The Advocate
June 2024: Volume 4, Issue 7
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In This Issue:
FDA Releases New Draft of Plans to Improve Clinical Trial Enrollment
Listening Session Explores Perspectives on Advisory Committee Reform
Updates on FY25 Appropriations for Agriculture-FDA and Labor-HHS
U.S. Supreme Court Overturns Long-Standing Chevron Doctrine
Events
Policy News
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ASGCT Advocacy
FDA Releases New Draft of Plans to Improve Clinical Trial Enrollment
The Food and Drug Administration (FDA) has released a new draft version of the guidance “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies.” This new draft guidance replaces a version released in 2022, which was not finalized. The document includes broad definitions of diversity and provides clarity on content and timing of these action plans. ASGCT previously commented on the first version of this draft guidance, highlighting key points such as the need for flexibility in approaches for rare diseases, the importance of post-market studies for smaller patient populations, and recommendations for trial enrollment and retention strategies. ASGCT plans to submit a response to this new draft guidance, which has a 90-day comment period. This guidance represents an important step for cell and gene therapy development. The document addresses a crucial aspect of CGT development while keeping focus on safety and health equity, which are particularly important for ensuring timely access to care in the field of cell and gene therapies.
Additionally, last week FDA released a draft guidance titled "Addressing Misinformation About Medical Devices and Prescription Drugs: Questions and Answers," which aims to provide clear recommendations for companies on how to address misinformation about their medical products on the internet or social media platforms. This guidance builds upon a previous draft from June 2014, offering updated insights into FDA's current thinking on voluntary correction of such misinformation. ASGCT does not plan to submit a response to this draft guidance.
Listening Session Explores Perspectives on Advisory Committee Reform
On June 13, 2024, FDA held a public meeting titled "Optimizing FDA's Use and Processes of Advisory Committees." The listening session focused on three main topics: the composition of advisory committees, service as a Special Government Employee (SGE), and public perception and understanding of advisory committees. Key takeaways included calls for enhanced diversity and patient representation, stricter conflict of interest rules, improved communication and transparency, and streamlined processes for SGE recruitment and service.
This meeting is particularly relevant to the CGT community, as advisory committees play a crucial role in evaluating novel therapies. Speakers highlighted the need to include disease-specific experts on committees, especially for rare diseases. Additionally, the emphasis on improving public understanding of the advisory process and enhancing patient involvement could lead to more informed discussions and decisions around innovative treatments. As the CGT field continues to advance, these potential changes to FDA advisory committees may significantly impact how new therapies are evaluated and understood by both experts and the public.
Updates on FY25 Appropriations for Agriculture-FDA and Labor-HHS
As the fiscal year 2025 (FY25) appropriations process unfolds, key bills for Agriculture-FDA and Labor-Health and Human Services (HHS) are in various stages of negotiation. Both bills are critical for the funding and support of cell and gene therapy research, directly impacting agencies like FDA, National Institutes of Health (NIH), and the Advanced Research Projects Agency for Health (ARPA-H).
The Agriculture-FDA funding bill has passed the House Appropriations Committee and is headed for a full floor vote on July 22nd. There have been proposals for significant funding cuts compared to the FY24 enacted levels and the President's FY25 budget request. Specifically, the House Appropriations Committee has proposed reducing FDA's budget by approximately 10%, which could have substantial implications for the agency’s ability to efficiently review and approve new cell and gene therapies. The Senate version passed the full appropriations committee with few cuts than their cameral counterparts’.
In parallel, the Labor-HHS appropriations bill, which funds both the NIH and ARPA-H, has seen contentious debates over proposed budget reductions. The House Appropriation Committee’s draft bill suggests a decrease in NIH funding by 8% from the FY24 enacted level, which is significantly below the President’s FY25 request. ARPA-H, a newer agency dedicated to innovative, high-risk health research, faces potential budget cuts that threaten its nascent programs designed to accelerate breakthroughs in gene and cell therapies. Tandem legislative efforts could also introduce new compliance requirements for NIH-funded research or entirely re-structure the Institutes, potentially complicating the research process for institutions across the country. A full floor vote has not yet been scheduled.
All FY25 appropriations bills are still in the early stages of negotiation, and will likely land somewhere between the House and Senate versions. The appropriations process is critical for researchers as it determines the availability of federal funds and sets the regulatory landscape for the upcoming fiscal year and ASGCT will continue to update.
U.S. Supreme Court Overturns Long-Standing Chevron Doctrine
The recent Supreme Court decision to overturn the Chevron Doctrine marks a significant shift in the landscape of administrative law. Historically, the Chevron Doctrine granted federal agencies broad deference in interpreting ambiguous statutes within their jurisdiction. The overturning of this doctrine will likely lead to increased judicial scrutiny of agency decisions, requiring more explicit statutory authority for regulatory actions. A lack of clear and precise legislative drafting in many cases could result in slower regulatory processes as courts take a more active role in interpreting statutes.
For federal agencies, including FDA, the implications are still being determined. Agencies will need to be more meticulous in justifying their regulatory actions and demonstrating their alignment with statutory mandates. This increased burden may impact the efficiency and predictability of the regulatory environment, which is critical for the development and approval of innovative therapies.
For cell and gene therapies, the overturn of the Chevron Doctrine could lead to increased uncertainty and delays for developers and regulators. These therapies often rely on novel and complex regulatory frameworks that interpret evolving science and technology. Without Chevron deference, CBER's regulatory decisions on cell and gene therapies may be subject to more frequent legal challenges, potentially slowing down the approval process and increasing the burden on developers. It is crucial for stakeholders in the cell and gene therapy field to stay informed about these legal developments and engage in advocacy efforts to ensure that the regulatory environment remains conducive to innovation and patient access.
Events
This Fall: Attend Inaugural Events on Muscular Dystrophy and Cancer Innovation
Don't miss out on two exciting ASGCT events this fall! Explore and dig into advancements in muscular dystrophy and cancer with these new hybrid events. Register today to join leading experts and stay ahead in the field with ASGCT:
Register Now for the 2024 Policy Summit
The 2024 Policy Summit will be September 23-24 at the Westin Washington DC, Downton Hotel, and our agenda is packed! Join your colleagues to tackle some of the hottest policy issues facing the CGT field. Our expert line up of presenters will host panel discussions on:
Find the full agenda online; speakers are being updated as they confirm. We hope you’re able to join us!
Community Quick Take Looks at Patient Access Issues Beyond Approval
AGSCT released a Community Quick Take unit with four short talks touching on critical access issues faced by the cell and gene therapy patient community. Experts in the field discussed the various barriers patients may encounter, such as disease eligibility, financial constraints, provider referrals, and logistical challenges within clinical settings.
This quarterly Community Quick Take emphasizes the complex journey patients face. These barriers will require multistakeholder, collaborative solutions to ensure the right treatments are meeting the right patients in a timely manner. Be sure to watch and share these quick insights to keep up with the current access landscape for CGTs.
Policy News
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Sarepta Therapeutics announced that FDA has granted full approval for their gene therapy, ELEVIDYS (delandistrogene moxeparvovec-rokl) for treating Duchenne muscular dystrophy, previously licensed under accelerated approval.
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FDA announced they will hold a public workshop on best practices for meeting types held under the Prescription Drug User Fee (PDUFA) program. FDA and pharmaceutical industry representatives agreed to this meeting as part of their negotiated PDUFA VII Commitment Letter.
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CMS released the State Request for Applications (RFA) for the Cell and Gene Therapy (CGT) Access Model, inviting states to express their interest in participating in this initiative. This model aims to enhance access to innovative gene therapies for Medicaid beneficiaries, specifically targeting treatments for sickle cell disease.
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FDA’s rare pediatric disease priority review voucher (PRV) program is up for reauthorization this fall. New data from the National Organization for Rare Disorders (NORD) outlines the rare disease populations that have benefited from the PRV program to support this reauthorization.
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The House Energy & Commerce Committee released a proposed framework recommending significant changes in the structure and operations of the NIH. Stakeholders who wish to submit any feedback on the framework or provide additional thoughts, ideas, and suggestions for reform can do so by emailing NIHReform@mail.house.gov by August 16, 2024.
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The Consumer Financial Protection Bureau (CFPB) has proposed a rule to remove medical bills from most credit reports, with the goal of enhancing privacy protections and boosting credit scores. Consumers can submit comments on the rule through August 12, 2024.
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FDA and the Clinical Trials Transformation Initiative (CTTI) have announced a joint workshop on artificial intelligence in drug and biological product development, scheduled to take place on August 13-14, 2024. This workshop will explore guiding principles for the responsible use of AI in developing safe and effective drugs, bringing together innovators and regulators to discuss this cutting-edge topic.