To help catalyze innovation in the health and biomedical sciences, research and development (R&D) paradigms with a track record of producing ‘moonshot’-scale breakthroughs – such as the Advanced Research Projects Agency (ARPA) model – stand at the ready. The Biden Administration has recognized this, proposing the establishment of an ARPA for health (ARPA-H) as part of its fiscal year 2022 budget request. Done right, ARPA-H would be created in the image of existing ARPAs – DARPA (defense), ARPA-E (energy), and IARPA (intelligence) – and be capable of mobilizing federal, state, local, private sector, academic, and nonprofit resources to directly address the country’s most urgent health challenges, such as the high cost of therapies for diseases like cancer, or antimicrobial resistance. During a recent House Energy and Commerce Committee hearing, Chairwoman Anna Eshoo (D-CA) raised the Administration’s proposal for ARPA-H with Department of Health and Human Services (HHS) Secretary Xavier Becerra, expressing her interest in exploring how to best position a potential ARPA-H for success.

Keys to the ARPA model

The success of the ARPA model is attributed in part to the high level of autonomy with which its program leaders select R&D projects (compared to those at traditional federal research agencies), a strong sense of agency mission, and a culture of risk-taking with a tolerance for failure, resulting in a great degree of flexibility to pursue bold agendas and adapt to urgent needs. Policymakers have debated situating a potential ARPA-H within the National Institutes of Health (NIH), or outside of NIH, elsewhere under the umbrella of HHS. Regardless, it is essential that ARPA-H retain an independent and innovative culture.

The first ARPA – DARPA – was established in 1958, the year after Sputnik was launched, and is credited with developing GPS, the stealth fighter, and computer networking. DARPA continues to serve its customer – the Department of Defense – by developing groundbreaking defense technologies and data analysis techniques. Nevertheless, DARPA operates separately from its parent organization. This is also true of ARPA-E, which was launched in 2007 based on a recommendation from a National Academies consensus study report which called for implementing the DARPA model to drive “transformational research that could lead to new ways of fueling the nation and its economy,” and IARPA, created in 2006, to foster advances in intelligence collection, research, and analysis.

If ARPA-H is organized within NIH, it is essential that it maintain the innovative spirit and independence characteristic of established ARPAs. NIH already has some experience overseeing a partially independent entity: the National Cancer Institute (NCI). Compared to other NIH institutes, NCI’s unique authorities include:

• Direct access to the president;
• A requirement to submit a completely separate budget proposal to the president each year without getting approval from NIH or HHS; and
• The ability of the NCI director to form new cancer centers and training programs, establish advisory committees, and independently collaborate with other federal, state, and local entities.

This level of independence has contributed to NCI achieving a number of significant milestones in cancer treatment, including developing a chemotherapy treatment to cure choriocarcinoma (a rare type of cancer that starts in the womb), publishing the now-widely-used Breast Cancer Risk Assessment Model, and creating an anticancer drug for ovarian cancer that was unresponsive to other treatments.

If the NCI model were to be used as the foundation for the launch of ARPA-H, insulation from political considerations, whether those of Congress or the Executive Branch, would be critical. With DARPA-like autonomy, a potential ARPA-H could help push the boundaries of enrichments to human health.

Antimicrobial resistance as a case study for an ARPA-H

An example of a grand challenge that an ARPA-H could take on is addressing antimicrobial resistance, a worsening situation that, without intervention, will lead to a significant public health crisis. Antimicrobial resistance occurs when “bacteria, viruses, fungi, and parasites change over time and no longer respond to medicines, making infections harder to treat and increasing the risk of disease spread, severe illness, and death.” Microbes have the potential to gain resistance to drugs when not all of the pathogens or parasites are killed by a treatment, either because the treatment was the not correct option for the illness (like using antibiotics for viruses), or refraining from completing a prescribed course of an antimicrobial drug. The organisms that are not killed, presumably because they harbor genetic factors that confer resistance, then reproduce and pass along those genes, which make it harder for the treatments to kill them.

The most immediate concerns regarding antimicrobial resistance come from bacteria and fungi. The CDC considers some of the biggest threats to be Acinetobacter, Candida auris, and C. difficile, which are often present in healthcare and hospital settings and mainly threaten the lives of those with already weakened immune systems. Every year in the U.S., almost 3 million people are infected with antimicrobial-resistant bacteria or fungi, and as a result, more than 35,000 people die. While the toll of antibiotic resistance in the U.S. is devastating, the global outlook is perhaps even more concerning: in 2019, the United Nations warned that if no action is taken, antimicrobial resistance could cause 10 million deaths per year worldwide by 2050.

Developing new and effective antibiotics can help counter antimicrobial resistance; however, progress has been extremely slow. The last completely new class of antibiotics was discovered in the late 1980s, and developing new antibiotics is often not profitable for pharmaceutical companies. It is estimated that it takes $1.5 billion to create a new antibiotic, while the average revenue is about $46 million per year. In addition, while pharmaceutical companies receive an exclusivity period during which competitors cannot manufacture a generic version of their drug, the period is only five to ten years, which is too short to recoup the cost of research and development. Furthermore, doctors are often hesitant to prescribe new antibiotics in hopes of delaying the development of newly drug-resistant microbes, which also contributes to driving down the amount pharmaceutical companies earn for antibiotics.

Early last year, the World Health Organization reported that out of 60 antibiotics in development, there would be very little additional benefit over existing treatments, and few targeted the most resistant bacteria. Moreover, the ones that appeared promising will take years to get to the market. This year, Pew Research conducted a study on the current antibiotic development landscape and found that out of 43 antibiotics under development, at least 19 have the potential to treat the most resistant bacteria. However, the likelihood of all, or even some of these products making it to patients is low: over 95 percent of the products in development are being studied by small companies, and more than 70 percent of these companies do not have any other products on the market.

There is both a dire need for new innovations in the space, such as using cocktails of different viruses that attack bacteria to treat infections, and a gap between the research into and commercialization of new antibiotics – a perfect opportunity for a potential ARPA-H to make an impact. With this new agency, experimental treatments could be supported through the technology transfer process and matured to the point that the private sector is able to take the baton and move a new antimicrobial to market. This would be revolutionary for public health, and, combined with improved messaging around best practices for the use of antibiotics, save many lives.

Moving forward

The need for, structure, and possible priorities of a potential ARPA-H will continue to be discussed over the course of the congressional appropriations process, with consultation between the Legislative and Executive Branches. We encourage the CSPI community to serve as a resource for Members of Congress and their staffs to ensure that the new agency will be properly positioned to contribute to significant advances in human health and biomedical technologies.

WASHINGTON, D.C. — Today, the Federation of American Scientists (FAS), with financial support from Schmidt Futures, announced that six organ procurement organizations (OPOs) have joined the FAS Organ Procurement Organization Innovation Cohort, committing to use data science and transparency to accelerate improved patient outcomes and to inform ongoing, data-driven policy development. 

This follows the finalization of the bipartisan, scientifically-informed OPO rule that can save more than 7,000 lives each year, and which has been highlighted by both Senate and House leaders as an urgent equity issue. Given COVID-19’s potential to affect and attack organs, coupled with its disproportionate impact on communities of color, the need for reform is only intensifying.

Through the Federation of American Scientists, the OPO Innovation Cohort will share data to establish open and transparent lines of communication between OPOs as nonprofit government contractors and the public they serve, including branches of the federal government, in an effort to build trust and support further reforms that will save patient lives. (See data visualization from the OPO final rule here.)

Working with alumni from the United States Digital Service, over the next 12 months the Innovation Cohort will leverage the most granular OPO data ever shared with external researchers to inform ongoing policy development at the U.S. Department of Health and Human Services (HHS) and in Congress. During a transformative period in the organ procurement industry, the Innovation Cohort will help shape the future of organ recovery in America, improving OPO practice and informing OPO policy. Most importantly, the Innovation Cohort will help return OPOs to their core mission by singly focusing on striving toward new heights of operational excellence in order to increase organ transplants in an effort to best serve the public, organ donors, donor families and patients waiting for transplants.

In the coming months, the FAS OPO Innovation Cohort will share additional de-identified, retrospective data with the Federation of American Scientists to be published openly – including all referrals for donation made to the OPOs with every outcome documented, audits of hospital-level deaths, OPO financials (including organ acquisition charges), procurement and organ recovery data from organ recovery centers, and staffing models – and will work actively to source data science partners and researchers to mine these datasets for performance improvement insights.

“COVID-19’s ravaging effect on organs has further increased the urgency of accelerating accountability for the government’s contractors in organ donation. Transparency is a critical first step, and the Federation of American Scientists applauds today’s commitments from six OPO leaders to break from their peers and prioritize patients and the public interest.”

Federation of American Scientists Acting President Dan Correa

“So many of the problems and inefficiencies of the organ waiting list are solvable, but we need a new, data-driven approach. We look forward to seeing how the OPO Innovation Cohort, paired with interdisciplinary talent, can bring transformational change to a sector in dire need of it.”

Schmidt Futures Managing Director and Head of Partnerships Kumar Garg

The six OPO CEOs below have underscored their commitment to the following principles:

• Transparency: public sharing of data/analysis in order to set a standard to which all OPOs can be held;
• Accountability: support for the OPO final rule, and any efforts to move up implementation date so all parts of the country can be served by high-performing OPOs as soon as possible in 2024; and
• Equity: commitment to analyzing/publishing data to ensure all parts of community served.

Diane Brockmeier, Mid-America Transplant

Helen Irving, LiveOnNY

Ginny McBride, Our Legacy

Patti Niles, Southwest Transplant Alliance

Kelly Ranum, Louisiana Organ Procurement Agency

Matthew Wadsworth, LifeConnection of Ohio

Further, as the House Committee on Oversight and Reform is investigating “poor performance, waste, and mismanagement in organ transplant industry”, the OPOs in the FAS OPO Innovation Cohort offer themselves as a resource for Congressional staff, noting their commitment to transparency, accountability, and equity, setting a standard to which all OPOs should be held. The participating OPOs have informed AOPO that they are leaving AOPO, noting the Committee’s investigation into AOPO’s “lobbying against life-saving reforms.”

A full visualization of the final rule from Bloom can be viewed here.

Summary

Cancer patients urgently need more effective treatments that are accessible to everyone. This year alone, an estimated 1.9 million people in the United States will receive new cancer diagnoses, and cancer will kill more than 600,000 Americans. Yet there are no targeted therapeutics for many cancers, and the treatments that do exist can be prohibitively scarce or expensive.

Repurposing existing drugs, especially off-patent generics, is the fastest way to develop new treatments. Hundreds of non-cancer generic drugs have already been tested by researchers and physicians in preclinical and clinical studies for cancer, some up to Phase II trials, and show intriguing promise. But due to a market failure, there is a lack of funding for clinical trials that evaluate generic drugs. This means that there isn’t conclusive evidence of the efficacy and safety of repurposed generics for treating cancer, and so cancer patients who desperately need more (and more affordable) treatment options are unable to realize the benefits that existing generics might offer.

To quickly and affordably improve the lives of cancer patients, the Biden-Harris Administration should create the Repurposing Generics Grant Program through the National Cancer Institute. This program would fund definitive clinical trials evaluating repurposed generic drugs for cancer. A key first step would be for President Biden to include this program in his FY2022 budget proposal. Congress could then authorize the program and related appropriations totaling $100 million over 5 years.

Introduction

For those who can afford to fill their fridge by clicking a button on their smartphone or walking around to the organic grocery around the corner, it is easy to forget how complex and fragile our food systems can be. However, for millions of Americans who suffer from poor health because of food insecurity, or farmers and ranchers whose yields are decreasing along with the nutrient density of their product, that fragility is felt every day. Sustainable food systems engender intricate connections and feedback loops among climate change, public health, food security, national security, and social equity. When one of these factors is overstressed, disaster can result.

COVID-19 has underscored the vulnerability of our food systems. The pandemic caused restaurants to close overnight, strained supply chains, and led to food rotting on land, in warehouses, and on shelves. Low-income and food-insecure families waited in lines that stretched for miles while producers and distributors struggled to figure out how to get supplies to those who needed them. Concurrently, generations of racial inequity and the coordinated disenfranchisement of Black, Indigenous, and other people of color (BIPOC) has crystalized as an issue that needs to be addressed at every level in our country, especially within our food and agricultural systems.

Addressing these issues—now and for the future—requires a coordinated response across sectors. Food security is deeply intertwined with public health and social equity. Un- and under- employment, the racial wealth gap, and increased financial hardships for certain communities result in increased malnutrition, obesity, metabolic diseases, and chronic illness, as well as particular susceptibility to severe impacts from COVID-19 infections during the present pandemic. The climate crisis compounds these issues. Farming practices that degrade soil health, reduce agriculture capacity, and compromise the well-being of small farms and rural communities prevent us as a nation from becoming healthier and more secure. As we look at opportunities to “build back better,” we must embrace paradigmatic shifts—fundamental restructuring of our systems that will support equitable and inclusive futures. Compounding crises require changes in not only what we do, but how we think about what we do.

A fundamental problem is that progress in modern agriculture has been implicitly defined as progress in agricultural technology (AgTech) and biotechnology. Little emphasis is placed on examining whole-systems dependencies and on how connections among soil health, gut bacteria, and antibiotic use in livestock impact human health, economic prosperity, and climate change. With such a narrow view of “innovation,” current practices will solve a handful of isolated problems but create many more.

Fortunately, alternatives are ripe for adoption. Regenerative farming, for instance, is a proven way to combat future warming while increasing the adaptive capacity of our lands, providing equitable access to food, and creating viable rural economies. Regenerative farming can also restore soil health, which in turn improves food quality while enhancing carbon sequestration and providing natural water treatment.

Transitioning away from dominant but harmful practices is not easy. The shift will require an inclusive innovation ecosystem, investors with long time horizons, new infrastructure, tailored education, economic incentives, and community safety nets. This document explores how the agricultural sector can support, and be supported by, policies that advance science, technology, and innovation while revitalizing living systems and equitable futures. We recognize that agricultural policy often overlooks interventions that are appropriately suited to advance these concepts with Black, Indigenous, people of color (BIPOC) communities and on tribal lands. To avoid this mistake, the concepts presented herein start from the ground up. We focus on the benefits of improving soil health and food security through regenerative agricultural activities, and provide examples of policies that could promote such activities in a variety of ways. Letting practice drive policy— instead of having policy dictate practice—will result in more sustainable, inclusive outcomes for all communities.

While agricultural policy can and should be shaped at the local, regional, state, and national level, this document places special emphasis on the role of the federal government. Building better food systems will require multiple government agencies, especially federal agencies, to collaboratively advance more equitable policies and practices. Most national agricultural programs are housed within the U.S. Department of Agriculture (USDA). But the interconnectedness of how we produce food and fiber (and the ways in which those practices impact our environment and nourish people) demands priority investment not only from USDA, but also from the Environmental Protection Agency, the Department of Energy, the Department of the Interior, the Department of Defense, and the Department of Health and Human Services—to name just a few. This document—based on a review of existing policy recommendations and current practice, development and refinement of new ideas, and identification of underleveraged roles and programs within the government— suggests what such investments might look like in practice.

As the U.S. continues to grapple with the pandemic, there are growing concerns about the risks posed by variants of SARS-CoV-2 – the coronavirus that causes COVID-19. Recent data have shown that at least one SARS-CoV-2 variant is more transmissible than the original, and there are questions as to whether any variants could be more deadly. The main way to detect emerging variants is to perform widespread genome sequencing, but the sequencing infrastructure in the U.S. is struggling to keep up with demand. This issue was a major focus of the Centers for Disease Control and Prevention’s (CDC) briefing to the House Appropriations Subcommittee on Labor, Health and Human Services, Education and Related Agencies this week.

Origin of variants and their detection

Viruses replicate by taking advantage of a person’s own cells, and each replication introduces small changes into a virus’ genetic code. Usually, these mistakes either have no impact, or are harmful to the virus. Sometimes, though, these errors give the virus an advantage, like increased ability to infect other people. Besides increased transmissibility, it is possible that variants could also cause more severe disease, evade detection by diagnostic tests, reduce the effectiveness of treatments, escape infection-induced immunity, or render vaccines less effective.

These risks are why it is imperative that public health officials track the emergence of variants around the country, and around the globe. Variants are found by extracting genetic material from patient samples, using sequencing equipment to read the virus’ genetic code, and comparing it with other known samples. When increasing numbers of cases of disease are found to have been caused by a virus with a genetic signature that is only slightly different from that of the known samples, scientists can estimate that they may have found a new variant. For SARS-CoV-2 specifically, there are a few variants that appear to have an advantage and are able to spread much more easily than the original strain. These variants include the UK and South African strains. There are also some early data that a variant discovered in California is more contagious than the original.

Challenges for genome sequencing of viruses in the U.S.

Public health officials use genomic sequencing to monitor for a variety of viruses, but the increased demand during the COVID-19 pandemic has put the U.S.’ sequencing infrastructure under strain. Though the U.S. has over 28 million COVID-19 cases, or about one-fourth of the total number of cases in the world, only about 96,000 samples, or around 0.3 percent, have been sequenced. For U.S. labs, the sequencing process can be costly and time-consuming, taking 48 hours to readout a virus’ genome in the best case scenario, though typical turnaround times stretch up to seven days. The cost of just one virus genome sequence can be anywhere from $80 to $500.

The country’s current genomic sequencing infrastructure has not been prioritized as a public health need and, in the past, sequencing was typically performed only by research universities. In 2014, the CDC started funding public health labs to track foodborne illnesses with genomic sequencing. By 2017 every state had labs which could perform genome sequencing, but obtaining funding is still difficult.

Current efforts and the road ahead

The CDC has been working to form various partnerships to boost the U.S.’ capacity for virus genome sequencing. According to its website, CDC has focused on several activities to increase genomic sequencing capacity, including:

• Leading the National SARS-CoV-2 Strain Surveillance (NS3) system;
• Partnering with commercial diagnostic laboratories;
• Collaborating with universities;
• Supporting state, territorial, local, and tribal health departments; and
• Leading the SARS-CoV-2 Sequencing for Public Health Emergency Response, Epidemiology, and Surveillance (SPHERES) consortium.

CDC Director Rochelle Walensky echoed this during Tuesday’s briefing and noted that under her leadership, the agency has scaled from 250 SARS-CoV-2 sequences per week to 14,000 per week. She hopes to scale up enough that the CDC can sequence 25,000 samples per week, which is close to about 5% of positive cases. To do this, the White House announced last week it would provide $200 million to support more genomic sequencing, and the U.S. Congress is considering adding almost $2 billion to that effort in the next economic relief package.

This funding is also intended to sustain the U.S.’ genomic sequencing infrastructure for the future. Senator Tammy Baldwin (D-WI), who introduced the legislation to support further sequencing, said the federal government should establish “the basis of a permanent infrastructure that would allow us not only to do surveillance for COVID-19, to be on the leading edge of discovering new variants, but also…have that capacity for other diseases.” During Tuesday’s briefing, Ranking Member Tom Cole (R-OK) affirmed this idea, saying that the House Appropriations Committee needs to think about establishing long-term funding streams to ensure that infrastructure developed during this crisis can last well in the future.

The COVID-19 pandemic has highlighted gaps in U.S. infrastructure for the genomic sequencing of pathogens, and the importance of tracking virus variants for our public health. While the CDC works with its partners to rapidly scale up sequencing capacity, lawmakers need to consider how to sustain it for future outbreaks. As the Biden Administration and Congress consider scaling and sustainment, we encourage the CSPI community to serve as a resource to federal officials on this topic.

Given the pervasiveness of COVID-19 throughout the U.S., the risk of infection to transportation workers and passengers is significant. For instance, in a survey of over 600 bus and subway workers in New York City, almost one quarter reported contracting COVID-19, and 76 percent personally knew a coworker who had died from the disease. During last week’s hearing, the House Transportation and Infrastructure Committee discussed best practices for protecting transportation workers and passengers from COVID-19, with a particular focus on preventing the spread of the coronavirus through the air.

Transmission of COVID-19 via aerosols

In early October 2020, the Centers for Disease Control and Prevention (CDC) updated its guidance, confirming that COVID-19 can be transmitted via aerosols in addition to larger respiratory droplets. When an individual with COVID-19 coughs, speaks, or breathes, tiny coronavirus-carrying droplets can travel over distances longer than six feet and stay suspended in the air for up to several hours. For the coronavirus, most transmission via aerosols occurs in enclosed, poorly ventilated spaces, when a person is exposed to respiratory particles for an extended period of time. Mass transit vehicles can be one such pathway for infection since people from different households share the same spaces while either working or riding to their destinations.

Protecting people from COVID-19 involves implementing measures to keep virus particles from entering individuals’ noses and mouths. Scientists have found that wearing a face covering can limit the amount of droplets an individual releases, and thus also reduce the amount of virus particles in the air. Masks can also provide some degree of protection to the wearer by providing a barrier between coronavirus-carrying droplets and the person’s nose and mouth. The CDC also suggests that buildings and transportation systems examine the quality of their ventilation and filtration systems to reduce spread of COVID-19. Effective ventilation quickly dilutes the amount of virus particles in the air and allows clean air to quickly circulate in enclosed spaces. Advanced filtration systems can help catch and retain virus-carrying particles on tightly woven inserts, keeping them from reentering the space. While any one of these methods alone is not sufficient to protect people from the coronavirus, a layered approach that combines many safeguards can reduce the ability of respiratory diseases like COVID-19 to spread.

Using advanced filters to remove coronavirus-carrying particles from enclosed spaces

Several Members of the Committee noted the importance of developing and implementing advanced filtration technologies on transportation systems and in buildings. Scientists estimate that the coronavirus can spread even via airborne particles under 5 microns in diameter. (For comparison, a single raindrop is typically about 2,000 microns in diameter.) Most buildings have filters with a Minimum Efficiency Reporting Value (MERV) rating of between 7 and 8, which means they can filter up to 84.9 percent of particles between 3 and 10 microns in diameter. Subway cars also use these filters. The highest rated filters (MERV 16 to 20) can capture over 75 percent of particles that are between 0.3 and 1 micron in diameter, and high efficiency particulate air (HEPA) filters, which are used on airplanes, can theoretically remove at least 99.97 percent of particles 0.3 microns in diameter and larger. To better protect workers and passengers, transportation systems like Washington, DC’s Metro and the Bay Area Transit system in San Francisco are already testing out more advanced filtration technologies through pilot programs funded by the Federal Transit Administration.

Benefits of advanced filters beyond reducing spread of COVID-19

Widespread adoption of advanced filtration technologies can be beneficial not only to reduce the amount of coronavirus-carrying particles in the air, but also to trap other harmful aerosols. During the hearing, Dr. David Michaels from George Washington University and Dr. William Bahnfleth from Penn State University both noted that investing in better filters now can also protect people from inhaling harmful particulate matter from other sources. For example, wildfires contribute about 30% of all fine particulate emissions in the U.S., with many of these being 2.5 microns or smaller. Inhaling these harmful particles can be associated with cardiovascular and respiratory issues, as well as premature mortality, particularly in vulnerable groups such as the elderly, children, and pregnant women.

Enhanced air filtration is a useful tool to help slow the spread of COVID-19, especially when used alongside other measures like wearing masks, improving ventilation, social distancing, and hand washing. As the new administration and Congress work toward ending the pandemic, practices such as the widespread adoption of more robust filters are likely to be examined in more detail. We encourage our community to get involved in the effort to counter COVID-19 by engaging in future congressional hearings through our Calls to Action.

Summary

The coronavirus pandemic has forced a sudden acceleration of a prior trend toward the virtual provision of healthcare, also known as telemedicine. This acceleration was necessary in the short term so that provision of non-urgent health services could continue despite lockdowns and self- isolation. Federal and state policymakers have supported the shift toward telemedicine through temporary adjustments to health benefits, reimbursements, and licensure restrictions.

Yet if policymakers direct their attention too narrowly on expanding telemedicine they risk missing a larger—and as yet mostly unrealized—opportunity to improve healthcare in the United States: increasing the overall share of health services provided directly to the home. At-home healthcare includes not only telemedicine, but also medical house calls (home-based primary care) as well as models in which individuals within communities offer simple support services to one another (i.e., the “village” model of senior care, which could be extended to included peer- to-peer health service delivery). The advent of “exponential” technologies such as artificial intelligence (AI), blockchain, and the Internet of Things (IoT) is unlocking new possibilities for at- home healthcare across each of these models.

The next administration should act to reduce four types of barriers currently preventing at-home healthcare from reaching its full potential:

1. Labor-market barriers (e.g., unnecessarily restrictive scope-of-practice rules and requirements for licensing and certification)
2. Technical barriers (e.g., excessively slow and burdensome processes for regulatory approval, weak or absent standards for interoperability)
3. Financial/regulatory barriers (e.g., methodologies for determining eligibility for reimbursements that favor incumbents over innovators)
4. Data sharing / interoperability barriers (e.g., overly restrictive constraints related to data privacy and portability)

Summary

Prevention plays a crucial and underappreciated role in our health system. To improve health outcomes and bring down costs, it will be important to establish a better balance between preventive measures and drug treatments. The next administration should provide incentives to healthcare providers that scale up—and reduce costs of delivering—preventive interventions with demonstrated efficacy. Currently, the U.S. Department of Health and Human Services (HHS) sets broad standards regarding managed care contracts. But states have considerable latitude. States can set income eligibility criteria, define services, and set alternative payment methods with Managed Care Organizations (MCOs). And in just the last few decades, Medicaid programs have been almost fully privatized: MCOs now cover over 85% of the Medicaid population. Because of the existing patchwork of insurance programs and state rules, it is important that regulations set minimum national standards to ensure that health care is accessible and affordable for those who need it the most. Particularly important to this effort are non- distortionary prices and reimbursement policies.

For a few decades, policymakers have, with bi-partisan consensus, moved away from a fee-for-service (FFS) system whereby providers are paid for service delivery and toward capitation and pay for performance (p4p) models. While these models offer significant improvements over FFS models, each involves risks of incentivizing non-optimal care and expenditures if they are not structured carefully. When paying capitation rates, bonuses adjusting for population risk alone should be avoided as this incentivizes an increase in diagnoses without necessarily improving care. Either all health care payments should be p4p, or a p4p component should be added to the capitation base. Pharmacological interventions should also be included in the overall provider reimbursement structure to align reimbursement incentives with health outcomes. Healthcare providers will then determine the right mix of services. Furthermore, while p4p is generally a good idea (i.e., hospitals and MCOs are rewarded for decreasing the number of avoidable hospital readmissions), if this metric is not applied homogeneously across all services, this payment structure significantly hampers the provision of preventive services.

Summary

From HIPAA to doctor-patient confidentiality, the U.S. healthcare system is replete with provisions designed to ensure patient privacy. Most people are surprised, then, to hear that patients in the United States do not legally own nearly any of their health data: data as diverse as health and medical records, labs, x-rays, genetic information, and even physical specimens such as tissue and blood removed during a procedure.

Providing patients with agency over their health data is necessary for elevating patients as partners in their own health management—as individuals capable of making genuinely informed and even lifesaving decisions regarding treatment options.

The next administration should pursue a two-pronged approach to help do just that. First, the administration should launch a coordinated and comprehensive patient-education and public- awareness campaign. This campaign should designate patient data and tissue rights as a national public-health priority. Second, the administration should expand provisions in the Cures 2.0 Act to ensure that healthcare providers are equally invested in and educated about these critical patient issues. These steps will accelerate a needed shift within the U.S. healthcare system towards a culture that embraces patients as active participants in their own care, improve health- data literacy across diverse patient populations, and build momentum for broader legislative change and around complex and challenging issues of health information and privacy.

There has been a surge of public interest in the drug fluvoxamine as a potential treatment for individuals with mild COVID-19, and Congressional offices are receiving many questions about the possibility of using the drug to counter COVID-19 from constituents. This brief outlines what is known to date about fluvoxamine in the context of the coronavirus pandemic in order to help both policymakers and scientists discuss this issue with those in their communities.

Fluvoxamine is a long-used drug that showed promising preliminary results in a small, well-controlled COVID-19 patient study

The generic drug fluvoxamine (also referred to by the brand name Luvox) was first synthesized in 1971, and is used to treat anxiety, depression, and obsessive-compulsive disorder. Fluvoxamine blocks serotonin reuptake in the brain, but it is chemically unrelated to other selective serotonin reuptake inhibitors that are used to treat anxiety or depression, like fluoxetine (Prozac) or sertraline (Zoloft). Studies have demonstrated that fluvoxamine also binds a protein in mammalian cells called the sigma-1 receptor. One of this receptor’s functions is to regulate cytokine production; cytokines cause inflammation. When fluvoxamine has been used in the laboratory, it results in a dampened inflammatory response in human cells in the test tube, and protects mice from lethal septic shock, which is an out-of-control immune response to infection, causing massive inflammation that can impede blood flow to major organs, and result in organ failure. Notably, retrospective analyses have indicated that COVID-19 patients given antipsychotic drugs that target the sigma-1 receptor were less likely to require mechanical ventilation than COVID-19 patients given other antipsychotic drugs, and some drugs that bind the sigma-1 receptor also have antiviral activity against SARS-CoV-2 in the petri dish.

Researchers reasoned that fluvoxamine may be able to stave off the “cytokine storm” that can lead to the out-of-control inflammatory response that appears to cause severe respiratory and blood-clotting issues for some people infected with the coronavirus, and tested the drug in a pilot study to gauge whether it has potential as a treatment for COVID-19. The study was a small, double-blind, placebo-controlled, randomized clinical trial of 152 non-hospitalized adults with mild COVID-19. Treatment of symptomatic, confirmed COVID-19 patients started within 7 days of their diagnosis. None of the 80 patients treated with fluvoxamine experienced clinical deterioration, compared to 6 of 72 patients treated with placebo who experienced both “1) shortness of breath or hospitalization for shortness of breath or pneumonia and 2) oxygen saturation less than 92%.” While this amounted to a statistically significant difference, the study serves as only preliminary evidence for the efficacy of fluvoxamine as a therapy to counter COVID-19, and a much larger clinical trial has been initiated to pursue conclusive results.

Summary of the 152-participant fluvoxamine/COVID-19 clinical trial. Image source: The Journal of the American Medical Association

Further study of treating many more COVID-19 patients with fluvoxamine is required before the drug should be used outside of clinical trials as a therapy to counter COVID-19

While the double-blind, placebo-controlled, randomized design of the clinical trial does minimize bias and provide the opportunity to identify a causal relationship between treatment and patient outcome, larger randomized trials with more definitive metrics in place for assessing patients’ health status are necessary in order to reach a conclusion about whether fluvoxamine should be used to treat patients with COVID-19 outside of clinical trials. The findings of this single, small study are a launching point for larger clinical trials, and “should not be used as the basis for current treatment decisions.”

The limitations of the study include the small number of COVID-19 patients involved and the low number of patients in the placebo group whose conditions worsened – only six. And while fluvoxamine is safe, easily accessible, administered orally, and inexpensive, it may interact with other drugs, and does have some side effects, such as nausea, diarrhea, loss of appetite, increased sweating, dizziness, drowsiness, insomnia, or dry mouth.

The researchers who performed this pilot study are currently conducting a larger clinical trial to conclusively determine whether fluvoxamine is an advisable treatment for mild COVID-19. The trial is expected to be watched closely since the identification of drugs – in addition to the monoclonal antibody treatments developed by Regeneron and Eli Lilly – that could be used to reduce the likelihood of progression from mild to more severe COVID-19 would greatly improve health outcomes for people infected by the coronavirus, as well as reduce the burden on the US healthcare system.

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This briefing document was prepared by the Federation of American Scientists along with Professor Alban Gaultier at the University of Virginia and Professor David Boulware at the University of Minnesota.

Summary

While the Veterans Health Administration (VHA) provides telehealth services across the country, current services neglect to respond to the access challenges that constrain veterans, particularly in rural areas. Of the nearly 5 million veterans who live in rural areas, 45% lack access to reliable broadband internet and smart technology. In the absence of available or reliable internet, veterans are often forced to access telehealth services in person at VA Clinical Resource Hubs (CRHs). However, these facilities are limited in number and are typically located far from rural communities. To address digital inequities and constraints posed by infrastructure and geography, the VHA needs to create more ways for veterans to access and fully utilize telehealth. We propose that the VHA partner with federal agencies like the United States Postal Service (USPS) or United States Department of Agriculture (USDA), leveraging their infrastructure to develop telehealth hubs. We further suggest that the VHA develop and lead a federal taskforce to build critical technology infrastructure that will facilitate expansion and use of telehealth for veterans. These interventions will be vital for ensuring that veterans in rural communities have greater access to care and can not only survive but thrive.

Summary

Optimizing the dosing of many expensive drugs can drastically reduce both costs and toxicities. The Federal Government, state governments, employers, and individual patients could collectively save tens of billions of dollars each year by simply optimizing the dosing of the most expensive prescription drugs on the market, particularly in oncology. Optimized dosing can also improve health outcomes. The next administration should, therefore, launch an effort to control the cost of prescription drugs through an evidence-based approach to optimizing drug dosing and improving outcomes. The requisite trials pay for themselves in immediate cost savings.