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Policy Update from the Washington Health Innovation Council

July 2024

Washington D.C. --

Throughout 2024, the Washington Health Innovation Council (WHIC) has featured challenges facing the rare disease community, including unique perspectives from patients, drug developers, investors and innovators. Another area of focus for rare disease is how changes in public policy can impact the possibility for advancements in the development of new treatments and cures. According to the National Organization for Rare Diseases (NORD) rare diseases affect 30 million people in the U.S, or 1 in 10 Americans.

You may be aware of the Rare Disease Priority Review Voucher Program (PRV) at the Food and Drug Administration (FDA) which is an incentive program for companies to develop therapies for challenging patient populations. Under this program, a company that has developed novel therapies for rare pediatric diseases can be awarded a priority review for their new drug application (NDA) or biologic license application that would otherwise not qualify for priority review. Without congressional reauthorization, the PRV program will expire September 30, 2024. Since the PRV program inception in 2006, 46 priority review vouchers have been awarded for 35 different rare pediatric diseases.

There is also important activity in both committees of congressional jurisdiction and oversight for the National Institutes of Health (NIH). The Health, Education, Labor and Pensions (HELP) Senate Committee Chairman Bill Cassidy and House Energy and Commerce Committee Chairman Cathy McMorris Rodgers have both recently proposed operational restructuring at the NIH. In response to an October 2023 Request for Information, this past May Senator Cassidy published a report of findings, including stakeholders concerns about NIH directing funding away from early-stage research in favor of supporting more late-stage research, neglecting funding in the crucial beginning stages of research could ultimately limit the creation of lifesaving cures and treatments. 

In the report, Cassidy emphasized the importance of NIH maintaining a balanced portfolio, so all stages of medical research and other public health priorities are adequately funded. The report also examined how the U.S. can sustain its advantage in biomedical research to ensure Americans receive the most innovative treatments as quickly as possible. Cassidy laid out several proposals to address this, including streamlining peer review of research, and addressing challenges in recruiting and maintaining our biomedical workforce. 

Also, in June 2024 the House Energy and Commerce Chairman, Cathy McMorris Rodgers published a new proposed Framework for NIH and requested feedback from stakeholders. The framework proposes reducing the number of institutes from 27 to 15, focusing some of the consolidations on biomedical research, and others on disease systems, such as neuroscience, disabilities and substance abuse. In the proposal Chairman Rodgers iterated we should heed misconduct made public in recent congressional hearings from the House Select Committee on Coronavirus and seek reforms to maintain a global competitive advantage for the U.S.

These proposals are far from passage and enactment into a true restructure at the NIH, however the recently released Labor-HHS FY 25 Appropriations bill text was released, outlining discretionary allocations four (4) percent below the FY24 enacted amounts. That level of cut – at an agency that is accustomed to annual increases – will force its own unofficial realignment internally at the NIH, affecting grant amounts for publicly funded lifesaving research and innovation.

From policy changes brought on in response to the COVID-19 pandemic, we have learned how our public health and research institutions can significantly impact the daily lives of Americans. While the FDA and NIH serve the rare disease community in different ways, their operational capacities play a meaningful role in how breakthrough therapies are developed and brought to market to ultimately benefit patients and their families. Follow the WHIC for important updates on these issues, and many others in the coming months.

By: Mia Heck, WHIC / JK Strategies Senior Consultant


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