Slow Aging, Extend Healthy Life: New incentives to lower the late-life disease burden through the discovery, validation, and approval of biomarkers and surrogate endpoints
The world is aging. Today, some two thirds of the global population is dying from an age-related condition. Biological aging imposes significant socio-economic costs, increasing health expenses, reducing productivity, and straining social systems. Between 2010 and 2030, Medicare spending is projected to nearly double – to $1.2 trillion per year. Yet the costly diseases of aging can be therapeutically targeted before they become late-stage conditions like Alzheimer’s. Slowing aging could alleviate these burdens, reducing unpaid caregivers, medical costs, and mortality rates, while enhancing productivity. But a number of market failures and misaligned incentives stand in the way of extending the healthy lifespan of aging populations worldwide. New solutions are needed to target diseases before they are life-threatening or debilitating, moving from retroactive sick-care towards preventative healthcare.
The new administration should establish a comprehensive framework to incentivize the discovery, validation, and regulatory approval of biomarkers as surrogate endpoints to accelerate clinical trials and increase the availability of health-extending drugs. Reliable biomarkers or surrogate endpoints could meaningfully reduce clinical trial durations, and enable new classes of therapeutics for non-disease conditions (e.g., biological aging). An example is how LDL (a surrogate marker of heart health) helped enable the development of lipid-lowering drugs. The current lack of validated surrogate endpoints for major late-life conditions is a critical bottleneck in clinical research. Because companies do not capture the majority of the benefit from the (expensive) validation of biomarkers, the private sector under-invests in biomarker and surrogate endpoint validation. This leads to countless lives lost and to trillions of public dollars spent on age-related conditions that could be prevented by better-aligned incentives. It should be an R&D priority for the new administration to fund the collection and validation of biomarkers and surrogate endpoints, then gain regulatory approval for them. As we explain below, the existing FNIH Biomarkers Consortium does not fill this role.
Currently, companies are understandably hesitant to invest in validation without clear rewards or regulatory pathways. The proposed framework would encourage private companies and laboratories to contribute their biomarker data to a shared repository. This repository would expedite regulatory approval, moving away from the current product-by-product assessment that discourages data sharing and collaboration. Establishing a broader pathway within the FDA for standardized biomarker approval would allow validated biomarkers to be recognized for use across multiple products, reducing the existing incentives to safeguard data while increasing the supply of validated biomarkers and surrogate endpoints. Importantly, this would accelerate the development of drugs which holistically extend the healthspan of aging populations in the U.S. by preventing instead of treating late-stage conditions. (Statins similarly helped prevent millions of heart attacks.)
Key players such as the FDA, NIH, ARPA-H, and BARDA should collaborate to establish a streamlined pathway for the collection and validation of biomarkers and surrogate endpoints, allowing these to be recognized for use across multiple products. This initiative aligns with the administration’s priorities of accelerating medical innovation and improving public health with the potential to add trillions of dollars in economic value by making treatments and preventatives available sooner. This memo outlines a framework applicable to various diseases and conditions, using biological aging as a case study where the validation of predictive and responsive biomarkers may be vital for significant breakthroughs. Other critical areas include Alzheimer’s disease and amyotrophic lateral sclerosis (ALS), where the lack of validated surrogate endpoints significantly hinders the development of life-saving and life-improving therapies. By addressing these bottlenecks, we can unlock new avenues for medical advancements that will profoundly improve public health and mitigate the fast-growing, nearly trillion-dollar Medicare spend on late-life conditions.
Challenge and Opportunity
By 2029, the United States will spend roughly $3 trillion dollars yearly – half its federal budget – on adults aged 65 and older. A good portion of these funds will go towards Medicare-related expenses that could be prevented. Yet the process of bringing preventative drugs to market is lengthy, costly, and currently lacking in commercial incentives. Even for therapeutics that target late-stage diseases, drug development often takes 10+ years and cost estimates range between $300 million to $2.8 billion. This extensive duration and expense are due, in part, to the reliance on traditional clinical endpoints, which require long-term observation and longitudinal data collection. The burden of chronic diseases is growing, and better biomarkers and surrogate endpoints are needed to accelerate the development of therapeutics that prevent non-communicable diseases and age-related decline. Chronological age, for instance, is a commonly used but inadequate surrogate marker for biological age. This means that, to date, clinical trials on therapeutics designed to improve the biology of aging take decades to validate, rather than years. As a result, pharmaceutical companies find more short-term rewards in treating late-stage diseases, since developing drugs that reduce overall age-related decline requires longer and currently uncertain endpoints.
The validation of reliable biomarkers and surrogate endpoints offers a promising solution to this challenge. Biological measures often correlate with and predict clinical outcomes, and can therefore provide early indications of whether a treatment is effective. If sufficiently predictive, biomarkers can serve as surrogate clinical endpoints, potentially reducing the duration and cost of clinical trials. Validated biomarkers must accurately predict clinical outcomes and be accepted by regulatory authorities, yet the validation process is underfunded due to insufficient commercial incentives for individual agents to share their biomarkers to be used as a public good. (From a purely financial standpoint, companies are better off targeting diseases with known endpoints.)
The most prominent existing efforts to advance biomarkers and surrogate endpoints are the Foundation for the National Institute of Health’s (FNIH) Biomarkers Consortium and the FDA’s Biomarker Qualification Program. Established in 2006, the Biomarkers Consortium is a public-private partnership aimed at advancing the development and use of biomarkers in medical research. Meanwhile, the FDA’s qualification program was the result of the 21st Century Cures Act, passed in 2016, which underscored the critical role biomarkers play in accelerating medical product development. The Act mandated the FDA to implement a more transparent and efficient process for biomarker qualification.
Despite the Consortium’s ambitious goals, the rate of biomarker qualification by the FDA has been slow. Since its inception in 2006, only a small number of biomarkers have been successfully qualified. This sluggish progress has been a source of criticism for stakeholders, especially given the high level of resources and collaboration involved. For example, the process of validating biomarkers for osteoarthritis under the Consortium’s “PROGRESS OA” project has been ongoing since Phase 1 and still faces hurdles before full qualification. We are of the view that this is the result of two issues. Firstly, the qualification process, which involves FDA approval, is seen as overly complex and time-consuming. Despite the 21st Century Cures Act aiming to streamline the process, resulting in the qualification pathway, it remains a significant challenge. The difficulty in navigating the regulatory landscape can limit the impact of Biomarkers Consortium (BC) projects. The Kidney Safety Project, for example, faced substantial regulatory hurdles before finally achieving the first qualification of a clinical safety biomarker. Secondly, even though the Consortium operates in a precompetitive space, there are ongoing challenges related to data sharing. Companies may still hesitate to share critical data that could advance biomarker validation out of concern for losing a competitive edge, which hampers collaboration. To address these issues, it is crucial to implement a framework that promotes data sharing in the academic and private sectors, providing strong incentives for the validation and regulatory approval of biomarkers, while improving regulatory certainty with a standardized regulatory process for surrogate endpoint validation.
The current boom in biotechnology underscores the urgency of addressing persisting inefficiencies. Without changes, we face a significant bottleneck in proving the efficacy of new drugs. This is exacerbated by Eroom’s Law—the observation that drug discovery is becoming slower and more expensive over time. This growing inefficiency threatens to hinder the development of new, life-saving treatments at a time when the American population is aging and rapid medical advancements are crucial to deter increasing medical and social costs. In just 11 years—between 2018 and 2029—the U.S. mandatory spending on Social Security and Medicare will more than double, from $1.3 trillion to $2.7 trillion per year. Yet the costly diseases of aging can be therapeutically targeted before they become late-stage conditions like Alzheimer’s. For federal policymakers, taking immediate action to improve data sharing and biomarker validation processes is vital. Failure to do so will not only stifle innovation but also delay the availability of critical therapies that could save countless lives and accelerate economic growth in the long run. Prompt policy intervention is essential to capitalize on the current advancements in biotechnology and ensure the development of new life-saving tests, tools, and drugs.
Implementing pull-incentives for data sharing now can help the United States adjust to its new demographic structure, where adults in advanced age prevail, while fertility rates decline. It can also mitigate the escalating costs and timelines of clinical trials, and accelerate the approval of life-saving, health-extending drugs. If our proposed framework is successfully implemented, a robust pool of biomarker data will be established, significantly facilitating the discovery and validation of biomarkers. This will result in several key advancements, including shortened clinical trial durations, increased R&D investment, faster drug approvals, and even increased drug efficacy. Additionally, new drug classes targeting non-disease endpoints, such as biological aging, could be developed. Just as the discovery of LDL as a surrogate marker of heart health was critical in enabling the testing and development of statins, the discovery of clinical-grade biomarkers may unlock new therapeutics designed to target the mechanisms that drive human aging, slowing down the progression of age-related diseases (like cancers) before they become deadly and socio-economically expensive.
Plan of Action
To address the challenge of inefficient data sharing, validation, and approval of biomarkers, we propose implementing a series of pull-incentives aimed at encouraging pharmaceutical companies to contribute their relevant biomarker data to a shared repository and undertake the necessary research and analysis for public validation. These validated biomarkers can then be formally accepted by regulators as surrogate endpoints for drug approval, accelerating the drug development process and reducing late-life costs.
Recommendation 1. An NIH-FDA initiative for Biomarkers and Surrogate Endpoints Within the NIA
Most existing agencies focus on single, often late-stage diseases. This is at odds with a holistic understanding of human biology. A new initiative within the National Institute on Aging (NIA) could be devoted to the discovery, collection, and validation of biomarkers and surrogate endpoints for overall human health and age-related decline. Most National Institutes of Health funds are currently devoted to the diseases of aging (think cancers, Alzheimer’s, heart disease, or Parkinson’s.) Within the NIA, research on Alzheimer’s disease alone receives roughly eight times more funding than the biology of aging, with few human-relevant results. Every federal agency and U.S. individual would benefit from better biomarkers of long-term health and from an understanding of how to measure the biology of aging. Yet no single agent has the incentives to collect and validate this data, for instance by shouldering the costs of validating predictive and responsive biomarkers of aging.
This new initiative could also be devoted to the development of preclinical, human-relevant methodologies that could broadly facilitate or streamline drug development. In 2022, the FDA Modernization Act 2.0 approved the use of in vitro and in silico New Approach Methodologies (NAMs) like cell-based assays (e.g. organs-on-chips) or computer models (like virtual cells) in preclinical development to reduce or replace animal studies, especially “where no pharmacologically relevant animal species exists.” This may be the case for human aging, where no single animal model reflects the full complex biology of our aging process.
At present, these technologies cannot accurately represent the multifactorial processes of aging, and they cannot model entire organisms. Much work remains to be done to even understand how to “code” aging into organs-on-chips. Yet if supplemented by approaches like in vivo pooled screening, next generations of human-relevant in vitro or in silico methodologies (like virtual cells) could be infused with the complex data needed to accelerate clinical trial results and increase drug efficacy. For in vitro and in silico models to reproduce key aspects of aging biology, a better understanding of how human aging works in living organisms — and what markers to include to represent it either virtually or in vitro — may be needed. Yet pharmaceutical companies, startups, health insurance firms, and even research hospitals again lack the incentives to shoulder the costs of collecting and validating this type of data. This means a new office within a federal agency may be needed to supply these incentives.
Recommendation 2. New Data-sharing Incentives
The specific incentives used would need to be developed in collaboration with policymakers and industry stakeholders, but a few are outlined below:
Pull-incentives
One possibility is offering transferable Priority Review Vouchers (PRVs) or similar pull incentives to companies that share their biomarker data. PRVs are currently awarded by the FDA to companies developing drugs for neglected tropical diseases, rare pediatric diseases, or medical countermeasures. A PRV allows the holder to expedite the FDA review of another drug from 10 months to 6 months, and holds significant financial value. Offering transferable PRVs for drugs designed to target biological aging, for instance, could create the incentives needed for pharmaceutical companies to target early-stage age-related conditions before they turn into diseases.
The creation of a new PRV category would require legislative action. Our proposed NIH-FDA initiative would be well positioned to oversee the issuance of PRVs, working with government agencies and think tanks to determine, for instance, what an “aging therapeutic” means, and what a company needs to achieve to gain a PRV for a longevity drug. The Alliance for Longevity Initiatives, for instance, has developed an advanced approval pathway for health-extending drugs that directly target the biology of aging. Another possible strategy would be for the FDA to encourage drugs that target multiple disease indications at once, perhaps offering discounts or incentives for every extra biomarker or surrogate endpoint validated. This could effectively encourage the development of drugs that do more than marginally improve on existing interventions.
We acknowledge that an overabundance of PRVs can saturate the market, decreasing their value and weakening the intended pull-incentive for pharmaceutical innovation. A response would be to demand that proposals to issue additional PRVs include a comprehensive market impact analysis to mitigate unintended economic consequences. Expanding the number of PRVs can also place extra demands on the FDA’s limited resources, potentially leading to longer approval times for other essential medications, even though PRV holders often delay redemption, preventing an immediate influx of priority review applications. The PRV system may inadvertently favor larger, well-established pharmaceutical companies that have the means to acquire and leverage PRVs effectively, creating barriers for smaller firms and startups. These are all spill-over problems worth solving for the potential upshot of mitigating late-life disease costs and encouraging drugs that holistically improve the human healthspan.
Biomarker Data Sharing as a Condition of Federal Funding
Federal funding recipients are legally obligated to make their research publicly accessible through agency-specific policies aimed at advancing open science. This mandate was strengthened by the 2022 OSTP Memorandum. Despite this clear mandate, the implementation of public access policies has been uneven across federal agencies, with progress varying due to differences in resources, technical infrastructure, and agency-specific priorities. The 2022 OSTP Public Access Memorandum aims to accelerate agency efforts to enhance public access infrastructure and policies. This updated guidance presents an opportunity for agencies to not only meet immediate data-sharing requirements but also to expand policy scopes to include essential clinical data, such as biomarker data from clinical trials. To meet these goals, agencies should ensure that funding agreements explicitly require the publication of comprehensive biomarker data and that suitable repositories are available to store and share these critical datasets effectively.
Case Study: Project NextGen
A prime example of the potential success of such initiatives is Project NextGen, a program led by BARDA in collaboration with the NIH to advance the next generation of COVID-19 vaccines and treatments. As part of its vaccine program, Project NextGen includes centralized immunogenicity assays with the overarching goal of establishing correlates of protection, which could serve as surrogate biomarkers for next-gen vaccines. These assays are collected during Phase 2b vaccine studies sponsored by Project NextGen, which have been designed to measure a number of secondary immunogenicity endpoints including systemic and mucosal immune responses. Developers share their assays so that they can be used as a public good, in return for federal funding. This effort demonstrates the feasibility and benefits of a federally led effort to share assay data to advance biomarker validation and drug development.
Recommendation 3. Create and Manage a Data Repository
To enhance collaborative research and ensure the efficient use of publicly funded clinical data, we recommend establishing a secure data repository. This repository will serve as a centralized platform for data submission, storage, and access. Management of the repository could be undertaken by a federal agency, such as the NIH, leveraging their experience with the Biomarkers Consortium, perhaps in partnership with non-governmental organizations like the Biomarkers of Aging Consortium. Drawing from existing models, such as Project NextGen’s assay data management, can provide valuable insights into the implementation and operationalization of the repository.
The cost of establishing and maintaining this repository, including data storage, management, and access controls, would be dwarfed by the socio-economic returns it could provide. This repository can facilitate data sharing, protect sensitive information, and promote a collaborative environment that accelerates biomarker validation and approval, while ensuring pharmaceutical companies that their hard-earned data is safely stored.
The securely stored data in the repository would primarily be accessible to qualified researchers, clinicians, and policymakers involved in biomarker research and development, including academic researchers, pharmaceutical companies, and public health agencies. Access would be granted through an application and review process. The benefits of this repository are multifaceted: it accelerates research by providing a centralized database, enhances collaboration among scientists and institutions, increases efficiency by reducing redundancy and improving data management, ensures data security through robust access controls, offers cost-effectiveness with long-term socio-economic returns, and supports regulatory bodies with comprehensive data sets for more informed decision-making.
Recommendation 4. Create A Regulatory Pathway with Broader Application
To accelerate the adoption of validated biomarkers and surrogate endpoints in drug development, we propose the creation of a streamlined regulatory approval process within the FDA. This new pathway would establish clear criteria and standardized procedures for biomarker evaluation and approval, facilitating their recognition for use across multiple products and therapeutic areas.
Currently, the FDA’s Center for Drug Evaluation and Research (CDER) operates the Biomarker Qualification Program (BQP), which allows drug developers to seek regulatory qualification for specific contexts of use. While this program fosters collaboration between the FDA and external stakeholders, biomarkers are qualified on a case-by-case basis, limiting their broader applicability across different drug development programs.
Additionally, the FDA maintains a Table of Surrogate Endpoints that have been used as the basis for drug approvals under the accelerated approval pathway. However, this table primarily serves as a reference and does not comprehensively address the need for a streamlined approval process for biomarkers and surrogate endpoints.
By developing a framework that moves away from traditional product-by-product assessments, the FDA could reduce existing barriers to biomarker and surrogate endpoint discovery and approval. This approach would encourage data sharing and collaboration among pharmaceutical companies and research institutions, leading to faster validation and broader acceptance of these critical tools in drug development.
This proposal builds upon existing legislative efforts, such as the 21st Century Cures Act of 2016, which includes provisions to accelerate medical product development and supports the use of biomarkers and surrogate endpoints in the regulatory process. Furthermore, it aligns with the FDA’s ongoing efforts to provide clarity on evidentiary criteria for biomarker qualification, as outlined in the 2018 guidance document “Biomarker Qualification: Evidentiary Framework.”
Inspiration for this approach can be drawn from the Advanced Approval Pathway for Longevity Medicines (AAPLM) proposed by the Alliance for Longevity Initiatives (See AAPLM-Whitepaper). The AAPLM includes provisions such as a special approval track, a priority review voucher system, and indication-by-indication patent term extensions, which align economic incentives with the transformative health improvements that longevity medicines can provide. These measures offer a valuable template for facilitating the recognition and approval of biomarkers. Adding to the existing FDA table of surrogate endpoints that can serve as the basis for drug approval or licensure, and referencing existing collaborations between the NIH and FDA, such as the Biomarkers Consortium, can provide a robust foundation for new biomarker evaluations. Ultimately, this regulatory innovation will support the development of life-saving drugs, enhance public health outcomes, and meaningfully contribute to economic growth by bringing effective treatments to market more quickly.
Conclusion
Today, over two thirds of all deaths in the United States are the result of an age-related condition. The burden of non-communicable diseases is growing, and better biomarkers and surrogate endpoints are needed to target diseases before they are life-threatening or debilitating. The next administration should implement a comprehensive framework to promote data sharing and incentivize the validation and regulatory approval of biomarkers and surrogate endpoints. This aligns directly with the administration’s goal to make Americans healthy. These solutions can substantially reduce the duration and cost of clinical trials, accelerate the development of life-saving drugs, and improve public health outcomes. It is possible and necessary to create an environment that encourages and rewards pharmaceutical companies to share crucial data that accelerates medical innovation. By discovering and validating predictive and responsive biomarkers of health and disease, new therapeutic classes can be developed to directly target biological aging and prevent most forms of cancers, heart disease, frailty, vulnerability to severe infection, and Alzheimer’s. This will enable the United States to remain at the forefront of medical research, and to respond to the growing demographic crisis of aging populations in declining health.
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.
A number of market failures stand in the way of the discovery and validation of predictive, reliable, and responsive biomarkers. First, it’s currently expensive to test drugs in multiple disease indications, which means pharmaceutical companies are often incentivized to focus on late-stage diseases (e.g. delaying death by a terminal cancer by three months), since this drug class is more easily and quickly trialed. The FDA also strongly assumes that a treatment ought to modulate a single outcome. (Think life/death; heart disease/no heart disease.) Therapeutics that target biological aging, for instance, would take decades to test without validated biomarkers or widely accepted surrogate endpoints.
Aging research, for instance, has seen a 70-fold increase in venture capital funding since the last decade. Yet so far—and this is a critical asterisk—misaligned commercial incentives have mostly optimized for unproven supplements, imprecise biological-age-tracking apps, and unsafe experimental therapies or cosmetics. The most well-meaning investors and founders in “longevity” often end up developing drugs for single disease indications (like osteoarthritis, or obesity) to avoid bankruptcy or as a path to self-fund their intent of developing drugs that more holistically target the mechanisms that drive aging. arket incentives need to be aligned to the pressing social needs these therapeutics could respond to.
The federal government is uniquely positioned to coordinate large-scale initiatives that require significant resources and regulatory oversight. While private sector companies play crucial roles in drug development, they often lack the incentives to self-coordinate and the authority to drive comprehensive data-sharing and biomarker validation efforts.
Cohesion from data-collection to regulatory approval of biomarkers is going to be key if surrogate endpoints are actually going to be adopted. Having the federal government oversee all stages will ensure this cohesion.
The Biomarkers Consortium has made meaningful strides in advancing biomarker research, but they have not succeeded in acquiring sufficient data. The consortium relies on voluntary, precompetitive collaboration without providing strong financial or legislative incentives for data sharing. It does not maintain a centralized, secure data repository, and struggles with fragmented data sharing. It also lacks influence over the FDA’s biomarker qualification process, which remains complex and time-consuming. This has resulted in slow progress due to hesitancy from private entities to share valuable data. Our solution differs by directly addressing this data-sharing hurdle through a series of targeted incentives that reduce the case-by-case assessment currently required, and enable broader application of validated biomarkers across multiple drugs and therapeutic areas.
By introducing legislative changes to authorize patent extensions and expand Priority Review Vouchers (PRVs), we create compelling reasons for companies to share their data. Additionally, our proposal includes the development of a centralized data repository with a streamlined regulatory approval process, inspired by the Advanced Approval Pathway for Longevity Medicines (AAPLM). This approach not only incentivizes data sharing but also provides a clear and efficient pathway for biomarker validation and regulatory acceptance. By leveraging existing frameworks and offering tangible rewards, our solution proposes an increase in incentives, to match the socioeconomic benefits that may be unlocked by more accessibility to the wealth of existing but undersupplied biomedical data.
The FDA’s Accelerated Approval Pathway is indeed a valuable tool that allows for the approval of drugs based on surrogate endpoints that are reasonably likely to predict clinical benefit. This pathway requires substantial evidence showing that these surrogate endpoints are linked to clinical outcomes, usually gathered from rigorous clinical trials. However, it typically applies to surrogate endpoints validated for specific uses or products. Our goal is to establish a new pathway that supports the validation and use of surrogate endpoints across multiple products. By validating biomarkers that can be used across various drugs, we can streamline the drug development process, reducing the time and cost associated with bringing new therapies to market. This broader approach would enhance efficiency, reduce drug development time and costs, and promote innovation by encouraging pharmaceutical companies to invest in research, knowing that successful biomarkers can have wide-reaching applications.
Pharmaceutical companies could push back on this proposal due to concerns over losing their competitive advantage by sharing proprietary data. They might reasonably fear that sharing valuable biomarker data could erode their market position and intellectual property. By involving pharmaceutical companies in the development of the proposal, we can better understand their concerns and tailor incentives accordingly. One effective strategy would be to offer significant financial incentives, such as Priority Review Vouchers (PRVs) or patent term extensions to companies that share their data. These incentives can offset the perceived risks and provide tangible benefits that make data sharing more attractive. By making PRVs transferable and offering additional incentives to small biotechnology companies, this policy can be implemented without overly favoring large pharmaceutical companies. Another possible strategy would be for the FDA to encourage drugs that target multiple disease indications at once, perhaps offering discounts or incentives for every extra biomarker or surrogate endpoint validated. Fostering a collaborative environment where the benefits of shared data (such as accelerated drug approvals and reduced R&D costs) are clearly communicated can reduce hurdles. Engaging economists to quantify the long-term economic gains to individual pharmaceutical companies as well as to society, while demonstrating how shared data can lead to industry-wide advancements, can further encourage participation. By providing competitive enough incentives, a framework can be created that balances the interests of pharmaceutical companies with the broader goal of advancing medical innovation and public health.
The first step to get this proposal off the ground is to introduce legislative changes that authorize patent extensions and expand the eligibility for Priority Review Vouchers (PRVs). These legislative changes will create the necessary incentives for pharmaceutical companies to participate in the program by offering tangible benefits that offset the risks associated with data sharing.
Simultaneously, developing and launching a pilot program for the centralized data repository is crucial. This pilot should focus on a specific subset of biomarkers for high-priority diseases and non-disease indications to demonstrate the feasibility and benefits of the proposed framework. By starting with a targeted approach, we can gather initial data, test the processes, and make any necessary adjustments before scaling up the program. This pilot will not only help in garnering support from stakeholders by showcasing the practical benefits of the framework but also refine the approach based on real-world feedback, ensuring a smoother and more effective broader implementation.
Similar efforts in the past have often been hindered by a lack of incentives for data sharing and collaboration, along with fragmented regulatory processes. Our proposal aims to overcome these obstacles by introducing strong incentives which will encourage companies to share their data. Moreover, we propose creating a standardized regulatory pathway for biomarker approval, which will streamline the process and reduce fragmentation. By involving key federal agencies, we ensure a coordinated and comprehensive implementation, thus avoiding the pitfalls that have doomed past efforts.
The status quo is unacceptable. Millions of lives are lost or debilitated every year due to the slow and costly process of bringing new drugs to market, which is hindered by the lack of validated biomarkers and surrogate endpoints. The recommended course of action leverages existing regulatory frameworks and incentives that have proven effective in other contexts, such as the use of Priority Review Vouchers (PRVs) for neglected tropical diseases. By adapting these mechanisms to encourage data sharing and biomarker validation, we can build on established successes while addressing the specific challenges of the current drug development landscape.
This approach ensures that we utilize proven strategies to accelerate drug development and approval, reducing the overall time and cost associated with clinical trials. By fostering a collaborative environment and providing tangible incentives, we can significantly enhance the efficiency and effectiveness of the drug development process. This targeted strategy not only addresses the immediate needs but also sets a foundation for continuous improvement and innovation in the field of medical research, ultimately saving lives and improving public health outcomes.
Small, fast grant programs are vital to supporting transformative research. By adopting a more flexible, decentralized model, we can significantly enhance their impact.
New solutions are needed to target diseases before they are life-threatening or debilitating, moving from retroactive sick-care towards preventative healthcare.
To improve program outcomes, federal evaluation officers should conduct “unmet desire surveys” to advance federal learning agendas and built agency buy-in.
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.