6

Recommendations

Therapeutic innovation in the United States has powered medical innovation around the world, but despite significant public and private investment, unmet medical needs remain. To better understand what drives mismatches among investment, disease burden, and unmet need and to inform their deliberations, the committee gathered and synthesized information from the literature and public testimony. This information is summarized in the preceding chapters, which analyze patterns of mismatch among investment, burden, and unmet need (Chapter 3); explore reasons driving this mismatch (Chapter 4); and discuss policy strategies to improve alignment (Chapter 5). Figure 6-1 outlines the committee’s conceptual approach to their charge, including identifying misalignments and the barriers to achieving alignment, employing levers of change to address misalignments, and the intermediate- and long-term outcomes that can be achieved.

This chapter presents the committee’s specific recommendations to better align investments in therapeutic development with disease burden and unmet need. Recognizing the complex, multisector landscape of therapeutic development, the committee identified several key areas for strategic action: public health data collation and availability, public investment in biomedical research, public–private partnerships, the regulatory environment, and payment and reimbursement policy incentives. The committee’s recommendations are thus organized around five goals:

1. Design a state-of-the-art publicly accessible system to assess and track unmet need associated with U.S. disease burden, with a critical focus on identifying areas of mismatch and reducing health disparities.

2. Support and strengthen public investment in innovative therapeutics that address unmet need.
3. Strengthen public–private partnerships to encourage the sharing of information and technology transfer to facilitate addressing unmet need.
4. Strengthen a regulatory environment that supports innovation to address unmet need.
5. Strengthen a fiscal and policy environment to align reimbursement policy with evidence-based therapeutic value and the extent to which products address unmet need.

In the rest of this chapter, each goal is discussed in turn, with recommendations made to a variety of key policy makers and decision makers in the therapeutic development landscape. The committee emphasizes specific, high-level actions needed to drive change by aligning incentives across multiple sectors that all play a role in drug development. Addressing each goal alone can mitigate a mismatch between investment in therapeutic development with unmet need, but when implemented in concert, these recommended actions will reinforce one another to drive meaningful change and bring to market effective therapies that address critical unmet needs.

GOAL 1: DESIGN A STATE-OF-THE-ART PUBLICLY ACCESSIBLE SYSTEM TO ASSESS AND TRACK UNMET NEED ASSOCIATED WITH U.S. DISEASE BURDEN, WITH A CRITICAL FOCUS ON IDENTIFYING AREAS OF MISMATCH AND REDUCING HEALTH DISPARITIES

Biomedical research is critical for improving the health of individuals worldwide. For millions of patients and their families, biomedical research provides hope for those dealing with life-threatening and chronic diseases. Although research and innovation is not a zero-sum game, the resources available are unable to address all current health and research needs in a timely manner. Therefore, it is critical that there is a system in place that makes it possible to set research priorities and make difficult decisions about what funding is available to address health needs. As discussed in Chapter 3, these decisions are difficult and involve value judgments for prioritizing. While these value judgments are political decisions that may shift with changes in administrations or leadership in organizations, there is currently a lack of data with which leaders can make evidence-based policy judgments. Therefore, without the data necessary to assess how investments in therapeutic development align with disease burden and unmet need, research prioritization cannot be done accurately or effectively.

While various data sources provide insight into disease burden through common metrics like incidence, prevalence, and disability-adjusted life-years (DALYs), this information is not systematically compiled, nor is it regularly updated, analyzed, and used for the purpose of prioritizing needs. Having a way to systematically collect these data would further improve efficiency and effectiveness in decision making, as it reduces the number of demands on federal agencies and institutes to share how research priorities are set. Moreover, disease burden is multidimensional (see Chapter 2), suggesting the need for a more comprehensive and coordinated characterization of burden and need. This information gap makes it difficult to optimize investment in research. Without these data, public research funds are being allocated each year with insufficient information about the extent to which the investment is aligned with health needs.

A publicly accessible, centralized system to aggregate relevant data and track unmet medical need associated with disease burden would enable more strategic investment of resources and would help policy makers and public and private funding groups better align innovation and investment with health needs. This system could be used to identify disease areas to prioritize investment. These data could highlight areas of mismatch between investment and disease burden—including both conditions that need greater attention and those where there is overinvestment compared with relative burden. Existing models that could inform the development of such a system are the Institute for Health Metrics and Evaluation (IHME) Global Burden of Disease (GBD) Compare tool and Impact Global Health G-Finder project, both of which host the types of data that would need to be incorporated into the centralized system (IHME, 2025; Impact Global Health, 2024).

Developing standardized categories of disease and therapeutic areas for use in the system would help to enable comparisons between disease burden and investments in order to identify areas of mismatch. Similarly, methods that promote data transparency and accessibility, such as open methodologies, will facilitate a more comprehensive analysis of disease burden and unmet need. Because there will be several areas of mismatch that differ along a variety of relevant factors, such as the age of the affected population, disease acuity versus chronicity, and quality-of-life impact, the committee recognizes that it would be impossible to generate a single, universal list of priorities, as discussed in depth in Chapter 3. Instead, providing accessible data on burden, need, and current measures of public and private investment would enable public and private funders to make informed decisions based on their priorities and values. For example, some may prioritize rare, low-prevalence diseases with high individual burden, while others prioritize diseases with high population burden or high health disparities.

Developing this system is challenging. For instance, both burden and unmet need are difficult to define (see the committee’s selected definitions

in Chapter 2) and must be carefully considered in designing this system. Similarly, current investments are difficult to track, especially in the private sector. In addition, while some data are readily available and could be aggregated for this purpose, the committee recognizes that not all the data described in the recommendation are currently or easily accessible. To advance public health and reduce health disparities, these data should be collected, and it is important to invest in doing so.

Finally, it is important that these data be made public to the extent possible. There has been recent reporting about critical datasets and web pages being pulled from federal health websites, some of which is back online but with key data missing; such missing information damages scientists’ ability to advance their research (Stone and Huang, 2025). Furthermore, given that these data are collected using public resources and do not contain proprietary information, it is critical that they remain accessible to the public. Therefore, recognizing that some information on private-sector investments may need to be aggregated to avoid sharing proprietary information, it is critical that the information collected on evaluating disease burden, unmet need, and the areas of investment be publicly available for all to use.

Finding 3-2: There are some existing data on disease burden, unmet need, and investment; however, these data are not regularly compiled and synthesized for assessing mismatch across factors. In addition, existing data are sometimes insufficient and have significant gaps. As a result, this committee lacked the data needed to produce a report that evaluates all aspects of disease burden, unmet need, and investment to fully assess the mismatch.

Finding 6-1: Disease burden and unmet need are multidimensional and challenging to measure, which suggests the need for multiple measures to effectively assess alignment or mismatch among these factors. A data repository that compiles multiple metrics of disease burden and unmet need would enable different organizations to access relevant data and create lists that reflect their priorities and values. For example, prioritizing reducing disparities across populations would require information on individual burden or data aggregated by relevant sociodemographic groups, not only population burden.

Finding 3-1: Characterizing past, current, and future priorities for investments in therapeutic innovation is multidimensional and complicated, especially given the longitudinal nature of research and development. Many of the data needed for this purpose are not publicly available. Some high-level data on industry investment are available, but the granularity of this information is limited.

Conclusion 3-1: More comprehensive, specific, timely, and accurate data on disease burden, unmet need, and innovation, as well as improved data aggregation, are essential for private and public funders to systematically use measures of disease burden and unmet need when making decisions about funding priorities.

Conclusion 3-2: Collecting and aggregating these data requires ongoing stewardship to most effectively address unmet clinical need and reduce health disparities.

Conclusion 3-3: The U.S. government has a responsibility to ensure that timely data on public investment and population health data be made publicly available to support research and strategic investment in areas of unmet need.

Recommendation 1: Congress should establish and fund an interagency consortium charged with tracking and assessing unmet therapeutic need associated with U.S. disease burden and current investments in innovation, with a critical focus on identifying areas of mismatch and reducing health disparities. The consortium should be led by a relevant unit of the Department of Health and Human Services (HHS) as determined by the Secretary of HHS.

This consortium should be charged with the following:

1. Generate a publicly accessible data repository on disease burden, therapeutic investment, and unmet needs that is updated on a triennial basis and used to generate derivative reports.
2. Produce a triennial report to Congress on the status of U.S. disease burden, extent of unmet need, and areas in which additional data are needed to reliably assess burden and unmet need. This report should collate, at minimum, for each disease area the current and projected incidence, prevalence, mortality, and disability-adjusted life-years.
3. Produce a companion assessment of the most reliable current estimates of investments from the public and private sectors for each therapeutic area, including public-sector funding by type and amount; private-sector investments; stage of the development pipeline for emerging treatments (e.g., drug discovery, preclinical research, clinical trials, regulatory review and approval, postapproval surveillance); the number, phase, and status of clinical trials; and sources of funding.
4. Identify areas in which additional research is needed to provide any missing information for each item above and recommending ways, such as statutory requirements or surveys, by which the data could be gathered for subsequent reports.

In addition to the data listed above, the triennial report to Congress would ideally contain additional information for each therapeutic area. For example, the report should include multiple additional measures of population burden and unmet need, such as years of life lost, equal value of life-years gained, and patient-reported outcomes. It should also include an assessment of individual burden, incorporating both patient and caregiver perspectives. The report should also list populations affected by age and at-risk subgroups that are disproportionately burdened, and current level of residual unmet need based on available treatments. The committee suggests categorizing unmet need by (a) no treatment exists, (b) treatment exists but is limited in its ability to address disease burden, overall or for specific at-risk subgroups of the population; (c) effective treatment exists, but access is limited: and (d) effective treatment exists and is accessible for all in need (i.e., no unmet need).

To implement this recommendation most effectively, it is critical that this consortium involve cross-agency and cross-disciplinary collaborators. Ideally, the following federal agencies and offices should be represented:¹ the Food and Drug Administration (FDA), National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), the Office of the Assistant Secretary for Planning and Evaluation (ASPE), Office of the Assistant Secretary for Health, Assistant Secretary for Technology Policy, Advanced Research Projects Agency for Health (ARPA-H), Agency for Healthcare Research and Quality, Health Resources Services Administration, Indian Health Services, Centers for Medicare and Medicaid Services (CMS), the Department of Veterans Affairs, and the Department of Defense. ASPE, or another relevant office, may be a good fit to lead the organization of the consortium. In addition, the consortium should engage other key partners, including the Patient-Centered Outcomes Research Institute (PCORI) as well as industry organizations such as Pharmaceutical Research and Manufacturers of America (PhRMA) and Biotechnology Innovation Organization (BIO), and establish mechanisms to incorporate patient input, such as public comment periods or public meetings.

It is important for this consortium not only to collate these data but also to analyze and synthesize data, including identifying gaps and approaches for addressing these gaps. As an example, information about private-sector investment is not necessarily readily available. Although FDA requires that sponsors register trials in ClinicalTrials.gov, which may provide some insights into industrial areas of investment, sponsors are not required to

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¹ At the time of publishing, the HHS agencies and offices listed here are still in operation. However, the committee recognizes that some of these agencies and offices may be part of an ongoing reorganization within HHS. Therefore, any similar office or agency that replaces the duties of the agencies or offices outlined here should be included in the cross-agency effort.

report phase I trials or small feasibility studies. Furthermore, there are often data missing, and researchers and investigators alike report difficulties using ClinicalTrials.gov (Chaturvedi et al., 2019; CTTI, 2024). ClinicalTrials.gov also does not provide insights into investment dollars that are spent on certain therapeutic areas.

Given the challenge of collecting information on private industry investments, the committee recognizes that companies may need to be incentivized to share or disclose some of these data that are not now publicly available. One way of collecting this information could be through a survey of pharmaceutical and biotech companies, which, while imperfect, could gather a useful sampling of data to gain insight into private investment in therapeutic development. An approach for incentivizing the collection of these data would be to delay public access for a period of time (e.g., 6 months) and allow early access to those companies that have contributed data. These data would provide a valuable, precompetitive resource that companies could use to make decisions and justifications for their intended actions. Some organizations, including the American Association for the Advancement of Science, IQVIA, and LEK Consulting, already report on private investment based on survey data. Similarly, data collected by the Association of University Technology Managers (AUTM) could be used to track investment from university venture funds. These data could be incorporated in the tracking system and reported in an aggregated and anonymized format and would still prove useful for understanding the landscape of private investment.

Although the consortium would ideally be housed within the federal government, the consortium might also exist with a trusted entity outside of the federal government. For example, the Foundation for the NIH (FNIH), an independent nonprofit organization, was recently contracted to be a trusted data broker between the private and public sector, on behalf of NIH, to develop a data access platform to implement the NIH’s Data Counts program (TAGGS, 2024). If successful, this could potentially be a model to implement for this consortium.

Another challenge, which is nuanced, is assessing disease burden. As noted above, population burden is multifaceted. Ideally, a system for assessing disease burden should include multiple metrics to enable insight into different aspects of burden; however, this information is often incomplete or unavailable. Moreover, population burden alone does not provide a complete picture of disease burden; individual patient and caregiver burden offers an important counterpart. In some cases, such as in rare diseases, a condition may have low prevalence but high individual burden and high unmet need. Efforts to assess and measure individual (and caregiver) burden should incorporate patient perspectives without overburdening patients and communities.

There are also challenges in considering how new technologies could potentially shift how we think about disease burden. Previously, disease burden was thought about as burden that is avertable and burden that is inevitable. However, new technologies, like cell and gene therapies and other platform technologies, have made some inevitable burdens into avertable disease burdens. Therefore, although these considerations are not part of disease burden, it is important that some funding be considered for larger platform technologies that can shift innovation in substantial ways.

GOAL 2: SUPPORT AND STRENGTHEN INVESTMENTS IN INNOVATIVE THERAPEUTICS THAT ADDRESS UNMET NEED

The establishment of a comprehensive system to track and assess disease burden and unmet need, as recommended in Goal 1, should directly inform public and private investment in medical research and development. Congress plays a key role in defining the parameters of public investment in medical research by setting appropriations among the institutes and centers of NIH. Information about burden and unmet need should be incorporated at key points of decision making and should be an important resource for determining congressional appropriations and funding decisions at NIH and at other federal agencies. Private funders, including philanthropic organizations, should also consider these factors when making decisions about their funding priorities. While some private funders, such as foundations, may have a particular focus or goals, these factors can still be useful in guiding investment decisions within their areas that overlap with their mission.

However, considering burden and unmet need should not be limited to high-level priority setting. It should be integrated throughout the research funding process, from program development to grant review criteria, ensuring that unmet need is considered alongside scientific merit in such grant mechanisms. For example, ARPA-H could consider developing new programs specifically focused on the areas of highest mismatch between unmet need and investment. FDA could work with other agencies to advance development and validation of biomarkers. NIH could also do more to integrate these principles in its Small Business Innovation Research and Small Business Technology Transfer (SBIR/STTR) programs by prioritizing higher-risk, potentially revolutionary ventures that could address unmet needs and prioritize therapeutic development sources that are not covered by other investment sources. Furthermore, recognizing that NIH staff, such as the Council of Councils, is closer to understanding how unmet needs could be met with research funding, there should be some funding that they have discretion to allocate, with consideration for disease burden and unmet need. This could include Congress appropriating more funding with flexibility to be directed toward areas of highest unmet need and underinvestment, such

as through the NIH Common Fund. This holistic approach would help align incentives to encourage investigators to consider unmet need early in the research process.

When funding agencies elevate the goal of addressing unmet need as a priority, researchers are more likely to develop studies to target these areas, leading to better alignment of public investment in medical research with public health needs.

Recommendation 2: Funders of biomedical research should consider disease burden and unmet need when setting research priorities and directing funding. Specific actions to ensure both population health needs and scientific merit are considered in grant funding mechanisms include:

1. Congress should consider disease burden and unmet needs when setting appropriations to agencies that fund biomedical research, including in allocating funding among National Institutes of Health institutes and centers.
2. Public and private funders should develop targeted research funding opportunities specific to diseases with the highest mismatch of burden and unmet need, including funding opportunities for innovative methods to enable the development of therapeutic products and new biomarkers for diagnostic test development in these areas.
3. Public and private funders should allocate funds for the development and validation of new biomarkers and surrogate endpoints for diseases with high unmet medical need.
4. Public and private funders should provide funding for studies of disease epidemiology or basic science for areas where there is a critical need for understanding the mechanisms of disease.
5. Public and private funders should include explicit criteria that include, but are not limited to, unmet need and disease burden for evaluating proposals in the grant review process and funding decisions.

The committee recognizes that NIH recently simplified peer-review criteria, and the “Importance of the Research” factor includes significance and innovation (NIH, 2024). However, incorporating unmet need and disease burden into reviewers’ scoring can be achieved without major revision or complication. For example, NIH guidance on defining significance could be revised to make clear it includes social significance in addition to scientific significance. Alternatively, a separate high-level heading could be added to “Importance of the Research” to focus on social significance, such as unmet need and disease burden.

GOAL 3: STRENGTHEN PUBLIC–PRIVATE PARTNERSHIPS TO ENCOURAGE THE SHARING OF INFORMATION AND TECHNOLOGY TRANSFER TO FACILITATE ADDRESSING UNMET NEED

Goal 2 focused on the importance of public-sector actions to address disease burden and unmet need in funding decisions, but it is also important that the private sector takes these factors into account when developing a therapeutic portfolio. Generally, industry is constantly assessing unmet need to develop products that are most likely to generate returns on capital. However, as reviewed in Chapter 4, there are therapeutic areas where there is not a strong business case for developing an asset, or where an asset has been shelved for reasons unrelated to therapeutic potential. One way to develop these shelved assets that may address an unmet need is to develop and expand public–private partnerships (PPPs).

As discussed in Chapter 5, the committee defines PPPs as any type of cost or risk sharing between the public sector (both government and academia) and the private sector (private and public sources of capital, along with small and large industry). This includes bilateral government funding and legal mechanisms, such as cooperative research and development agreements (CRADAs), as well as larger multistakeholder projects between government, industry, academia, and nonprofits, such as those conducted by the congressionally authorized nonprofit organizations paired with the government scientific agencies.

Many strong global examples of PPPs are working to incentivize investment in areas of high disease burden and unmet need, including the Innovative Health Initiative in the European Union and the Global Health Innovative Technology Fund. The United States also has a number of PPP programs, described in detail in Chapter 5, such as Accelerating Medicines Partnerships (AMP), the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT), the National Cancer Institute Experimental Therapeutics (NExT) program, and Bridging Interventional Development Gaps (BrIDGs), that provide nondilutive funding for preclinical and clinical development. These programs are typically not focused on developing therapeutics that will benefit only one company or organization with its return on investment. However, they can be particularly useful for innovation in areas where cost and risk sharing are necessary, such as for novel drug target discovery, biomarker test development, or preclinical development models.

PPPs can be better used to address the misalignment for therapeutic areas where cost-sharing mechanisms can help address important unmet needs that are currently not being met owing to market disincentives. The committee is particularly interested in the potential for PPPs to fill three

areas of unmet needs: (1) expanding the development of better diagnostics, which, as discussed in Chapter 4, is an area that is difficult to get funded by private-sector investment because of payment structures for diagnostic tests; (2) limiting situations where therapies that are effective and meeting an unmet need are taken off the market or where assets demonstrating early signs of efficacy are shelved before making it to market; and (3) providing an avenue for the development of drugs for diseases where there exists no economic incentive for the private sector to develop therapeutics.

As discussed in Chapter 4, diagnostics can efficiently and precisely identify the patients most likely to benefit from therapy, leading to more approvals of drugs that are safer and more effective. However, the payment structure for diagnostics is complicated and there are limited incentives for companies to develop diagnostics, unless they are linked directly to a therapeutic, such as with companion diagnostics. Furthermore, FDA approval for diagnostic tests require both analytical and clinical validation, and CMS may consider evidence of improved outcomes, or clinical utility, when the test is used, in determining coverage and reimbursement of the diagnostic. Such outcome studies can be lengthy and thus costly. Therefore, despite the potential for diagnostics to address unmet needs and to facilitate accurate diagnosis for drug development for unmet health needs, incentives to innovate in this space are lacking. As a result, the committee believes that PPPs could be one way to develop better diagnostics, such as for some elements of generating evidence supportive of patient access. As noted in Chapter 5, PPPs are often most effective when focusing on the precompetitive space, such as developing better biomarkers. Therefore, this could be a useful area for PPPs to focus and drive innovation for unmet needs.

PPPs could also be particularly useful in keeping effective therapeutics meeting an unmet need on the market when there is a threat of them being pulled. For example, Novo Nordisk recently discontinued a long-acting insulin, Levemir, in part because of market forces (Alltucker, 2024). In a congressional hearing focused on the decision to pull Levemir from the market, the CEO of Novo Nordisk said the company would be willing to share the drug’s formulation with other manufacturers, including the U.S. government. As discussed in Chapter 5, there are many private businesses that work to obtain abandoned assets from other companies to develop. The committee is not proposing that PPPs could replace these industries, but they would fill in gaps where either the financing model is not favorable because of risk or the market dynamics do not make the asset desirable. By using nonprofit organizations for the federal agencies, such as the Foundation for the NIH and the Reagan-Udall Foundation, federal health agencies could work to continue manufacturing therapeutics in such situations so patients gain access to effective therapeutics for the public good, even when there is not a profitable market for industry.

Finally, PPPs could be useful for developing drugs in therapeutic areas where little to no economic incentives exist for the private sector. Chapter 4 outlines several reasons why market forces prevent innovation in areas of unmet need, including small patient sizes and limited applicability of therapeutics. PPPs could be used to provide creative sources of financing to develop therapeutics in these areas and thus advance innovation where potential profits are limited.

Finding 5-11: PPPs can effectively enable the private sector to share both costs and risks.

Finding 5-3: Addressing unmet clinical need sometimes requires novel diagnostic tests to characterize diseases and disease states. For example, novel drugs often depend on accurate diagnostics to identify who should receive the treatment.

Finding 4-2: Despite early signs of efficacy or even FDA approval of a drug, some therapeutics are shelved or pulled from the market because there is not a large enough economic incentive or return on investment for a company to fully develop the drug or continue manufacturing it once approved.

Finding 5-12: Every scientific health research agency has a congressionally mandated nonprofit organization that could be useful in building PPPs.

Conclusion 5-1: Diagnostics to characterize and detect disease states are critical for developing innovative therapies, targeting therapeutics to those who will benefit, and ensuring that patients have access to therapeutics early enough in their disease progression for therapeutics to be effective, and they are an important component of addressing unmet clinical need. Further incentives for development of innovative and accurate diagnostic tests that are necessary for drugs that address unmet medical needs could help resolve the mismatch among therapeutic investment, disease burden, and unmet need.

Conclusion 5-9: Strengthening and expanding public–private partnerships, such as the Network for Excellence in Neuroscience Clinical Trials, the National Cancer Institute Experimental Therapeutics program, and Bridging Interventional Development Gaps, could help address innovation challenges for therapeutic areas with unmet needs.

Recommendation 3: U.S. federal scientific agencies with congressionally authorized nonprofit organizations, such as the Foundation for the National Institutes of Health, Centers for Disease Control and Prevention Foundation, Reagan-Udall Foundation, and Henry M. Jackson Foundation for the Advancement of Military Medicine, should increase use of their nonprofits in order to focus on building public–private partnerships in areas of mismatch between unmet need (encompassing both therapeutics and diagnostics) and innovation.

Despite the many successes of PPPs, as discussed in Chapter 5, federal agencies do not always have insight into what shelved assets exist in pharmaceutical companies and therefore are unable to approach companies about developing these assets. Therefore, having an independent organization house a searchable repository of assets to which companies voluntarily submit information could help overcome this barrier. The committee recognizes that some companies may not be willing to share this information. However, it could be used by other industries looking to purchase a shelved asset, which may bring profits to industries looking to offload assets.

There may also be concerns about liability in selling an asset to a PPP or to another company. While outside the scope of this committee, it may be worth further attention to mitigate potential liability either through indemnification or other statutory measures. This repository might not be publicly available in order to safeguard proprietary information, but it would be viewable by federal agencies, other foundations, companies, and nonprofits that apply for access. Although it likely would not contain a complete list of shelved assets, it would be a start for advancing PPPs in areas of unmet need and where the market does not support further innovation.

The New Therapeutic Uses program from the National Center for Advancing Translational Sciences (NCATS) aims to accomplish some of this by focusing on drug repurposing. However, the New Therapeutic Uses program focuses on repurposing already approved drugs for other conditions, rather than assets under development. Furthermore, it does not appear that awards have been granted through this program since 2021, indicating the need for additional resources toward this goal.

Although created for different purposes, the National Cancer Institute (NCI) Formulary is a PPP between NCI and pharmaceutical and biotechnology companies where the companies volunteer assets that have shown promise in preclinical testing to be tested by NCI-funded investigators. Although the purpose of this specific PPP is to expedite investigator access to therapeutics in clinical trials and not necessarily to host a list of searchable assets, the success of the NCI Formulary could lay the groundwork for a more generalized searchable repository.

Recommendation 4: The National Institutes of Health should work with a neutral third-party entity to set up a searchable repository of assets no longer under development by commercial sponsors to be shared with foundations and other entities to take forward for testing. The information in the repository could be voluntarily provided by companies potentially looking to enter public–private partnerships to develop an asset.

Another barrier to successful PPPs, as discussed in Chapter 5, is the absence of a “warm” clinical trial infrastructure that is ready to go. Often, clinical trial networks are set up, only to be disbanded when a specific project ends because funding does not exist to sustain the staffing or infrastructure. Currently, industry does not typically use these public networks (outside of NCI) because many of the contractual and intellectual property elements are not viewed as favorable by the private sector. For example, one area of friction between industry and academia in the precompetitive space arises over intellectual property (Perkmann et al., 2011). This can be seen in one form in partnerships where academics are using company assets. Academic researchers are accustomed to freely publishing results, and academic research institutions have their own appetite for entrepreneurship. However, industry is often concerned with intellectual property (IP) protections and regulatory filings and are not eager to share data or results on the timelines desired by academics. This is especially true for highly novel assets being tested clinically.

The clinical trial infrastructure set up by NIH institutes involves academic researchers and institutions in addition to government. This can cause even more complex disagreements over IP between government, company, and academic institution policies being in conflict and creating a barrier to setting up effective PPPs due to the necessary complex negotiations. Even the simplest bilateral partnerships between NIH and industry can encounter difficulties. Currently, each NIH institute has its own rules for developing CRADAs for these PPPs. Some institutes, such as NCI, are more sympathetic to IP concerns, while other institutes require more data sharing. This lack of standardized contractual frameworks across NIH often results in confusion about entering into CRADAs and difficulties in negotiating the CRADAs across institutes. Therefore, having an established set of contractual and IP frameworks across NIH that protects patients’ interest in access to the resulting products could potentially expedite PPPs. The committee recognizes that there may be circumstances where different frameworks are desired, such as during a public health emergency, so the committee recommends a set of frameworks that accounts for these scenarios.

Recommendation 5: The director of the National Institutes of Health (NIH) should establish a set of contractual and intellectual property frameworks for industry allowing greater incentivization and smoother use of the NIH clinical trials infrastructure that balances the interests of NIH, NIH-funded investigators and their institutions, patients, and industry partners.

GOAL 4: STRENGTHEN A REGULATORY ENVIRONMENT THAT SUPPORTS INNOVATION TO ADDRESS UNMET NEED

Chapter 4 of this report details the challenges that lead to a misalignment of innovations with disease burden and unmet need. One of the challenges causing a misalignment is a lack of understanding of the underlying pathophysiology of many diseases. Without an understanding of how diseases work and how therapeutics could potentially target those diseases, innovation is unlikely to advance. As the committee heard throughout its public sessions, life science investors and industry scientists depend on advances in basic and preclinical biomedical research to attract their attention and funding. And, as described in Chapter 5, too often the problem facing patients is not that FDA is standing in the way of good drugs, but that there are not enough good drugs moving their way through the research and development process. Therefore, advancing basic and preclinical research in disease areas with a high disease burden and high unmet needs would improve innovation and reduce therapeutic gaps.

Finding 4-1: Investment in basic and preclinical biomedical research is essential to driving innovation in disease areas with significant disease burden that have unmet needs.

In the committee’s analysis of reasons for misalignment between investment in therapeutic development and unmet need, it did not find that the misalignment was caused by action or inaction from FDA. Although FDA sometimes is cited as a barrier to innovation, the committee did not find evidence to support this claim.

Finding 5-2: Many of the current drivers contributing to the misalignment of innovation and investment with disease burden and unmet need are not within FDA’s immediate control.

Furthermore, as outlined in Chapter 5, FDA has several ongoing programs to drive and support innovation in much-needed areas. These programs generally fall into three categories: (1) efforts to encourage investment to address unmet need; (2) efforts to address broad scientific

challenges, improve coordination, and encourage data generation to address unmet need; and (3) efforts to facilitate and expedite development, review, and approval to address unmet need.

Chapter 5 reviews a number of FDA programs designed to encourage investment in certain disease areas. Although there are differing opinions on the efficacy of some of these programs, particularly for priority review vouchers, there was not sufficient evidence to support expanding these programs to address unmet needs.

Finding 5-4: Whether or not the Orphan Drug Act (ODA) is narrowed or otherwise adjusted, there is no convincing evidence to suggest that the recent growth in rare disease innovation is a result of ODA incentives.

Finding 5-5: Without taking a position on whether priority review voucher programs should be renewed, the committee finds no convincing evidence to suggest that they should be expanded as a tool to better match investment and unmet need.

Conclusion 5-2: The committee did not find sufficient evidence to recommend expansion of priority review vouchers, orphan drug exclusivity, or patent term extensions in their current form to support drug development to better meet unmet needs.

As for the second category of FDA programs that address broad scientific challenges, improve coordination, and encourage data generation to address unmet needs, many of these programs are pilot programs or have only been in place for a short time. Therefore, there is not yet sufficient evidence to recommend expansion of these programs. However, the committee strongly supports the ideas behind these programs, many of which are intended to improve science and expedite innovation without lowering safety or efficacy standards.

Finding 5-6: Because FDA programs to address broad scientific challenges, with the exception of pediatric study requirements, have only been in place for a few years, it will be important to carefully evaluate their effect and resource burdens, both individually and collectively, and to devote additional resources to successful programs.

Finding 5-9: Through a wide range of programs, FDA has demonstrated creativity and commitment to supporting the science necessary for successful drug development and to providing regulatory support for sponsors seeking to address unmet need, especially in the context of rare disease.

Conclusion 5-3: Additional resources are needed for FDA programs that successfully support endpoint development and validation, innovative trial design, and the resolution of other broad scientific challenges that impede drug development for unmet needs, as well as programs designed to support communication between sponsors and FDA to quickly resolve challenges arising in specific development programs.

The last category of FDA programs is intended to facilitate and expedite the development and review of new therapeutics to address unmet needs, including through heightened regulatory support, shortened review periods, and alternative approaches to demonstrating effectiveness, thereby encouraging industry investment in these areas.

Finding 5-10: FDA has several programs designed to drive innovation in therapeutic areas of unmet need, including the breakthrough therapy and fast track designations, priority review, and accelerated approval, as well as less formal approaches to regulatory flexibility.

The most controversial of these programs has been the accelerated approval pathway, which accepts uncertainty regarding a drug’s clinical benefit in exchange for early approval, with the expectation that benefit will be established through postapproval confirmatory studies. In response to a number of concerns about confirmatory study delay, one of the goals of the 2022 Food and Drug Omnibus Reform Act (FDORA) was to improve the timely completion of confirmatory studies following accelerated approval. FDORA affirms FDA’s discretionary authority to require confirmatory studies be underway before approval is granted (FDA, 2025), which has been demonstrated to improve timely completion and conversion to regular approval (Fashoyin-Aje et al., 2022), though timely studies may not be feasible in extreme cases, such as ultrarare diseases. FDORA also created a requirement that sponsors submit more frequent progress reports on confirmatory studies (now every 6 months instead of annually) and requires that these reports be published on the agency’s website. Because these programs were recently enacted, it is premature to evaluate success, although they are strong steps toward promoting timeliness, especially given known challenges that make recruitment and retention difficult for confirmatory studies once patients can access approved drugs on the market.

In addition to timely completion of confirmatory studies following accelerated approval, it is also important to ensure that accelerated approval is granted only when drugs truly are reasonably likely to demonstrate clinical benefit (i.e., based on appropriate surrogate endpoints), that confirmatory studies are rigorously designed in addition to being completed on time, and that drugs whose studies fail to clearly confirm clinical benefit

are quickly withdrawn. Yet, evidence indicates concern in each of these domains: FDA has sometimes accepted controversial surrogate endpoints to support accelerated approval (Largent et al., 2022; McIntyre, 2024); confirmatory studies often lack important features of rigorous design, such as being conducted without randomization or a control group or continuing to rely on surrogate endpoints (Gyawali et al., 2019; Naci et al., 2017; Sachs et al., 2022); and drugs are sometimes converted to regular approval even when they fail to confirm benefit (Gyawali et al., 2021).

Although FDORA included provisions to require that key confirmatory study design parameters be established no later than the point of accelerated approval and to potentially expedite withdrawal proceedings when initiated by FDA, neither the law nor subsequent FDA guidance has sufficiently clarified the level of appropriate flexibility in accepting new surrogate endpoints to support accelerated approval, addressed poor rigor in confirmatory study design, or addressed FDA’s response when confirmatory studies fail to clearly demonstrate a drug’s clinical benefit. Therefore, these concerns remain unresolved, jeopardizing the promise and success of the accelerated approval program. In addition, although FDA has a Biomarker Qualification Program intended to support the identification and development of new biomarkers and to qualify biomarkers for specific contexts of use (FDA, 2021), sponsors have little incentive to undertake the necessary studies as they are complex and expensive, and qualification is not a required condition of using biomarkers to support approval.

Conclusion 5-4: Given important concerns about the accelerated approval pathway, recent adjustments need to be carefully monitored and further adjustments may be needed to promote rigor and confidence that accelerated approval drugs will, as quickly as is possible, confirm meaningful clinical benefit for patients or be withdrawn.

Conclusion 5-5: Current legislation and polices for FDA are sufficient to foster the approval of innovative drugs for unmet needs.

Recommendation 6: To maintain the appropriateness of Food and Drug Administration (FDA) programs that expedite the development and review of therapies in areas of unmet need, including the accelerated approval program, FDA should generously use its authority to impose postmarket study requirements, ensure that required postmarket studies are appropriately designed to confirm clinical benefit, and strictly enforce postmarket study requirements. The following steps will support these goals:

1. FDA should ensure that confirmatory studies are well designed to evaluate clinical benefit and should prespecify the study results that will be deemed acceptable for conversion to traditional approval.

2. FDA should continue recent efforts to ensure that confirmatory studies are underway before approval is granted, making exceptions only in extreme cases.
3. If concerns about timely study completion arise during progress reports, then FDA and the sponsor should determine the steps needed to address barriers; any modification to study requirements should prioritize ensuring rigor.
4. For drugs whose studies fail to confirm clinical benefits following flexible approval, FDA should use its authority to rapidly withdraw approval.
5. FDA should lead an effort, in collaboration with the National Institutes of Health and the Centers for Medicare & Medicaid Services, to enable more efficient conversion of unvalidated endpoints to validated endpoints to advance therapeutic innovation.

Although it is not a specific program, FDA sometimes uses informal regulatory flexibility to expand patient access to therapeutics. This informal flexibility is applied to both accelerated approval products and to those receiving regular approval (see Chapter 5 for specific examples where FDA has used regulatory flexibility). Regulatory flexibility can be appropriate as long as there is strong reason to expect that clinical benefit will be confirmed later and those expectations are enforced by requiring that rigorous confirmatory studies be promptly completed.

As discussed in Chapter 5, FDA regulatory flexibility can include approval based on a single pivotal study, acceptance of single arm trials, acceptance of poor surrogate endpoints for accelerated approval, acceptance of surrogate endpoints outside accelerated approval, and approval despite trials missing prespecified endpoints, among other flexibilities. FDA acknowledges that for serious diseases, the agency may accept weaker trial designs and endpoints, fewer trials, or less statistical confidence, pointing to perceived patient “willingness to accept less certainty about effectiveness in return for earlier access to much needed medicines” (FDA, 2019).

Despite the substantial flexibility exercised both within the accelerated approval program and beyond it, some argue that FDA is still not flexible enough and that even greater regulatory flexibility is needed. The suggested solutions range from encouraging new regulatory flexibilities in certain cases, such as for rare diseases (Quandt, 2022; Woodcock et al., 2024), to calls for greater regulatory flexibility in general (Foundation for Government Accountability, 2022).

While greater regulatory flexibility can improve access to innovative therapeutics, there is an important balance between being appropriately flexible and weakening regulatory standards to such a great extent that patients and clinicians lose sufficient confidence that a drug will confer its

purported benefit. For example, regulatory flexibility may be critical for disease areas, such as ultrarare diseases, where it is nearly impossible to meet standard expectations. However, when well-designed trials are completed but fail to demonstrate a benefit, approving the drug does not meet patient needs; unmet need is addressed by access to good, effective drugs, not simply by allowing more drugs on the market.

Weak approval standards risk harming patients in a variety of ways, including through a lack of adequate information necessary for patients and clinicians to make informed treatment decisions, possible exposure to side effects (and costs) not justified by benefits, and lost opportunity to participate in trials of alternative therapies or to pursue other treatment or palliation plans that may be more suitable. In addition, weak standards can inhibit innovation by impeding the conduct of clinical trials for new products by, for example slowing patient recruitment (as patients pursue the uncertain-but-approved product instead of trial participation) or by complicating study design through requiring comparison to an unproven standard of care. Most importantly, weak approval standards fail to incentivize true innovation for the benefit of patients, instead accepting unproven or weak clinical options.

In the case of regulatory flexibility, it is in the public’s interest to maintain reasonably high standards for drug approval and when flexibility is offered, to ensure that benefit is confirmed after approval in a timely manner.

Finding 5-7: FDA currently exercises a great deal of regulatory flexibility both within and beyond existing programs and sometimes extends this flexibility too far by approving drugs that are unlikely to—or fail to—confirm benefit.

Finding 5-8: Given the remaining unmet need, FDA is under pressure to increase regulatory flexibility beyond current approaches.

Conclusion 5-6: When traditional approval standards cannot be satisfied for scientific reasons, regulatory flexibility can be appropriate as long as the initial approval is based on a reasonable likelihood of clinical benefit and clinical benefit is rigorously and rapidly confirmed following approval.

Conclusion 5-7: Although patients facing serious unmet needs may reasonably be willing to accept greater uncertainty and risk, weak approval standards harm patients seeking clear information to guide treatment decisions, may impede the development of strong treatment options, and fail to incentivize investment in true innovation.

Recommendation 7: To ensure that regulatory flexibility is exercised in a manner that promotes the approval of drugs that are both safe and effective, the Food and Drug Administration (FDA) should uphold strong regulatory approval standards. When FDA exercises flexibility, whether through accelerated approval or outside that pathway, the agency should require rigorous, timely confirmatory studies.

As discussed earlier in Chapter 5, FDA has a number of efforts underway to encourage investment to address unmet needs; to address broad scientific challenges, improve coordination, and encourage data generation to address unmet needs; and to facilitate and expedite development, review, and approval to address unmet needs. For FDA to maintain many of these pathways and initiatives, the agency requires appropriate funding and staffing. “FDA is a staff-intensive agency, with approximately 80 percent of its budget devoted to personnel costs” needed to appropriately recruit and retain individuals with the expertise needed to regulate food and drug products (NTEU, 2024). However, FDA has faced a number of concerning staff disruptions recently, including layoffs, retirements, forced resignations, and departures (Cranmer, 2024; Karlin-Smith, 2024; Lawrence, 2024; Lawrence and Feuerstein, 2025; Lawrence and Parker, 2025; Wilkerson, 2023), threatening the agency’s ability to manage its intensive workload and advance innovative approaches (Hopkins, 2025).

Maintaining and expanding the FDA workforce is essential to allowing the agency to keep pace with technological and scientific breakthroughs (Deloitte, 2018). For example, holding frequent meetings between FDA staff and sponsors is critical to ensure that applications are as robust as possible. Even prior to recent layoffs, FDA needed additional support to advance innovation for areas of unmet need. With the recent layoffs and departures, and the associated uncertainty they have wrought for federal employees who already accept far less than they could earn in industry in order to support FDA’s public health mission, it is unlikely that FDA will be able to maintain or expand its pilot programs helping spur research and expedite approval for needed therapeutics. Ultimately, without congressional action to adequately fund FDA and protect appropriate staffing levels, the agency will not be able to support the kind of workforce, in terms of expertise and experience, needed to advance therapeutic development for unmet needs.

Conclusion 5-8: Support for innovation in the pre- and postmarket settings requires a well-resourced regulatory environment, including attracting and retaining FDA staff with the necessary experience and expertise.

Recommendation 8: Congress should authorize a significant expansion of Food and Drug Administration (FDA) staffing and consistent resources to support the implementation of Recommendations 6 and 7, and especially to ensure that FDA has sufficient resources to monitor and enforce requirements for postmarketing surveillance and drug evaluation research.

GOAL 5: STRENGTHEN A FISCAL AND POLICY ENVIRONMENT TO ALIGN REIMBURSEMENT POLICY WITH EVIDENCE-BASED THERAPEUTIC VALUE AND THE EXTENT TO WHICH PRODUCTS ADDRESS UNMET NEED

The current U.S. drug pricing and reimbursement system creates strong incentives for innovation—particularly for novel therapies addressing unmet needs—but, at the same time, it may perpetuate inequitable access to effective treatments. Notably, the United States spends more on health care per capita than other high-income countries (Gunja et al., 2023; Peter G. Peterson Foundation, 2024; Wager et al., 2024), while not delivering commensurate levels of quality of outcomes.

Reimbursement policy through public (Medicare and Medicaid) and private plans (employer-sponsored and individual market health plans) determines whether a novel drug is covered and, if covered, the amount paid by beneficiaries. As reviewed in Chapter 4, disincentives for innovation can occur if novel drugs that address unmet need are not covered or are not readily accessible by patients through access restrictions, such as utilization management strategies or by cost-sharing (i.e., copay obligations) that is too high for patients to afford. In addition, providing high coverage and payment for therapies without demonstrated clinical benefit or substantive innovation undermines the potential of reimbursement policy to incentivize the development of high-value treatments over marginal improvements. One approach that may increase the clinical value of a product is through the development of diagnostics that can enable segmentation of a broader patient population for personalized medicine approaches. Aligning reimbursement policy with evidence-based value, of which unmet need must be a component, is key to this committee’s aims of promoting innovation and addressing unmet need.

A revised approach to reimbursement policy could help address innovation incentives for therapeutic development by linking pricing to evidence-based value assessments that incorporate clinical effectiveness, patient perspectives, and assessments of unmet need. Several other countries offer useful models for this approach—prioritizing payment for new therapies that address unmet need and limiting reimbursement for products that offer marginal improvements or whose clinical benefit remains unproven. For

example, the United Kingdom and Germany both have systems for evaluating the clinical effectiveness of a treatment to determine payment (Lemley et al., 2020). (See Chapter 5 for more information about reimbursement and payment policy.) Linking pricing to value is also important because prioritizing payment for high-value innovative therapies (while limiting overpaying for lower value treatments) could make more resources available for improving coverage for effective therapies. That is, coverage should also follow evidence.

Accordingly, there is an impetus to align reimbursement policy in a way that incentivizes companies to invest in innovative new therapies that provide value by addressing unmet need. Research indicates that pharmaceutical companies respond to financial incentives and direct innovation efforts toward areas with greater financial incentives (Hemel and Ouellette, 2023). Thus, this is a promising avenue to pursue.

Reforming reimbursement policy and incentivizing innovation to address unmet needs requires grappling with the definition and value assessment of unmet need. Value-based pricing can be implemented through different approaches, which need not be directly linked to unmet need. However, if the value of a new therapy is defined based on the benefits it provides relative to the current standard of care, then value will be aligned with the degree to which the therapy addresses unmet need. Societal value and addressing unmet need are thus mutually reinforcing concepts that are central to this committee’s recommendations. Furthermore, a frequent criticism of aligning reimbursement policy with value is that it will lead to increased insurance premiums, resulting in fewer people buying insurance and getting access to therapeutics. Another criticism is that negotiating drug prices will make innovation more difficult or costly. However, in this report, the focus is on spending more efficiently on therapeutics, not spending more. This means redistributing spending to align better with the value of drugs that are available to patients, so that insurers are paying more for higher value drugs and less for lower value drugs. Therefore, net spending would be unchanged, but for the same amount of spending, more patients that need high value medicines would gain access.

Finding 5-13: An expectation of increased financial return for a certain class of pharmaceutical product has been shown to increase R&D effort on those products.

Finding 5-14: Many countries negotiate drug prices and set reimbursement terms based on the product’s cost-effectiveness, including considerations of unmet need.

Finding 5-15: CMS has statutory authority to use comparative effectiveness and unmet medical needs for a limited number of older drugs under the Medicare price negotiation and New Technology Add-On Payment programs, but it otherwise lacks authority to control Medicare or Medicaid reimbursement amounts for most new drugs.

Conclusion 5-10: If public and private payer reimbursement policies were more aligned with evidence of product value and the extent to which a drug addresses unmet medical need, greater innovation would occur in therapeutic areas with high unmet need.

Conclusion 5-11: Congressional action is needed to more directly tie prices and public insurance reimbursement for novel drugs that address unmet need to evidence-supported measures of value or impact.

Recommendation 9: Congress should reform the statutory framework that regulates public reimbursement for novel drugs to better align reimbursement rates with evidence of clinical benefit as compared with existing therapeutic alternatives, if any. This could include:

1. Expand the Centers for Medicare & Medicaid Services’ authority and capacity to negotiate prices beyond the scope of the Inflation Reduction Act to account for the value of the drug relative to alternatives or a standard of care, including the extent to which it addresses unmet need.
2. For drugs with negotiated prices, set reimbursement terms that maximize patient access through more favorable cost-sharing, robust formulary coverage, and more tailored application of utilization management tools (e.g., prior authorization, step-therapy, and quantity limits).

Recommendation 10: The Centers for Medicare & Medicaid Services should use its existing regulatory authority to reduce the mismatch between Medicare reimbursement for a drug and that drug’s ability to address unmet medical needs, including through its implementation of the drug price negotiation program and the New Technology Add-on Payment program.

Although Recommendation 10 is targeted at CMS, the goal is for both public and private insurance systems to align payment with evidence of clinical benefit. However, because CMS is the largest single provider in the U.S. market, the prices it negotiates are likely to have ripple effects, affecting drug prices in the private market as well.

One-time, curative or regenerative medicine therapies face particular challenges in the current market and reimbursement system. Novel onetime, or limited duration curative, therapies have been developed in recent years but have often not easily reached patients, in part because of high up-front costs and the fragmented nature of U.S. health insurance coverage. Recent examples of such therapies have included treatments for beta thalassemia, immunologically driven diseases, and sickle cell anemia. With new developments in cell and gene therapies in recent years, the number of such therapies is increasing. It is important that these therapies continue to be developed—some are highly effective and innovative with the potential to revolutionize the future of targeted therapeutics for other conditions and provide long-term cost savings, but the market and access barriers are significant.

The benefit designs and utilization management employed by many insurance payers in the United States make it difficult for clinicians, individual patients and manufacturers, and innovators to navigate coverage and patient access for these very high-priced drugs. This has resulted in lower or no access to certain drugs, depending on insurance coverage and even geography (e.g., variation by states among Medicaid insured populations). Private market efforts to ameliorate these challenges, such as outcomes-based contracts, have not been broadly effective. Recent efforts by the Center for Medicare and Medicaid Innovation through its Cell and Gene Therapy Access Model targeting Medicaid coverage for sickle cell therapies reflect the growing recognition for the need of more consolidated efforts for determining reimbursement, market access, and data collection needs for these therapies.

Among commercially insured patients, access is also a challenge since smaller insurers and employers may be unable to afford the high costs of practically any of the curative therapies, and traditional sources of reinsurance and risk pooling may explicitly exclude coverage for these therapies (Phares et al., 2024). Even among larger employers, there may be resistance to covering high-cost therapies where benefits are perceived to accrue to future employers, insurance plans, or government payers.

Recent evidence also suggests that as product commercialization has, in some cases, underperformed investor expectations, new investments in cell and gene therapies and curative therapies are being challenged and drug developers have begun to retreat from this market. Given the exceptional benefits of these products for patients (and especially for those with very rare diseases), it is imperative to improve market access for these novel therapies that aim to address unmet needs. To improve affordability and access for patients and to sustain investments in these needed innovative treatments, novel solutions for payment, coverage, and market access for drug developers, payers, and patients are needed (Phares et al., 2024).

Conclusion 4-1: Innovative therapies are emerging for rare diseases and other complex conditions, offering a potential for cure. However, the fragmented payment system within the United States is a barrier to patient access, resulting in underinvestment in developing curative therapies. The current U.S. market and policy environment is unprepared to manage these one-time, very high-cost therapies. There is a need for a clearer reimbursement structure for innovators developing these high-cost curative treatments.

Recommendation 11: Congress should support the development of a negotiation and access model for one-time curative therapies to ensure access for patients and market access for innovators of novel therapies.

Congress should instruct an organization or agency, such as the Medicare Payment Advisory Commission or the Medicaid and CHIP Payment and Access Commission, to study and develop recommendations for legislation to create a new program addressing the unique deficiencies of the nation’s current insurance system in enabling access to curative therapies. The details of designing this system are beyond the scope of this report, but several possibilities and considerations have been identified.

The program would likely be based on carving coverage of these therapies out of the myriad current insurance programs and into a new national risk pool that would serve as the source of coverage, access, and payment for these medicines. The program could be managed by either private entities or public, depending on the preferences of Congress. Sustainable fair prices would be negotiated by the administrating entity along with guidelines for appropriate access. The process for price benchmarking and negotiations will have to be defined and would likely benefit from the greatest possible transparency. The program design will need to contemplate the nature of the medicines eligible for inclusion, such as the size of the eligible population, the price of the treatment, the duration of treatment, and the curative success rate and sustainability, including evidentiary requirements. The program will likewise need to consider whether all such medicines are to be automatically enrolled or whether participation would be voluntary for both parties and so based on a successful negotiation of mutually satisfactory fair prices and fair access. Funding for the new program will also have to be developed. Some possibilities might include a new tax on insurance premiums making up for the carve out, a new Medicare tax, a new general income tax, or a new tax on generic medicines.

Medicare coverage through Social Security disability insurance could provide one potential pathway for access. For example, Medicare coverage could be expanded to include coverage for one-time curative therapies, similar to its coverage for individuals diagnosed with end-stage renal disease or

amyotrophic lateral sclerosis. Coverage could be limited to a defined treatment episode for individuals insured by commercial or Medicaid payers. In addition, Medicare could use an approach to price negotiation similar to the one used under the Inflation Reduction Act, allowing for fair prices and fair access for drugs that otherwise may not reach patients under the status quo.

REFERENCES