Leadership and Program Changes Affecting the Rare Disease Community—And How You Can Take Action
The EveryLife Foundation Update
April 7, 2025
Navigating Uncertainty the Rare Way
The rare disease community is starting a new week facing a significantly different reality following last week’s stream of changes to the biomedical, regulatory, and public health programs on which the community relies. On March 27, the U.S. Department of Health and Human Services (HHS) announced a dramatic restructuring following President Trump’s Executive Order, “Implementing the President’s ‘Department of Government Efficiency’ Workforce Optimization Initiative.”
The plan included the elimination of more than 20,000 federal roles as part of a major restructuring that is impacting operations at the U.S. Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH), the Food and Drug Administration (FDA), the Centers for Medicare and Medicaid Services (CMS), the Health Resources and Services Administration (HRSA), the Administration for Community Living (ACL), and others with direct impact on our rare community.
Last week, the plan was put into place. Here are a few details that we know about the changes to leadership and programs most directly impacting the rare disease community:
Approximately 10,000 HHS employees were laid off, in addition to the reported 10,000 that had already been let go or left voluntarily. While the layoffs affected agencies across the board, divisions focused on scientific, regulatory, and public health policy, as well as communications offices and public health surveillance offices, were particularly hard hit.
Significant leaders that the rare disease community has built relationships with over years have either resigned or been laid off. The forced departure of Dr. Peter Marks, the former head of the Center for Biologics Evaluation and Research, the removal of five heads of NIH Institutes, and the loss of leaders of programs across the board mean the loss of powerful allies and institutional knowledge that has generated the progress the rare disease community has seen over the last decade.
Shockingly, on April 3, we also learned that HHS terminated the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). This committee reviews the evidence and makes recommendations for conditions to be added to the Recommended Uniform Screening Panel (RUSP), which states rely on to guide what screenings newborns receive. Since its creation in 2004 and the adoption of the first RUSP in 2010, the ACHDNC has been the engine fueling a robust, evidence-based newborn screening system that is saving lives.
Take Action – Please Restore the Federal Newborn Screening System
The biomedical research, public health, and therapy development ecosystems have all had major changes that we will continue to assess in the days and weeks to come. The EveryLife Foundation will work together with our community to continue to build new champions to advocate for the policies that matter to us and fight to create ecosystems that do not lose sight of the rare disease community. We will keep you updated on ways you can use your voice and tell your story to ensure that the urgency and priorities of the 10% of our nation living with rare diseases are understood.
Our Moment is Now
The Rare Pediatric Disease Priority Review Voucher (PRV) Program is a market-based incentive that has led to the development of new treatments for 40 rare diseases at no cost to taxpayers. Unfortunately, this life-saving program lapsed on December 20, 2024, despite being extended for five years in the House-passed Give Kids a Chance Act and having broad bipartisan support.
Over 200 patient organizations have advocated for the extension of the PRV Program. In the early days of 2025, the rare disease community maintained that momentum, calling on Congress to take immediate action to rectify this oversight.
Your efforts are working, but we need both the House and Senate to urgently schedule markups of the Give Kids a Chance Act, the bill that reauthorizes the Rare Pediatric PRV Program. A markup is an important step toward reauthorizing the PRV Program. We can’t let up now.
This is an incredibly challenging time. There is no roadmap to help us navigate this uncertainty. But you are not alone. The EveryLife Foundation team is working for you – and alongside you. Our North Star is our resilient rare community – each and every one of us. United. Amplified. And stronger than ever.