WASHINGTON — The National Academies of Sciences, Engineering, and Medicine has issued a sweeping new report urging the U.S. government to bridge the growing gap between where research dollars are spent and the diseases that most severely affect Americans. The study calls for the creation of a new federal consortium, major shifts in funding strategies, and coordinated action from Congress to ensure that therapeutic development aligns with the real medical needs of the population.
Despite being the world leader in biomedical innovation, the report warns that America’s research priorities are often misaligned with the actual burden of disease. This mismatch leaves many common and deadly illnesses without adequate treatment options, while other areas receive far more attention than their public health impact warrants.
Misalignment Between Funding and Need
The National Academies highlight a stark imbalance: illnesses such as cardiovascular disease, chronic obstructive pulmonary disease (COPD), and chronic pain conditions — which collectively affect tens of millions of Americans — receive disproportionately low levels of investment compared to their enormous social and economic cost. Cardiovascular disease alone remains the leading cause of death in the United States, yet research funding for it lags far behind areas like Alzheimer’s or certain cancers.
Meanwhile, even in areas that receive heavy investment, like Alzheimer’s disease, treatments remain largely ineffective. Despite billions spent on research, the therapies available today still fail to meaningfully address the devastating effects of neurodegenerative decline, underscoring the report’s central argument: research investment does not always translate into real-world medical progress.
Donald Berwick, president emeritus and senior fellow at the Institute for Healthcare Improvement and co-chair of the report’s committee, emphasized the urgency of realignment. “As a global leader in scientific innovation, the United States should be able to deliver treatments that meaningfully reduce the burden of disease on every family,” he said. “But the chronic disconnect between public health needs and where our research dollars go has slowed progress and left millions without effective options.”
Rethinking Funding Priorities
The report calls for federal and private research funders to explicitly consider disease burden and unmet medical needs when allocating resources. It urges Congress to integrate these priorities into its funding decisions, particularly when distributing support among the various institutes and centers that make up the National Institutes of Health (NIH).
The NIH, the report notes, provides the essential foundation for drug development — de-risking early research, informing private sector investment, and driving innovation across the biomedical ecosystem. But without careful alignment, even well-funded programs can fail to address the conditions that cause the most suffering.
To tackle this, the report proposes the establishment of a cross-agency consortium, led by the U.S. Department of Health and Human Services. This consortium would track current research investments, measure unmet therapeutic needs, and identify areas of imbalance. It would also provide Congress with a data-driven basis for future funding decisions.
Ellen MacKenzie, Bloomberg Distinguished Professor and Dean of the Johns Hopkins Bloomberg School of Public Health, and co-chair of the committee, said this approach could transform how research dollars translate into health outcomes. “Our nation’s scientific infrastructure is second to none,” she said. “By coordinating federal research priorities and focusing on unmet needs, we can bring life-changing treatments to the people who need them most.”
Encouraging Innovation and Access
The report also points to the rapid emergence of cutting-edge therapies — particularly in the areas of gene and cell therapy — that have the potential to cure or significantly reduce the impact of once-incurable diseases. However, the fragmented nature of the U.S. healthcare payment system has created new barriers to patient access and discouraged long-term investment.
To counter this, the committee recommends that Congress design a new payment model specifically for one-time curative therapies. Such a model would allow insurers to spread the costs of high-priced cures over time, while ensuring patients can access them immediately. This structure would also provide greater financial predictability for innovators developing transformative but costly treatments.
Additionally, the report calls for reforms in how reimbursement rates are determined, proposing that rates better reflect a therapy’s proven clinical benefit. This would reward true innovation and ensure that funding follows evidence rather than market momentum.
Strengthening Collaboration and Transparency
Another key recommendation is for the NIH to develop a public, searchable repository of therapeutic candidates or research pathways that have been discontinued by private companies. These “orphaned” projects, often abandoned for commercial reasons rather than scientific ones, could represent significant opportunities for continued development by academic researchers, nonprofits, or smaller biotech firms.
By creating a centralized database, NIH could help bridge the gap between early-stage discovery and real-world application. Pharmaceutical companies could voluntarily share unused intellectual property, enabling public-private collaborations that might otherwise never occur.
The report also urges federal agencies to make greater use of existing nonprofit foundations, such as the Foundation for the NIH and the CDC Foundation, to support cross-sector partnerships. These entities can act as flexible intermediaries, attracting philanthropic and private sector participation in areas where traditional market incentives fall short.
Strengthening FDA Oversight
The Food and Drug Administration (FDA) plays a critical role in ensuring that promising therapies reach patients safely and efficiently. The committee recognized the FDA’s existing programs that accelerate development for areas of unmet medical need, but expressed concern that post-approval studies are often delayed or incomplete.
To address this, the report recommends that the FDA expand its capacity to enforce timely completion of confirmatory studies and ensure that new treatments deliver the expected clinical benefits. Congress, it argues, should authorize additional staffing and stable funding to support these responsibilities.
Victor J. Dzau, president of the National Academy of Medicine, described the recommendations as a roadmap for the future of American health innovation. “This report lays out a clear strategy to align our nation’s scientific and economic power with the realities of disease burden,” he said. “Through better coordination, transparent funding, and stronger public-private partnerships, we can accelerate progress toward therapies that will transform lives.”
A Vision for the Future of U.S. Biomedical Research
The report concludes that a holistic, evidence-based realignment of research priorities could dramatically improve the health of the U.S. population. This would require unprecedented coordination between federal agencies, research institutions, private companies, and Congress — but the potential payoff is immense.
The committee behind the study, formally known as the Committee on Strategies to Better Align Investments in Innovations for Therapeutic Development with Disease Burden and Unmet Needs, conducted its work with sponsorship from Gates Ventures and the Peterson Center on Healthcare.
Since their founding in 1863 under a congressional charter signed by President Abraham Lincoln, the National Academies of Sciences, Engineering, and Medicine have served as independent, nonprofit advisors to the federal government. Their mission is to provide evidence-based recommendations that help guide public policy in science, technology, and medicine.
This latest report, the Academies say, is not merely a critique of the current system — it is a call to action. The United States possesses the scientific talent, infrastructure, and resources to revolutionize healthcare outcomes. What remains is to ensure that these resources are directed where they will have the greatest human impact.
As the report concludes, aligning investments in therapeutic innovation with the realities of disease burden is not just a scientific necessity — it is a moral imperative. By rethinking how the nation invests in health research, America has the opportunity to transform the landscape of medicine, address decades of unmet needs, and ensure that the next generation of cures truly serves those who need them most.
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