, voting is hereby mandatory for all Advisory Committee meetings that are convened where the purpose is to review and assess the safety and efficacy of medical products. 

Involved Stakeholders

In order for this process to successfully occur, the FDA will need to amend their guidance with the aforementioned updates. Consideration should be given to incurred costs for personnel required to complete these updates. Personnel needed for these amendments may include, but are not limited to, the (a) Office of the Commissioner, Office of Clinical Policy and Programs, Office of Clinical Policy, (b) Center for Devices and Radiological Health, (c) Center for Biologics Evaluation and Research, and (d) Center for Drug Evaluation and Research.

FDA Staff, Leadership, and AdComm Disagreements

Update Type: Process

Best Practices for Implementation 

Implementing these recommendations will improve conflict resolution internally and between the Agency and Advisory Committee members. Best practices for implementation include (a) building the Scientific Dispute Resolution at FDA guidance into the official FDA onboarding process for new hires to raise awareness, (b) provide annual employee trainings in an effort to stay up-to-date with dispute resolution processes and procedures, and (c) develop a guidance that delineates the process for resolving conflicts between the Agency and Advisory Committees when there are differing opinions. 

Note: Guidance for resolving disputes between the Agency and Advisory Committees should be submitted to the Federal Register for public comment. Guidance should also include plain language that designates the avenue to be used for official decision notifications, the timeliness of these notifications after convenings have concluded, and circumstances in which the FDA cannot notify Advisory Committees that their decision is in direct opposition of the Committee’s vote (e.g., – if this notification would breach a confidentiality agreement with the applicant). Implementing a transparent process to communicate with AdComm members regarding differences between the Agency and the AdComm will assist in improving morale between both parties, but also encourage continued support of the AdComm.

Involved Stakeholders 

Successful implementation of these recommendations will require the capacity of human resources personnel and individual center leadership. 

Update Type: Regulatory

Best Practices for Implementation

Center leadership can assign FDA staff to make the necessary guidance amendments which should include the requirements for inclusion in the onboarding of all FDA employees. Staff should also be responsible for obtaining feedback on amendments from all necessary internal parties and submitting proposed amendments to OMB for review and addition to the Federal Register. Federal register comments will then be reviewed by FDA staff, guidance will be updated accordingly, and a final draft submitted to OMB for review. If approved, the final regulation will be published in the Code of Federal Regulations. Congressional involvement should not be necessary.

FDA Center leadership should delegate the task of creating an annual mandatory training program for all FDA employees to review this guidance in an effort to stay abreast of the procedures for dispute resolution. 

Involved Stakeholders 

FDA Center leadership, FDA staff, and the Office of Management and Budget (OMB) are the intended stakeholders for implementation of these recommendations. To incorporate this guidance into FDA regulations, Center leadership will need to assign FDA staff to amend guidance and submit to OMB.

Leveraging AdComm Membership

Update Type: Process

Expanding Committee Representation

Amplifying the Role of the Chair

Establishing Training and Regulatory Procedures for Incoming Members

Best Practices for Implementation

With respect to Committee composition, the FDA should consider adding patient representatives to all Committees that review medical products. The addition of a patient representative will ensure that the voice of the population who the medical product affects is heard. The FDA can select individuals best suited to fill these roles through connecting with patient advocacy organizations. If patient representatives are selected, the FDA should develop an onboarding program to familiarize the patient representative with basic knowledge of the federal regulation process. This program should educate the patient representative on (a) the types of questions presented to Advisory Committees, (b) how the FDA views the role of the patient representative in the process, (c) the internal review process for data that is submitted, and (d) other pertinent topics related to medical product regulation.

Leveraging the role of the Advisory Committee Chair can help the FDA fully optimize the use of their Committee. “Chairs possess extensive networks that could support the identification of permanent or temporary expert participants for AdComms” (Banks, 2024). Allowing Chairs the ability to identify relevant issues or products for their respective committees to review can provide an additional layer for the FDA to keep abreast of critical public concerns via appropriate committee evaluation (Banks, 2024).

Finally, while Committee members may be experts in their own right, training should be provided for all. The FDA should provide basic 101 training courses that can cater to the needs of members with various knowledge bases. Training should include information on the relationship between the FDA and Advisory Committee members, best practices for understanding statistical analysis, and the different types of clinical trial designs. Training should provide real-world examples of statistical analysis and trial design in use (this can be done by providing examples from prior medical product review). 

Involved Stakeholders

FDA Center staff (including statisticians and scientists for the development of training programs).

Conflict of Interest (COI) Auditing

UPDATE TYPE: PROCESS 

Best Practices for Implementation

To prevent recurring COIs, the FDA should develop a database of experts for various categories of expertise that can be selected to replace those with known COIs. This database should include names, contact information, credentials, all areas of expertise for each expert, and should link to public financial interest databases that can serve as a source for identifying conflicts (e.g., Open Payments, Dollars for Docs).

To prevent public confusion, the FDA should amend their Guidance for the Public, FDA Advisory Committee Members, and FDA Staff: Public Availability of Advisory Committee Members’ Financial Interest Information and Waivers to specify circumstances that warrant a COI waiver being administered. This will increase transparency and help Advisory Committee members and the public understand the reasoning in allowing members with conflicts to participate in meetings. This guidance can then be submitted to the Federal Register for public comment

UPDATE TYPE: PROCESS | REGULATORY

Best Practices for Implementation

Streamlining the COI process will assist the FDA with retention efforts for Advisory Committees while maintaining compliance with conflict of interest regulations. A digital system should be developed that allows Advisory Committee members to select whether their financial information has changed through the use of a dropdown or check box. This will prevent duplicative work and also contribute to a sustainable (green) process.

Involved Stakeholders

The Office of the Commissioner, Office of Clinical Policy and Programs, Office of Clinical Policy would be the interested stakeholder to issue updates to the COI policy and would work with the General Services Administration if necessary. 

The Role of Patient Advocacy in the AdComm Process

Update Type: Process

Best Practices for Implementation

Public comments are a crucial part of the regulatory process. The FDA should focus on increasing participation in this process, as well as notifying participants of what should be expected from the public comment period. By law, the FDA must allow a public comment period for all Advisory Committee convenings. To increase participation, the FDA should allow public comments to be vocalized in-person and virtually, in addition to the submission of public comments to the docket. 

Regarding Committee composition, please refer to Best Practices for Implementation under Leveraging AdComm Membership for information on the addition of patient representatives to Advisory Committees (see prior section above).

Patient engagement in the regulatory process is necessary to inform evidence-based decisions. While the FDA currently has ways to engage these communities through the use of public comment and initiatives such as Patient Focused Drug Development (PFDD), engagement should be an ongoing process. As mentioned, the FDA can develop and leverage existing relationships with public health agencies and advocacy organizations who can then serve as the liaison of feedback to the FDA. The FDA can also consider expanding their current initiatives and programs to engage communities twice a quarter instead of quarterly or annually. Consistent engagement in this form will help to establish trust between the FDA and the public who they serve, as well as give them the needed information from the communities who are most impacted from their decisions.  

Involved Stakeholders

Implementation of these recommendations and expansion of current initiatives will require the involvement of FDA Center leadership and Center staff.

Improving Public Awareness and Understanding of Advisory Committees

Update Type: Process

Best Practices for Implementation

The FDA should develop a monthly content plan to utilize its current interactive and social media outlets and disseminate information related to the role of Advisory Committees and their convenings, while also maintaining compliance with the FDA’s social media policy. The social media content plan should be centered around (a) what an Advisory Committee is, (b) how members are selected, (c) information regarding votes of Advisory Committees and how they are specific to safety and efficacy, but are not voting on the approval of a medical product, (d) discussing upcoming Advisory Committee meetings, their location, and inviting the public to participate via public comment, (e) sharing information about what a public comment is, requirements for making public comments, and how the FDA reviews them, and (f) sharing a webpage where the general public can input their personal email to be notified of upcoming Advisory Committee meetings.

FDA staff should develop a plain language disclaimer to be placed on all social media posts, meeting materials, and websites related to Advisory Committees. This disclaimer should illustrate that Advisory Committee members will provide unbiased expertise to assist the FDA with their decision. However, while the Committee’s vote is included in consideration for the decision, their vote is non-binding which leaves the FDA as the final decision maker for approval.

Finally, the FDA should identify state/local public health agencies, as well as advocacy organizations that they can potentially partner with to disseminate information more broadly. These agencies and advocacy organizations have strong relationships with various communities who should be engaged in the regulatory process. Developing a relationship with these agencies and organizations in an attempt to engage the community will assist the FDA with building connections and trust, as well as mutual understanding of Advisory Committee roles. Potential partners can be identified using the linked list under involved stakeholders. 

Involved Stakeholders

Implementation of these recommendations and expansion of current initiatives will require the involvement of FDA Center leadership, Center staff, Office of External Affairs (OEA) Web and Digital Media staff, Office of Information Management and Technology (OIMT), state/local public health departments, and advocacy organizations.

Note: These listings of state/local public health departments and advocacy organizations are intended to be used as a starting point in the identification of potential partners and not to be considered an exhaustive list.

For questions related to this toolkit, please contact

Cheri Banks
Health Regulatory Specialist
cbanks@fas.org

Grace Wickerson
Health Equity Policy Manager
gwickerson@fas.org

The Role of Patient Advocacy in the AdComm Process

From January 2024 to July 2024, the Federation of American Scientists interviewed 30 current and former Advisory Committee (AdComm) members. Based on these discussions, we were able to source potential policy recommendations that may assist with enhancing the FDA’s ability to obtain valuable advice for evidence-based decision-making. The results of these discussions are presented in case study format detailing the recurring themes that emerged and policy recommendations for improvement.

The regulation of medical products is the responsibility of the Food and Drug Administration (FDA). To ensure effective decision-making regarding these products, the FDA recognizes the importance of patient advocacy and the perspectives of patients. In 1988, the FDA initiated the patient engagement process through the Office of Aids Coordination, and within five years, the first patient representative was appointed to an FDA Advisory Committee. Since then, the FDA has significantly enhanced its methods of engaging patients, caregivers, and patient advocates. This includes the establishment of various offices, programs, collaboratives, listening sessions, public guidance, and more.

The Evolution of Patient Engagement at the FDA

The FDA employs several avenues to engage patients in the regulatory process. Some avenues include the Patient Engagement Advisory Committee (PEAC), public comment, the Patient Focused Drug Development Initiative (PFDD), the Patient Listening Session Program, Patient Engagement Collaborative (PEC), and the Patient Representative Program (PRP). This list does not cover all the ways in which the FDA engages patients and advocates but provides an overview of the key operations involved in patient engagement efforts. 

The Patient Engagement Advisory Committee is the only Committee that is completely composed of caregivers, patients, and patient representatives from various organizations, as a way to ensure that the lived experiences of these populations and their opinions are included in the deliberations and regulatory decision-making of medical products. Public comment is a requirement by law for federal agencies, allowing the public to provide feedback on proposed actions or new rules and regulations. Public comment is also sought during Advisory Committee meetings to gather information and perspectives from the public. PFDD meetings provide a platform for the FDA to obtain insights from patients on specific diseases and their treatments. To identify the issues most important to patients, the FDA has a series of guidance documents that are used specifically for PFDD meetings. 

The Patient Listening Session Program facilitates informal meetings between patients, their representatives, and FDA staff. These sessions cover a range of topics, including treatment preferences, quality of life, unmet medical needs, and the impact of diseases and their symptoms. The PEC offers a forum for patients, caregivers, and advocates to discuss patient engagement operations. Lastly, the PRP allows patients, caregivers, and advocates who serve as special government employees the opportunity to provide advice to the FDA’s Commissioner or a designated representative on matters related to medical devices and their regulation.

Although there are various avenues for patient engagement and advocacy participation in the medical product regulation process, there are also ways in which these avenues can be expanded or improved.   

Patient Advocacy Problems

For many years, patients and their caregivers have not seen significant or sustainable treatments that have been developed to treat many illnesses and diseases. Some treatments have proven to be ineffective yet still made it to market approval. On the other hand, there are treatments that met safety and efficacy standards but were not approved. There are also those treatments that are simply not affordable to the populations that need them most. In many of these scenarios, the patient representative voice was lost as they did not have the option to express their concerns or perspectives on certain treatments with decision makers. This further confirms that the role of patient advocacy and allowing space for the patient representative voice is crucial to the regulation process of medical products. 

At the moment, the patient voice is not always heard because there are some FDA Advisory Committees that do not have a patient representative.

While the role of patient advocacy is crucial, it is important to note there should be boundaries in which patient perspective is considered for decision-making. Although patients and their advocates seek treatments that better address their needs, this desire can sometimes obscure their judgment concerning long-term treatment effectiveness. Frequently, patients and their supporters present powerful arguments to Advisory Committees and the FDA for approval of particular medical products which can lead to expedited medical product approval in the absence of supportive evidence. 

The endorsement of eteplirsen, which was intended for the treatment of Duchenne muscular dystrophy (DMD), illustrates this point. Despite a 7 to 3 vote by the FDA’s Advisory Committee against approval due to insufficient evidence of its benefits, opposition from the FDA’s Center for Drug Evaluation and Research’s former leader resulted in the drug’s authorization. This sparked substantial internal and public criticism and led Dr. Ellis Unger from the FDA’s Office of Drug Evaluation to challenge the approval decision. Dr. Unger emphasized that “patient-focused drug development is about listening to patient perspectives about what matters to them; it is not about basing drug approvals on anecdotal testimony that is not corroborated by data.”

This approval was perceived by many as having been heavily influenced by patient advocacy and raised concerns about potential long-term implications for patient health. It also signaled a need to further examine both patient education and the appropriate limits of patient involvement in the regulatory process. This could have been mitigated had there been a list of criteria in place to be followed for public comment. 

Incorporating Patient Perspectives

The Food and Drug Administration (FDA) is committed to understanding the balance of benefits and risks acceptable to patients as they relate to medical products. The FDA defines the role of patient representatives that serve on Advisory Committees as “Special Government Employees” who provide direct input to agency staff and share valuable insight on their experiences with various diseases, conditions, and devices while gaining access to confidential information. These representatives are selected by the FDA to serve on Advisory Committees using a specific set of criteria including, but not limited to: 

This criteria ensures the FDA will understand the patient perspective as it relates to various medical products and ensures those selected to serve on Advisory Committees are knowledgeable about the areas in which they are aiming to provide guidance. Currently, there are some FDA Advisory Committees that do not have a patient representative. Further, the patient representatives serving on committees do not always have voting privileges. The absence of consistent voting privileges for some patient representatives on Advisory Committees and not having a standing patient representative on all committees hinders these individuals from providing an official stance on behalf of the community they represent. Additionally, public comment plays a significant role at Advisory Committee meetings by permitting individuals—including patients, caregivers, and advocacy organizations—to highlight concerns and propose solutions that may not have been previously considered by decision-makers. This process also helps the committee and agency gauge patient acceptance or opposition related to medical products, thereby enhancing their ability to make decisions that more accurately reflect public needs. 

When Sarepta was seeking approval of eteplirsen for the treatment of DMD, a patient advocacy organization brought hundreds of patients, caregivers, and other advocates to the Advisory Committee convening so they could make a public comment to the Committee and the agency. Shortly after, the drug received a swift approval. Although it presented much controversy within the agency and the public, it showed how influential patient advocacy can be. Personal lived experiences, compelling stories of debilitating illnesses, and experiences with current treatment have the ability to impact regulatory decision-making. 

The role of patient advocacy continues to be important in the Advisory Committee process and FDA regulatory decision-making process because it is crucial to assisting with decisions that affect the American public. Patient advocacy can be presented in the form of patient representatives that serve on Advisory Committees, those who make public comments during Advisory Committee convenings, and various outreach programs by advocacy organizations. The role of advocacy gives patients and caregivers support, promotes and protects their rights, and allows broader visibility for the issues that are most important to them. All of these avenues for patient perspective are important to understand how treatments perform, the current needs of the patient population, and how to tailor care for these populations by truly understanding their condition, diagnosis, and current management. Therefore, their voice is critical to truly understanding how various medical products will benefit their population, how they will access and afford these products, and how they will fill an unmet medical need.

Policy Recommendations

In an effort to better leverage Advisory Committee membership, the potential policy recommendations are as follows: 

Conclusion

The landscape of disease burden and associated symptoms is ever-evolving. To ensure the FDA is best prepared for this changing landscape, patient advocacy and amplifying the patient voice should be considered vital to the development and regulation of medical products. Involving those who are the most impacted by these products is essential. The FDA can further promote the patient perspective and advance patient-centered health through incorporating patient representatives on all Committees that are reviewing medical products, making patient engagement an ongoing process, hosting town halls for patients to allow a broader audience the opportunity to voice opinions, and having dedicated staff to sort through public comments from patients.

FDA Staff and Leadership Disagreements and the Role of the AdComm in the Regulatory Process

From January 2024 to July 2024, the Federation of American Scientists interviewed 30 current and former Advisory Committee (AdComm) members. Based on these discussions, we were able to source potential policy recommendations that may assist with enhancing the FDA’s ability to obtain valuable advice for evidence-based decision-making. The results of these discussions are presented in case study format detailing the recurring themes that emerged and policy recommendations for improvement.

The FDA relies on its scientific staff and Advisory Committees to provide conclusions from trial and study data, which aid in the process of regulating and approving medical products. However, there are instances when disagreements arise between the agency’s scientists, statisticians, Advisory Committees, and leadership on the accelerated or full approval of medical products. The resolution of these disagreements present a growing concern about FDA leadership overruling the expert opinions of scientific staff and proceeding with official approvals, thus undermining staff expertise, decreasing agency morale, and potentially diminishing public trust.

When Disagreements Between FDA and AdComms Arise 

The Federal Advisory Committee Act governs the FDA’s Advisory Committees and establishes a process in which the FDA can seek expert advice on various issues related to science, regulatory policy, and the evaluation of products under the FDA’s jurisdiction. When the FDA has differing opinions on safety and efficacy requirements of medical products, certain products may be referred to an Advisory Committee for further data review by an impartial entity. To aid in this matter, the FDA has developed guidance detailing the process for assembling Advisory Committees. Since the resources required for convening these committees are significant, the FDA ensures there is substantial uncertainty or disagreement regarding the data. Advisory Committees will discuss the evidence and provide feedback with the goal of producing the most optimal evidence-based resolution. This part of the regulatory process is crucial to the agency’s regulatory decision-making as it involves unbiased parties and leads to transparency, upholding public safety, and maintaining public trust. However, there are times that FDA leadership disagrees with the votes of their Committees and proceeds with controversial approvals. One example of this scenario was approval of the drug Aduhelm. Aduhelm, which was marketed as a treatment for Alzheimer’s, received an overwhelmingly negative vote from the Advisory Committee to move forward with its approval for market distribution. Ten of eleven Committee members stated the data did not support adequate efficacy for approval. Nonetheless, the FDA granted accelerated approval, sparking resignations from a third of their Committee and outrage amongst the public. This disregard for expert opinion was viewed as the FDA exhibiting an approval bias, a perspective the public currently maintains of the FDA. 

In 2023, various organizations and coalitions, such as Doctors for America, Jacob’s Institute of Women’s Health, National Center for Health Research, TMJ Association and more publicly expressed concerns regarding the FDA’s leadership and their approach to drug approvals through a letter addressed to the FDA’s Chief Scientist, Dr. Namandje N. Bumpus. They highlighted FDA leadership had ignored claims by scientific staff that safety and efficacy standards were not being met for the drug Elevidys. Manufactured by Sarepta Therapeutics, Elevidys was granted accelerated approval by FDA leadership. Dr. Bumpus responded to this letter defending the FDA’s approval of Elevidys by referring to the agency’s comprehensive review of the data and consideration of the potential risks of the approved treatment, nature of the disease and its impact on patients, and the limited amount of therapies available. However, the response did little to quell public scrutiny of the controversial approval. Critics were apt to point out that the FDA had a record of approving products in this manner. Years prior, the FDA had approved the drug Eteplirsen for the treatment of duchenne muscular dystrophy (DMD), despite the objections of their scientific staff which disclosed a lack of evidence for the efficacy of this treatment. Criticism was also directed at FDA leadership suggesting the approval of some medical products were potentially due to favoritism toward companies seeking approval.

Resolving Internal FDA Disagreements

While it is acknowledged that the FDA must consider technological and political implications alongside scientific evidence in decision-making, it is essential to address the concerns of these organizations and coalitions. As part of an ongoing project to reform the FDA’s Advisory Committee system and assist the FDA with getting the advice needed to make the best evidence-based decisions, FAS engaged in discussions with current and former Advisory Committee members to seek their input on resolving disagreements between FDA staff and leadership. These conversations highlighted the breach of trust between the FDA, its staff, and the public.

Quotes from Experts

“FDA leadership needs to make is clear what data was used and why they’re moving forward when there is opposition”

“Disagreements should be addressed by a non-biased source because it affects the public safety.”

“There will be times where there are disagreements between staff and leadership. However, there’s a critical need for transparency within the FDA about why decisions are made. These are not decisions about evidence only. Ever.”

“Disagreements should be a matter of public scrutiny. There should be transparency that doesn’t jeopardize confidentiality.”

The FDA’s Commissioner has acknowledged the lack of trust within the institution and expressed a commitment to address the issue. There are many reasons for the lack of trust. However, one reason stems from the FDA’s approval of medical products despite clear opposition from its scientists and sometimes Advisory Committee members.

This raises the question of how the FDA intends to address these internal disagreements, which have the potential to impact the health and safety of the American public. Currently, the FDA has implemented a program called Scientific Dispute Resolution (SDR) to handle such conflicts. This document was initially developed in 2009 and recently updated in 2021 with the purpose of outlining the process for communication regarding internal scientific disputes within FDA Centers. It defines scientific disputes as disagreements that arise from the interpretation of science and the resulting decisions. This definition clearly distinguishes the circumstances in which the guidance should be utilized to resolve discrepancies between FDA scientists, statisticians, and their respective Center leadership. The guidance offers valuable examples of best practices for resolving formal and informal scientific disputes within the agency. Some of those best practices include, but are not limited to: 

However, it should be noted these best practice recommendations are not obligatory and their adoption is left to the discretion of the individual Centers. Furthermore, the document provides a process by which any of the internal parties involved can appeal the resolution of their dispute if they find it unsatisfactory through the Office of Research  Integrity at Health & Human Services (HHS).

Policy Recommendations

To increase morale and improve the approach and resolution of internal disagreements within the agency, the policy recommendations are as follows:

  1. Ensure that all FDA staff and leadership are fully cognizant of the existence and details of the Scientific Dispute Resolution at FDA guidance and the process for submitting disputes for review.
  2. Incorporate the Scientific Dispute Resolution at FDA guidance into FDA regulations
    1. Note: To incorporate this guidance into FDA regulations, the FDA will propose the regulation for OMB review. OMB will review and open the regulation up for public comment through the Federal Register. Responses to comments will be developed, and a final draft submitted to OMB for review. If approved, the final regulation will be published in the Code of Federal Regulations (Congressional Research Service, 2013). The involvement of Congress will not be necessary.
  3. Amend the Scientific Dispute Resolution at FDA guidance to dictate the mandatory execution of best practices within the dispute resolution process.
    1. This guidance should identify additional non-biased parties (that may not be government-affiliated) to provide impartial guidance on complex scientific matters affecting public safety. 
  4. Develop guidance that clearly explains a transparent process to communicate effectively with AdComm members regarding decision making when parties have opposing viewpoints
    1. Implementing a transparent process to communicate with AdComm members regarding differences between the agency and the AdComm will assist in improving morale between both parties, but also encourage continued support of the AdComm.
    2. This recommendation is also supported by a survey conducted by 3D Communications with 400+ AdComm members where 94% of members concurred that the FDA should develop a process to communicate their reasoning for decisions in opposition to Committee recommendations (3D Communications, 2024).

Conclusion

While FDA leadership ultimately holds the authority to grant approvals, it is crucial that the perspectives of all experts are duly considered. This includes the valuable input from the agency’s scientists, statisticians, and advisory committees. To regain public trust and restore integrity, it is imperative to first rebuild trust internally among the dedicated public servants within the FDA. Adoption of the aforementioned recommendations would start the trust-rebuilding process and lead to increased safety and precaution measures when approving drugs and medical devices.

Leveraging AdComm Membership

From January 2024 to July 2024, the Federation of American Scientists interviewed 30 current and former Advisory Committee (AdComm) members. Based on these discussions, we were able to source potential policy recommendations that may assist with enhancing the FDA’s ability to obtain valuable advice for evidence-based decision-making. The results of these discussions are presented in case study format detailing the recurring themes that emerged and policy recommendations for improvement.

The FDA relies on its scientific staff and Advisory Committees to provide conclusions from trial and study data, which aid in the process of regulating and approving medical products. Discussions have been centered around how to appropriately leverage the membership of Advisory Committee experts to assist with areas of difficulty surrounding the safety and efficacy of medical products. Nonetheless, the methods by which these systems currently generate the evidence the Government needs can be improved. This case study focuses on five key areas we believe can assist in fully utilizing the capacity in which AdComms serve and improve overall engagement with AdComms membership.

AdComm Membership Problems

Advisory Committees serve as the core for expert engagement in the Food and Drug Administration’s (FDA) decision-making processes and are composed of medical professionals, industry representatives, patient advocates, and scientific experts. Their primary role is to provide the FDA with informed advice and recommendations on issues spanning science, regulatory policy, and the evaluation of products under the FDA’s jurisdiction.

The intricacies of being an effective AdComm member, however, have been somewhat overlooked. Conversations with current and ex-members have highlighted areas for enhancement that would strengthen the function of AdComms and enrich the advice provided. Feedback indicated a lack of transparency in the FDA’s recruitment methods for committee positions, insufficient orientation or training for new members, limited understanding of regulatory procedures among members, and an onerous conflict of interest protocol that served as a deterrent for some members who were asked to return or renew their membership. 

Pathways to Improving AdComms Membership

Committee Composition

The composition of Advisory Committees vary depending on the charter that has been set in place. In some cases, committee composition has been set by law. However, where there is flexibility in determining the composition of a committee, consideration should be given to all categories of expertise that should be included and diversity of voices that are selected to participate in these meetings. Committee composition should reflect the diversity of the world and populations of whom their recommendations could potentially affect. For this reason, discussions with current and former Advisory Committee members indicated the need for three additional areas of expertise that should be included on all Committees. Insights discovered that all Committees should include a patient representative who has the knowledge from lived experience and understanding of how treatments affect day-to-day life. This recommendation was further corroborated by a 3D Communications survey conducted with 400+ FDA AdComm members where results indicated that 48% agreed there should be a patient and consumer representative on all Committees. Members also stated that pharmacists should be included because drugs and devices eventually pass through their hands to give to patients. Pharmacologists should serve on the Committee due to their clinical application knowledge of drugs and devices. Finally, a roster of temporary members should be created for varying categories to use when additional expertise is needed on a Committee because of a conflict of interest or when a certain skillset or knowledge base is lacking on the current Committee. 

Role of the Advisory Committee Chair

The FDA describes the purpose of an Advisory Committee chair as one who will “preside at committee meetings and ensure that all rules of order and conduct are maintained during each session”. The chair also has the responsibility of ensuring all recommendations and advice from AdComm members are clear and evidence-based. Moreover, the role of the chair should be used to enhance the overall committee experience as well as be of service to the FDA. Despite these requirements, there’s an underutilization of the chair’s role in terms of communication and stakeholder coordination, as evidenced by the chair not being listed as a primary point of contact for the Advisory Committee and a lack of coordination amongst stakeholders.

Chairpersons are usually selected due to their critical domain knowledge, understanding of best practices, ability to identify risks and keep members engaged, and expansive relationships within their industry. Maximizing the chairperson’s role requires discussion on how to utilize their valuable domain knowledge and professional networks. Chairs possess extensive networks that could support the identification of permanent or temporary expert participants for AdComms, aiding the FDA’s mission to recruit top talent for guidance. This would ensure the FDA’s continued success in recruiting the brightest minds in the industry to assist with providing advice. Additionally, chairs should have oversight in identifying relevant issues or products for their respective committees to appraise, which can provide another layer for the FDA to keep abreast of critical public concerns via appropriate committee evaluation. 

Training

Training is a significant part of many Federal Government service positions. However, besides ethics and conflict of interest trainings, there is no set training program in place for most new Advisory Committee members. Considering Advisory Committee members come from different professional backgrounds with varying levels of expertise, the FDA should develop an onboarding training program to assist with acclimating all new AdComm members into their roles. Many former and current AdComm members mentioned that no formal training was provided as part of the onboarding process. Some members who were new to the FDA AdComm process or who were not physicians or scientists stated they had no knowledge of statistical analyses, clinical trial design, or how the FDA views the role of the AdComm in the regulatory process. 

A foundational training, covering these aspects, would greatly benefit those members such as consumer and patient representatives who may lack this shared base of expertise. An investment in such an onboarding experience would promote stronger rapport among members and guarantee their preparedness in analyzing scientific and technical submissions.

Quotes from the Committee
“There should be basic training for all laypeople on the committee. This would help to prepare them for fulfilling their duties.”
“There should be an orientation that explains the relationship between the advisory committee and the FDA, and how the FDA thinks about this process.”

Learning about the FDA Regulatory Process

The Food & Drug Administration (FDA) was established with the purpose of regulating drugs and medical devices to ensure their safety and effectiveness for all citizens in the United States. Many Advisory Committee members join these committees without basic knowledge of the FDA’s regulatory process. During FAS’ discussions with current and former AdComm members, approximately 71% of members stated that basic knowledge of the regulatory process and how the FDA makes their decisions was unknown to them.

Providing AdComm members with an introductory course on the FDA’s regulatory process could enhance their comprehension, potentially allowing them to make more effective contributions and informed clinical decisions (based on their occupation). Although the FDA provides some online resources about its processes, like FAQs and guidelines, an expansion of this material should be considered for inclusion in AdComm orientation activities.  

Conflict of Interest (COI) Process

18 U.S.C. 208(a) prohibits Advisory Committee members who are designated as special government employees (SGE) from serving on federal advisory committees or any other Federal Government form of service that will have a “direct or predictable effect” on their financial interests. Similarly, the FDA describes a conflict of interest as an occurrence “when an individual selected to serve on an advisory committee has financial interests that may be impacted by the individual’s work on the advisory committee”. The auditing process for conflicts of interest is designed to confirm that the members of the advisory committee maintain impartiality and ensure the integrity of public health safety. Prior to any committee gathering, the FDA mandates that each participant, classified as either an AdComm member or SGE, complete an FDA 3410 form that reveals all financial connections that could be seen as potential COIs.

However, the process of what happens after the 3410 form has been completed is ambiguous. In 2007, the FDA submitted draft guidance to the federal register for comment entitled, Guidance for the Public, FDA Advisory Committee Members, and FDA Staff on Procedures for Determining Conflict of Interest and Eligibility for Participation in FDA Advisory Committees in an effort to determine if there is an inappropriate COI that should exclude members from participating in a committee meeting. Moreover, the official guidance is not easily accessible. Another draft guidance was developed with a detailed listing of considerations to be given when examining conflict of interests can be found in the FDA’s draft guidance on Procedures for Evaluating Appearance Issues and Granting Authorizations for Participation in FDA Advisory Committees.

Discussions with current and former AdComm members about the COI auditing process sparked varying views regarding whether flexibility should be exercised for COIs. 82% of members concurred that while they recognize the necessity for such a system, it tends to be overly demanding due to repetitive paperwork, especially when their circumstances remain unchanged. The strenuous nature of this routine has even deterred some from continuing their membership each year and remains a key aspect as to why members choose to end their service.

Despite having a COI process in place, there are loopholes that allow members with conflicts of interest to remain as voting members for specific AdComm meetings. A certain incident involved an Advisory Committee where 10 members who had financial ties to the sponsor were allowed to participate in an AdComm meeting. These individuals ultimately took part in endorsing the TriClip G4 System by Abbott and unanimously agreed that its benefits outweighed the potential risks. To further complicate matters, this information was not disclosed to the public at the time of approval.

While the COI process has resulted in members being rightfully disqualified from meetings due to actual or apparent conflicts, there is room to refine how these conflicts are identified and the standards employed to judge permissible COIs.

Policy Recommendations

In an effort to better leverage Advisory Committee membership, the potential policy recommendations are as follows:

Committee Composition

Role of the Chair

Training and Regulatory Process

COI Auditing Process

Conclusion

Advisory Committees are pivotal to maintaining trust with the public. It is essential for public safety to ensure that the most qualified experts are selected to serve on these Committees and that they have the tools to provide the FDA with informed and evidence-based recommendations. In an effort to increase public health safety, the FDA should enhance the AdComm structure by expanding the chair’s role, creating training programs for all new Advisory Committee members, and revising the conflict of interest procedures.