The Advocate

May 2025: Volume 5, Issue 5

Want to stay up to date on news and events relevant to patients, caregivers, and others affected by rare diseases?

In This Issue:

Dr. Vinayak Prasad Becomes New Head of CBER
#ASGCT2025 Highlights: Baby KJ, Regulatory Deep Dives, Ensuring Access to CGTs
Senate Hearing Explores U.S. Biomedical Innovation
Congress Begins Hearings on President’s FY26 Budget Request
FY25 Reconciliation Advances Amid Debates on Medicaid Cuts
In Brief: Congressional and Executive Agency Updates
Events
Around the Field

ASGCT Advocacy

Dr. Vinayak Prasad Becomes New Head of CBER

Dr. Vinayak Prasad has been officially appointed as the new director of the FDA’s Center for Biologics Evaluation and Research (CBER), the division responsible for overseeing vaccines, gene therapies, blood products, and other biologic medications. Vinayak Prasad, MD, MPH, is a hematologist-oncologist and professor at the University of California, San Francisco.

#ASGCT2025 Highlights: Baby KJ, Regulatory Deep Dives, Ensuring Access to CGTs

The ASGCT 28th Annual Meeting in New Orleans gathered thousands of cell and gene therapy stakeholders to share the latest research and policy developments. Registrants can watch recorded sessions on-demand until July 18.

An infant patient is the first-ever person to be treated with a custom CRISPR gene editing therapy, as announced at our Annual Meeting and published in The New England Journal of Medicine. The team of researchers caring for the patient, known as KJ, was led by Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunuru at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine. In just six months they worked with a network of academic and industry partners to build a base editor, complete preclinical testing, receive FDA authorization for an n-of-1 clinical trial, and manufacture the therapy. Three months post-treatment, doctors are monitoring baby KJ’s health and seeing promising signs on clinical efficacy. This announcement is a testament to the strength of the U.S. biomedical enterprise, the promise of NIH-funded research, and the critical role of FDA in moving innovative therapies forward.
Patient Access to Therapies: Sessions featured researchers including Dr. Ahrens-Nicklas, who explored the role of biomarkers; patient advocates; and private insurers. Speakers across sessions discussed barriers to access and the need to center patient experience in CGT policy decisions and clinical trial design.
Regulatory and CMC: Presenters dove into topics including a 1:1 fireside chat with a representative from the European Medicines Agency (EMA), global Chemistry, Manufacturing, and Controls (CMC) harmonization efforts, FDA’s START Pilot Program, and a survey of current FDA draft guidances for CGTs.
Innovative Funding and Development: A fireside chat on Funding the Future of CGT Development brought together representatives from public-private funders to examine funding available beyond R01 grants. Another session focused on Reviving Hope in Deprioritized CGT Programs, highlighting challenges for CGTs to sustain development.

Senate Hearing Explores U.S. Biomedical Innovation

The U.S. Senate Appropriations Committee held a hearing on April 30 titled Biomedical Research: Keeping America's Edge in Innovation. ASGCT submitted a statement for the record outlining the remarkable growth in the CGT pipeline; the need for robust National Institutes of Health (NIH) funding and protection of facilities and administrative (F&A, or indirect) reimbursements that underpin research infrastructure; and adequate FDA funding and staffing to ensure safe, timely access to groundbreaking therapies.

During the hearing, participants echoed ASGCT’s priorities. Chair Susan Collins (R-ME) asked about the consequences of imposing a 15% F&A cap, discussing the risk such changes pose to the operational capacity of research institutions. Collins also highlighted the economic benefits of NIH funding across the U.S. Vice Chair Patty Murray (D-WA) raised the issue of proposed major funding cuts federal health agencies, warning that reductions in grant availability and agency instability could undermine U.S. leadership in biomedical innovation.

Congress Begins Hearings on President’s FY26 Budget Request

In early May the President’s Fiscal Year 2026 (FY26) Budget Request was publicly released. Each administration puts together an annual president’s budget, in which they request funding from Congress for priorities in the upcoming fiscal year. Congressional appropriators take the President’s budget request into consideration and craft their own appropriations bills, which then must be signed by the President before becoming law. Thus, while the President’s Budget Request does not have the force of law, it is an important guide to the Administration’s priorities.

The President’s budget request proposes significant funding reductions for federal health agencies. The proposal would reduce NIH funding by nearly 26%, or approximately $18 billion. FDA funding levels are not outlined in the proposal. For NIH, the budget request also includes a proposal to consolidate the current 27 NIH institutes into five institutes and cut funding for several existing offices. ASGCT joined stakeholders in voicing concerns to Congressional leaders via letters: one encouraging robust NIH funding overall, and two others on funding for ARPA-H and the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).

Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr. testified in three Congressional Committees over the last week: the Senate Health, Education, Labor and Pensions (HELP) Committee and House Appropriations Subcommittee on Labor-HHS on May 14, and the Senate Appropriations Subcommittee on Labor, HHS, and Education on May 20. Specific to CGTs, Senator Tim Scott (R-SC) asked about the impact on U.S. leadership in biomedical innovation, while Senator Tammy Baldwin (D-WI) highlighted the case of baby KJ as an example of groundbreaking research made possible by federal funding. Members in all three hearings addressed NIH F&A rates and disrupted clinical trials, as well as recent HHS layoffs and restructuring plans. Up next is the Senate Appropriations Subcommittee on Agriculture-FDA on May 22, with FDA Commissioner Marty Makary scheduled to testify.

FY25 Reconciliation Advances Amid Debates on Medicaid Cuts

On May 18 the House Budget Committee advanced the FY25 reconciliation package on a 17–16 vote, but at the time of publication it has not yet been advanced to the full House. The bill proposes significant changes to Medicaid. The Energy & Commerce Committee's portion, debated and advanced on May 14, includes $625 billion in Medicaid cuts over ten years. The Congressional Budget Office estimates that changes in the bill could result in 10.3 million people losing Medicaid coverage and up to 7.6 million becoming uninsured, a particular concern for the rare and pediatric disease communities. The package also includes health care measures from the Ways & Means Committee, such as the expiration of premium subsidies for Affordable Care Act marketplace plans and reforms to pharmacy benefit managers (PBMs).

In Brief: Congressional and Executive Agency Updates

Commissioner Says No Major FDA Reorganization: An internal HHS memo had proposed consolidating FDA’s product review divisions into one office. Commissioner Makary has since said he is not planning a major reorganization at this time.
PDUFA VIII Kickoff: FDA posted a notice that the first public meeting to begin PDUFA VIII negotiations (FY28-FY32) will take place July 14. FDA Deputy Commissioner Grace Graham told a conference on May 16: “It is worth exploring whether and how restructuring and simplifying the user fee programs may help rebuild trust in FDA decisions, and take advantage of the upcoming reauthorization to do so.”
AdComm Participation: FDA Commissioner Makary announced a policy restricting individuals with certain industry financial interests from serving on advisory committees.
FDA Leaders on Surrogate Endpoints: During a May 20 webinar, An Evidence-Based Approach to COVID Vaccination, Drs. Makary and Prasad briefly discussed surrogate endpoints for cancer therapeutics and Accelerated Approval for rare disease.
Deregulation Order: An executive order in January established a stringent deregulatory framework for federal agencies; a recent memo from OMB provides more information on implementation. For FDA, new regulations or guidance documents could be delayed by the requirement to offset costs through the elimination of existing regulations.
New Report on U.S. Biotech Leadership: The National Security Commission on Emerging Biotechnology (NSCEB) issued its final report to Congress, warning that the U.S. is “dangerously close to falling behind China” in biotechnology leadership. The report highlights gene therapies as critical national security assets.
Indirect Cost Caps: The federal government has appealed a permanent injunction that prevents enforcement of its proposed 15% F&A cap for NIH grants. The court found that NIH acted “arbitrarily and capriciously” and failed to follow proper rulemaking.
CGT Access Model: HHS announced that 35 states are participating in the CMMI CGT Access model. The full list of participating states is expected to be released in June.
Most Favored Nation Proposal: HHS shared initial details on a proposed “Most Favored Nation” drug pricing policy, which would require pharmaceutical companies to match U.S. prices to the lowest price offered in certain other countries.
EO on Prescription Drug Costs: President Trump issued an executive order (EO) outlining approaches to reduce prescription drug costs. Many of the proposals outlined in the EO echo those proposed in the President’s first term. HHS released more details on the implementation of this EO on Monday.
EO on Domestic Drug Manufacturing: President Trump issued an executive order directing FDA, EPA, and other agencies to streamline regulations and permitting processes for pharmaceutical manufacturing facilities in the U.S.

Events

Save the Date for the 2025 Policy Summit

The Policy Summit will return Sept. 25-26, 2025, at the Washington Marriott Georgetown in Washington, D.C.! Save the date now and watch this newsletter in the coming months to learn the latest on speakers, topics, and registration.

Register for NORD's Rare Disease Symposium

Registration is open for NORD’s Rare Disease Scientific Symposium on June 2-3 in Washington, D.C. Through a series of case study presentations and extended Q&A, attendees will explore innovative strategies for overcoming challenges in rare disease research, from patient data collection to AI integration.

Join NIST's Genome Editing Consortium Workshop

The NIST Genome Editing Consortium (GEC) Workshop will take place June 12.
Register to join valuable discussions that will shape measurement standards.

Around the Field

The Standards Coordinating Body is inviting feedback via its updated Needs Assessment Survey. If you’ve filled it out in the past, you’re encouraged to participate again – responses are reviewed regularly to inform ongoing standard-setting efforts.
The National Academies Committee on Strategies to Enhance Pediatric Health Research Funded by NIH is seeking feedback by July 1 from individuals conducting pediatric health research who have applied for or received NIH funding, including academic researchers, physician-scientists, non-clinical faculty scientists, and nurse researchers.