The Advocate

August 2025: Volume 5, Issue 8

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

In This Issue:

Policy Summit Features FDA, NHGRI, ARPA-H, and CMMI
Congressional Briefing Addresses Barriers to Treatment for SCD Patients
Read Our Comments to FDA on PDUFA VIII
OBBBA Provisions Impact CGT Field
FY26 - FDA Appropriations Landscape
NIH Funding Developments
Learn About Updates to the Newborn Screening System
Events + Resources
In Brief: Policy Updates

ASGCT Advocacy

Policy Summit Features FDA, NHGRI, ARPA-H, and CMMI

We're thrilled to share additional speakers at the 8th Annual Policy Summit to discuss the latest developments in cell and gene therapy policy:

Dr. Denise Gavin, Director of the Office of Gene Therapy at FDA’s Office of Therapeutic Products, will bring years of regulatory experience to the table in CMC harmonization session.
Dr. Carolyn Hutter, Acting Director at the National Human Genome Research Institute (NHGRI) will highlight NHGRI’s strategic priorities for research and how NIH supports critical scientific endeavors like the Somatic Cell Genome Editing Consortium.
Dr. Mimi Lee & John Schiel from the Advanced Research Projects Agency for Health (ARPA-H) will detail their agency’s efforts to promote tech innovation and explore distributed manufacturing solutions.
Melissa Majero, Model Lead for the Cell and Gene Therapy Access Model at the Centers for Medicare and Medicaid Innovation (CMMI), will chat about lessons learned from the pilot program and how this could serve as a roadmap for future access.

This is your opportunity to engage directly with the key policy decision-makers who are shaping the future of CGTs. Don't miss this chance to ask questions, share your perspectives, and network with fellow leaders in the field. Register today!

Congressional Briefing Addresses Barriers to Treatment for SCD Patients

ASGCT co-hosted a congressional briefing in Washington, D.C. with the Children's Hospital Association. The briefing, Access to a Cure: Ensuring Equitable Access to Cell and Gene Therapies for Children with Sickle Cell Disease, drew congressional staff, clinical experts, and patient advocates. ASGCT extends our gratitude to Senator Tim Scott (R-SC), Senator Cory Booker (D-NJ), and Senator Raphael Warnock (D-GA) for their bipartisan leadership on SCD policy and for supporting the briefing.

The briefing addressed barriers to cell and gene therapy treatments for Americans living with sickle cell disease. Presenters outlined key areas for congressional engagement: supporting NIH research funding, renewal of the Rare Pediatric Disease Priority Review Voucher, addressing Medicaid reimbursement inconsistencies across states, and authorization of wraparound services for patients receiving gene therapy. Our blog has more detail on the conversations at the event.

Read Our Comments to FDA on PDUFA VIII

ASGCT submitted comments to FDA on the launch of the Prescription Drug User Fee Act (PDUFA) VIII negotiation process, outlining key priorities for the CGT community. Building on the progress made under PDUFA VII - including expanded review capacity at the Center for Biologics Evaluation and Research (CBER), new meeting types such as INTERACT, and greater focus on modernized manufacturing - ASGCT emphasized the importance of sustaining and strengthening these advances. ASGCT also recommended continued investment in CBER’s Office of Therapeutic Products to meet the growing complexity and volume of CGT submissions, with particular focus on CMC issues, advanced manufacturing technologies (AMTs), and platform technologies. Comments further called for the continuation and expansion of successful PDUFA VII pilot programs - including the Rare Disease Endpoint Advancement (RDEA) pilot, the CMC Development Readiness Pilot (CDRP), and the Split Real-Time Application Review (STAR) pilot.

OBBBA Provisions Impact CGT Field

The law passed in the reconciliation process, officially known as the One Big Beautiful Bill Act (OBBBA), included a number of provisions with implications for CGTs. For one, it broadens the Inflation Reduction Act’s orphan drug exception, now covering drugs and biologics with multiple rare disease indications, as long as all approved uses are for rare conditions. Further, it delays the start of price‑negotiation eligibility until a therapy receives its first non‑orphan approval, extending the negotiation-free period for CGT products focusing on rare diseases.

OBBBA also establishes a $50 billion Rural Health Transformation Program (RHTP), meant to offset a portion of Medicaid cuts in the bill. Under the program, states may apply for federal funding to support rural hospitals and healthcare access. Funds can be used to support rural hospital operations, infrastructure upgrades, and service delivery innovations, with the aim of sustaining access to care in underserved areas.

FY26 - FDA Appropriations Landscape

The Senate and House Appropriations Committees have advanced differing FY26 Agriculture–FDA spending bills, each with significant implications for CGT oversight and development.

The Senate-passed bill maintains FDA's overall funding at $7 billion, with $3.4 billion from user fees, level with FY25. The Committee report commends FDA's prioritization of CGTs and directs the agency to continue modernizing its approach. Other key passages include:

Clinical Holds and Communication: The Committee acknowledges clinical holds as an appropriate regulatory mechanism but urges FDA to apply them judiciously and work with sponsors to address issues without unnecessarily impeding critical research. The Committee calls for clear communication of rationale when applying clinical holds, addressing industry concerns that ASGCT has previously raised about predictability and transparency in CGT oversight.
Rare Disease Innovation Hub: The Committee requests that FDA’s Rare Disease Innovation Hub continue developing draft guidance on evidentiary standards for rare diseases and submit a report to Congress. The Society supports regulatory innovation that enables faster patient access to newly approved CGTs.
Advanced Manufacturing Focus: The Committee encourages FDA to continue facilitating advanced manufacturing technologies and requests a plan for coordinating with external experts on industry standards and technical challenges. ASGCT has similarly urged FDA to provide clear guidance and collaborative frameworks to accelerate adoption of advanced manufacturing as a means to expand patient access and reduce costs.
Domestic Manufacturing and Supply Chain Resiliency: The Committee supports FDA's work to promote domestic manufacturing of drugs and biological products to bolster supply chain resiliency, and to expand and relocate drug manufacturing to the US. ASGCT has previously emphasized that strengthening domestic supply chains is important to mitigate risks from global manufacturing dependencies.
Expert Panel Transparency: The Committee expressed concern with panels that FDA recently deployed to evaluate scientific matters including CGTs, talc, and infant formula. The Committee emphasized that FDA should prioritize engagements that allow public participation or provide for public comment. ASGCT concurs that public input, in particular the patient voice, is critical for regulators to consider.

The House bill provides $6.8 billion for FDA - $200 million below the Senate level - and has not yet received a floor vote. The House is expected to take up their appropriations bills in September when they return from recess. The House report takes a notably different approach to CGT modernization:

Clinical Trial Design Innovation: The Committee calls for FDA to modernize its approach by considering innovative clinical trial designs that better reflect the range of patient populations. The report encourages FDA to establish modern pathways that embrace novel endpoints for safety and efficacy assessment. ASGCT has previously supported these efforts, emphasizing the importance of decentralized clinical trials, real-world data and real-world evidence, and natural history studies.
Risk-Benefit Assessment: The Committee encourages enhanced communication between FDA centers and the patient community regarding risk tolerance. For conditions where CGTs offer significant clinical outcomes, including type 1 diabetes, the Committee suggests that a broader risk-benefit assessment may be warranted..
Accelerated Development Pathways: The Committee recommends greater utilization of accelerated development pathways. The Society has reiterated to FDA that CGTs are uniquely suited for accelerated approval pathways due to their large treatment effect and limited patient populations. In parallel, the Committee underscores that advanced manufacturing technologies can help speed time-to-market for such therapies, improve quality, and address shortages.
Proactive Field Engagement: The report emphasizes the need for clear communication and proactive engagement with sponsors to address regulatory concerns efficiently, ensuring that clinical holds are applied judiciously and with transparent rationale.

NIH Funding Developments

The Senate Appropriations Committee recently recommended a $400 million increase for NIH on a bipartisan basis, despite the President's budget requesting a 40% cut. The House Appropriations Committee is expected to discuss their proposed NIH budget in early September.

The Senate Labor-HHS appropriations bill maintains all 27 NIH institutes and centers in their current configuration The accompanying report underscores strong bipartisan support for advancing cell and gene therapies across research, access, and manufacturing. It provides $33 million for the C.W. Bill Young Cell Transplantation Program to expand donor diversity and patient access to lifesaving transplants, and directs NIH to include updates on newly approved CGTs in its FY27 budget justification. The Committee highlights the promise of advanced therapies for cancers and inherited blood disorders, and calls for a dedicated NIH workshop - together with FDA and stakeholders - on their long-term safety and efficacy. In addition, the report urges ARPA–H to continue supporting its EMBODY program to address technical and manufacturing barriers that currently limit scalability for engineered cell therapies.

The Senate report language on NIH funding also preserves the current facilities and administrative (F&A) cost reimbursement system for the time being. It acknowledges the Joint Associations Group (JAG) effort to craft a replacement for F&A, dubbed the Financial Accountability in Research (FAIR) model, and notes that appropriators plan to review the proposal further. Feedback from the research community during JAG community townhalls identified several potential implementation challenges. Participants shared concerns about administrative burden (particularly for smaller institutions) and timeline feasibility, questions about treatment of multi-PI awards and shared facilities, and technical concerns around data collection. ASGCT will continue to monitor this JAG effort and provide updates to our members.

Finally, a new Executive Order directs agencies to review all new and existing grants to ensure they align with the Administration’s policy priorities and national interests. The order also empowers agencies to terminate grants more easily and requires justification for drawing down federal funds. Implementation details for are still unclear, but ASGCT will share updates as we learn more.

Learn About Updates to the Newborn Screening System

The federal Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) was disbanded earlier this year, and there have been several recent updates on the path forward. First, the Health Resources and Services Administration (HRSA) has issued requests for comment on adding Duchenne muscular dystrophy (DMD) and metachromatic leukodystrophy (MLD) to the Recommended Uniform Screening Panel (RUSP) without the ACHDNC. Second, EveryLife Foundation for Rare Diseases is hosting a virtual Newborn Screening Evidence Review Workshop on September 12. Finally, a recent House Energy & Commerce subcommittee held a hearing on the Newborn Screening Saves Lives Reauthorization Act, with bipartisan support for early screening access.

Events + Resources

Watch New Community Quick Take: Why Manufacturing Matters

Watch Drs. P.J. Brooks, Steven Gray, and Angelica Meyer in the latest Community Quick Take. They explain the vital role of plasmids, the importance of quality checks, and the typical timelines it takes to make a batch of AAV vectors for use in clinical trials. Content was developed in partnership with the Bespoke Gene Therapy Consortium (BGTC), which works to remove bottlenecks during the manufacturing and regulatory approval processes during development of gene therapies.

Attend FDA + Duke Margolis Workshop on CDRP Program

On September 10, the Duke-Margolis Institute for Health Policy is hosting a virtual workshop with FDA to examine challenges and strategies for faster chemistry, manufacturing, and controls (CMC) development. Industry leaders and FDA regulators will share best practices and insights from their CMC Development and Readiness (CDRP) pilot program experience. Register here

In Brief: Policy Updates

Citeline Q2 Landscape Report: The recently released 2025 quarterly landscape report highlights FDA’s approval of Zevaskyn for recessive dystrophic epidermolysis bullosa (RDEB) and mNexspike for COVID-19. Notably, 64% of new gene therapy trials initiated were for oncology indications, the highest proportion in the past year.
Commissioner’s NPV FAQ: FDA published a new FAQ for the Commissioner’s National Priority Review Voucher Program, outlining eligibility criteria, submission procedures, and key timelines. FDA is accepting statements of interest for participation in the pilot program.
User Fee Announcements: FDA announced next year’s user fee rates for prescription drugs with an 8.6% increase for applications and 9.5% increase in the annual program fee. Priority review voucher fees have not yet been published.
OTP Public Listening Meeting: FDA’s Office of Therapeutic Products will hold a public listening session on September 18 to gather input from CGT sponsors on leveraging internal and public knowledge, including CMC, nonclinical, and clinical data, across therapeutic contexts. Written comments are due by October 17.
FDA PreCheck Program: FDA launched the PreCheck program for companies establishing or expanding US drug manufacturing facilities. The program offers additional regulatory engagement on facility readiness and CMC submissions. FDA will also host a public meeting September 30 to seek feedback. Written comments are due October 30.
CGT Access Model: CMS announced that the Cell and Gene Therapy Access Model will include 33 states, the District of Columbia, and Puerto Rico, offering outcomes‑based Medicaid coverage for sickle cell gene therapies. Roughly 84% of Medicaid beneficiaries with sickle cell disease are represented in those states.
IPPS FY26 Final Rule: The FY26 IPPS final rule maintains current policies for MS-DRG 018, continuing to exclude clinical trial cases from relative weight calculations, and retains the New Technology Add-On Payment (NTAP) framework.
CBO Report on NIH Funding: A new report from the Congressional Budget Office found that a permanent 10% cut to NIH research funding would reduce the number of new drugs reaching the market by ~4.5% over a decade, or about two fewer drugs per year.
Rare Disease Funding Opportunity: FDA posted two new funding announcements aimed at accelerating research in rare diseases. One supports clinical trials evaluating efficacy and safety for new indications or labeling changes, while the other seeks innovative natural history studies that address unmet needs in rare conditions.
BIOSECURE Revived: The BIOSECURE Act has been filed as an amendment to the National Defense Authorization Act. It would restrict government contractors and grant recipients from procuring equipment and services from “companies of concern,” a designation that would be made by OMB.
MAHA Report Draft: A draft of an expected MAHA report leaked before release this week. The draft, which may undergo further edits, signals changes for CGT developers and patient access. The draft places stronger emphasis on integrating real-world evidence alongside clinical trial data, with heightened scrutiny of trial design and long-term follow-up. It also signals potential adjustments in Medicaid reimbursement approaches and outlines priorities for directing research funding toward innovative therapies.

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