WASHINGTON — A new report from the National Academies of Sciences, Engineering, and Medicine calls for a new consortium among federal agencies, changes in funding practices, action from Congress, and other steps to address the misalignment between disease burden in the U.S. and the areas of therapeutic development in which public and private funders invest.  

The report says that despite the U.S. leading in biomedical innovation, this misalignment means existing therapeutics do not sufficiently manage or treat the diseases that Americans face, leaving them with unmet medical needs. The report points to diseases such as cardiovascular disease, chronic obstructive pulmonary disease (COPD), and pain conditions, which affect millions of Americans and cost billions of dollars to treat annually, but have received relatively low levels of research funding to develop treatments relative to their large disease burden. At the same time, diseases like Alzheimer’s receive significant research funding yet still represent a critical unmet need — because the treatments that research has so far been able to produce do not adequately address the disease’s profound impact.  

“As the global leader in scientific innovation, the United States should be able to offer treatments that ease the burden of diseases that affect virtually every family and community,” said Donald Berwick, president emeritus and senior fellow at the Institute for Healthcare Improvement, and co-chair of the committee that wrote the report. “However, the chronic misalignment of the actual medical needs of the public with where research dollars go has hindered progress.”  

The report recommends that funders of biomedical research consider disease burden and unmet needs when directing funds — including Congress as it allocates support among NIH institutes and centers. NIH provides the foundation for the development of new drugs and helps to inform and lower the risk of industry investments, the report says. Congress should also establish and finance an interagency consortium, led by the U.S. Department of Health and Human Services and charged with tracking and assessing unmet therapeutic need and current investments, with a critical focus on identifying areas of mismatch.  

“Our report lays out steps the federal government should take to bring our nation’s incomparable research capabilities to bear in producing treatments that could change the trajectory of hundreds of millions of lives,” added committee co-chair Ellen MacKenzie, Bloomberg Distinguished Professor and the dean of the Johns Hopkins Bloomberg School of Public Health. “Public investments in biomedical research, including from NIH, are critical for driving discovery and developing new therapeutics to address unmet needs.”

Supporting Emerging Innovations

Innovative therapies and cures for many diseases are emerging — such as cell or gene therapies — but a fragmented payment system impedes patient access and leads to underinvestment in developing cures, the report says. Congress should support the creation of a new payment model to promote patient access to one-time curative therapies, which would also create clearer reimbursement strategies for innovators pursuing high-cost curative treatments. Congress should also reform regulation around reimbursement for new drugs so that reimbursement rates better align with evidence of clinical benefit.

Building New Collaborations

NIH should set up a searchable repository of therapeutic candidates or research avenues that are no longer in development by commercial sponsors — such as pharmaceutical companies — so that it can be shared with foundations and other entities that could potentially support further research or testing. The information in the repository could be voluntarily provided by companies that may want to enter public-private partnerships to develop a product.   The report recommends federal agencies increase use of their nonprofit entities, such as the Foundation for the National Institutes of Health or the Centers for Disease Control and Prevention Foundation, in order to build public-private partnerships in areas of mismatch.

Reviewing and Approving Therapies

The Food and Drug Administration plays an important role in regulating and reviewing new therapies. FDA already has several programs designed to drive innovation in therapeutic areas of unmet needs, but the committee found that postmarketing studies are not always completed in a timely fashion and recommended that FDA do more to ensure timely completion of these confirmatory studies. Congress should authorize a significant expansion of FDA staffing and consistent resources to support the implementation of the report’s recommendations.  

“This report presents an innovative road map that aligns our immense capability to produce breakthrough therapies with the nation’s unmet medical needs and disease burdens. We can achieve this alignment through increased collaboration among federal agencies, establishment of an interagency consortium, prioritization in funding, supportive congressional actions, and public-private investment,” said Victor J. Dzau, president of the National Academy of Medicine. “This is a path we must pursue now to improve health outcomes for all.”  

The study — undertaken by the Committee on Strategies to Better Align Investments in Innovations for Therapeutic Development with Disease Burden and Unmet Needs — was sponsored by Gates Ventures and the Peterson Center on Healthcare. The National Academies of Sciences, Engineering, and Medicine are private, nonprofit institutions that provide independent, objective analysis and advice to the nation to solve complex problems and inform public policy decisions related to science, engineering, and medicine. They operate under an 1863 congressional charter to the National Academy of Sciences, signed by President Lincoln.

Contact:

Megan Lowry, Media Relations Manager Office of News and Public Information 202-334-2138; email news@nas.edu