The Advocate

October 2024: Volume 4, Issue 10

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In This Issue:

Apply for the Congressional Policy Fellowship!
Flexibility, Enrollment Approaches Among ASGCT Concerns on FDA's Diversity Action Plans
Policy Summit Experts Address Legislative, Regulatory Impacts to CGT
Congress Extends Key Programs and FY24 Federal Funding Through Dec. 20
In Response to Congressional RFI, ASGCT Emphasizes Regulatory Consistency
Society Feedback Reflected in Final MDRP Rule
Events
Policy News

ASGCT Advocacy

Apply for the Congressional Policy Fellowship!

We're now accepting applications for the Congressional Policy Fellowship! ASGCT’s first Congressional Policy Fellow, Adriana Bankston, PhD, started her role in September. ASGCT’s Congressional Policy Fellows are hosted in Washington, D.C. in partnership with the American Association for the Advancement of Science (AAAS) Science & Technology Fellowship Program. Fellows choose their fellowship placement independently, performing high-quality scientific policy work for their office. Dr. Bankston will spend her fellowship year in the office of U.S. Representative Bill Foster (D-IL). Dr. Bankston brings a wealth of experience as an accomplished scientist and a passionate advocate for STEM workforce development and biomedical research.

Legislative policy and advocacy are critical to advance the CGT field. Congress plays a key role in biomedical innovation, federal research funding, and ensuring patient access to life-saving therapies. ASGCT's Congressional Policy Fellowship program offers an exciting opportunity for scientists to apply their technical knowledge to the legislative process. We encourage members to apply for the 2025-2026 fellowship; applications are open now through Feb. 24, 2025.

Flexibility, Enrollment Approaches Among ASGCT Concerns on FDA's Diversity Action Plans

ASGCT has submitted comments on FDA’s updated Diversity Action Plans draft guidance, which aims to improve enrollment of underrepresented populations in clinical studies. ASGCT previously provided feedback on the initial draft in 2022 and continues to support the congressional intent behind this guidance. However, gene therapies face unique development challenges compared to traditional small molecule drugs, and the Society has expressed some concern about the guidance’s implementation.

Key points from ASGCT's comments include:

Enforcement Mechanisms: ASGCT seeks clarification on FDA's expectations when sponsors cannot fully meet enrollment goals despite good-faith efforts. Comments emphasized the need for flexibility while prioritizing diversity in clinical study.
Modern Enrollment Approaches: The Society recommends guidance on leveraging digital health tools, decentralized clinical trials, and Real-World Evidence (RWE) to address enrollment challenges. ASGCT highlighted the need for advanced planning around socioeconomic factors that can impede enrollment, particularly in gene and cell therapy trials.
Cultural Competency and Global Recruitment: The comments stress the importance of addressing cultural and systemic barriers through targeted community engagement and education. ASGCT also requested clarity on evaluating global efforts to recruit diverse patients, considering international data protection laws.
Rare Disease Considerations: ASGCT emphasizes the need for clearer criteria for Diversity Action Plan waivers in the context of rare diseases with limited patient populations, and requested examples and clarification on justifiable circumstances for Action Plan waivers.

ASGCT recognizes the challenges of advancing equitable access to CGTs while also addressing the unique challenges in clinical trial diversity for these innovative treatments. We encourage all stakeholders in the CGT field to review ASGCT's full comments for a better understanding of these critical issues and their potential impact on future clinical trials and development.

Policy Summit Experts Address Legislative, Regulatory Impacts to CGT

ASGCT hosted its annual Policy Summit in Washington, D.C. on Sept.23-24, bringing together a diverse group of experts and stakeholders in the CGT field. The event featured an impressive lineup of speakers who addressed critical challenges in CGT development.

The Summit focused on three key themes: payment policy, regulatory, and clinical considerations. From a regulatory standpoint, experts discussed the global convergence of regulatory standards and its potential to streamline approval pathways across countries, emphasizing the need for harmonization to reduce uncertainties in CGT development. FDA officials also discussed the new Rare Disease Innovation Hub. On the payment policy side, the conversation centered on emerging payment models, particularly value-based arrangements and the challenges of Medicaid reforms. These discussions highlighted how policies, such as the CMMI CGT Access Model, could impact access of CGTs, especially for rare diseases. The clinical aspect explored the evolving landscape of risk-benefit analysis in CGT development, including the use of surrogate endpoints, biomarkers, and real-world evidence in clinical trials. Attendees also delved into the challenges of developing therapies for ultra-rare conditions and insider views from different types of investors on the funding landscape. Overall, the Policy Summit underscored the critical role of cross-sector collaboration in advancing the CGT field and addressed the unique challenges of bringing these transformative therapies to patients.

Professionals spanning the field – including industry leaders, regulatory bodies, patient advocates, venture capitalists, and government relations experts – engaged in robust discussions, and networked throughout this dynamic event. For registered attendees who may have missed any sessions or wish to revisit the content, we encourage you to catch up on demand. If you haven’t registered, make sure to do so before the on-demand period closes on Oct. 24.

Congress Extends Key Programs and FY24 Federal Funding Through Dec. 20

Congress passed a Continuing Resolution (CR) to extend Fiscal Year 2024 (FY24) federal funding through Dec. 20, temporarily avoiding a government shutdown. Without that extension, FY24 funding for the federal government would have run out on September 30. The stopgap CR ensures that federal agencies, including those critical to advancing gene and cell therapies, remain funded while lawmakers continue negotiating full-year appropriations for FY25.

Notably, the CR includes a critical extension of the Rare Pediatric Disease Priority Review Voucher (PRV) program, which was set to expire Sept. 30. The PRV program, established in 2012, incentivizes the development of therapies for rare pediatric diseases by awarding a voucher to sponsors who develop products for these conditions. This voucher can be used to expedite FDA review of the recipient’s future drug application or sold to another company, potentially worth hundreds of millions of dollars.

The program is particularly important for gene and cell therapy development in rare diseases, as it provides a significant financial incentive for companies to invest in these often challenging and costly areas of research. Many rare pediatric diseases lack effective treatments, and the PRV program has encouraged innovation in this space.

Under the CR, the PRV program has been extended until Dec. 20. However, its long-term future remains under discussion. Two bills are currently under consideration in the Senate that could further extend the program:

The House-passed Give Kids a Chance Act (H.R. 3433), which proposes to extend the program through Sept. 30, 2029.
A Senate companion bill (S. 4583) to the Creating Hope Reauthorization Act, which would extend the program through Sept. 30, 2030.

We can expect renewed discussions on the PRV program's future in December. Lawmakers could include a longer-term extension in the final FY25 appropriations package, include it in a separate end-of-year omnibus, or pass standalone legislation. The debate will likely center around the program's effectiveness in stimulating rare disease drug development and its overall impact on the FDA's review process. ASGCT will continue to monitor these developments closely and advocate for policies that support the advancement of CGTs for rare pediatric diseases.

In Response to Congressional RFI, ASGCT Emphasizes Regulatory Consistency

ASGCT has submitted its response to a congressional Request for Information (RFI) on policy solutions to secure and enhance domestic medical supply chains. In the letter, ASGCT emphasized the unique regulatory and manufacturing challenges faced by the CGT field, highlighting the need for improved regulatory consistency, advanced manufacturing technologies, and more flexible clinical trial designs. ASGCT also encouraged Congress to focus on innovative solutions, such as public-private partnerships, to facilitate the adoption of advanced manufacturing techniques and support the development of transformative CGT products. These measures are critical to meeting patient needs and advancing the CGT pipeline, which currently includes over 4,000 gene therapies in development.

ASGCT appreciates members of Congress for their leadership and continued support of policies that strengthen biomedical innovation in the United States. The Society looks forward to working with the federal government to ensure that policies supporting regulatory modernization, supply chain resilience, and efficient manufacturing are advanced to secure the future of biomedical research and patient access to groundbreaking CGTs.

Society Feedback Reflected in Final MDRP Rule

CMS has released the final rule for the Medicaid Drug Rebate Program (MDRP), introducing several key updates for drug manufacturers. The rule broadens the definition of covered outpatient drugs, now including more physician-administered drugs, which are subject to rebate requirements. Additionally, the final rule reinforces state obligations to report National Drug Code (NDC) information for these drugs, impacting how rebates are calculated. For CGT manufacturers, this means an increased need for precise reporting and rebate compliance.

CMS opted not to include a proposed "stacking" provision, which would have required manufacturers to combine multiple discounts and rebates when determining the "best price" for Medicaid rebates. ASGCT advocated against the proposed stacking provision in previous comments, arguing that it would impose significant complexity, particularly for smaller manufacturers of cell and gene therapies. By requiring manufacturers to account for various pricing adjustments across the supply chain, the rule could have distorted the true "best price" and created additional reporting burdens. ASGCT also advocated against a proposed drug price verification survey for similar reasons, noting that it would have introduced unnecessary administrative hurdles without providing CMS with data essential to managing the rebate program. That survey was dropped from the final rule. The Society appreciates CMS for considering stakeholders’ feedback and is pleased the final rule does not introduce unnecessary administrative hurdles for CGT manufacturers.

Events

This Fall: 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:

Advancing Gene and Cell Therapies for Cancer: Oct. 16-17 | Philadelphia + Virtually
Breakthroughs in Muscular Dystrophy: Nov. 19-20 | Chicago + Virtually

Register for Empowering Patients 2025: A Cell and Gene Therapies Summit

Coming next spring: A new, free, virtual event hosted by ASGCT on March 12-13, 2025. This event will be two half-days of educational material geared toward foundation leaders, patients, and caregivers to provide timely information to support their CGT journey. It will feature educational sessions and engaging discussions on the CGT landscape, exploring the unique challenges impacting patient communities at various stages of the CGT pipeline. Register today!

New FDA-Partnered Workshop Will Focus on AAV Class Considerations

Registration is open for this ASGCT and FDA-hosted virtual scientific workshop that will be held over two half days on Jan. 22 and 23, 2025. This free, public event will explore key immunology-related topics that have implications across the class of AAV gene therapies, including immune responses, antibody testing, and redosing strategies. The workshop will provide valuable insights for professionals in both academia and industry as it addresses pressing challenges in AAV gene therapies and new approaches.

Watch + Register for CBER Patient and Care Partner Listening Meetings

FDA is holding listening sessions to engage with patients and caregivers on their experiences, perspectives, and informational needs regarding the short-term and long-term risks of approved gene therapies. These meetings will help inform patient-centered protocols for long-term post-market studies of these transformative treatments. Watch the recording of the first meeting and register for the second meeting on Dec. 4.

NORD Breakthrough Summit to Focus on Inclusion and Access

The National Organization for Rare Disorders (NORD) is hosting their hybrid Rare Disease and Orphan Products Breakthrough Summit, in Washington, D.C. Oct. 20-22. With a theme of “Equitable Access to Innovation,” this event will feature thought-provoking discussions and actionable strategies on the latest advances in clinical trials, gene therapy, medical devices, AI and patient-focused drug development with an underlying focus on inclusion and equal access for all. Review the full agenda on their website and register!

Oct. 30: FDA Webinar on Finding Your Support Team While Participating in a Clinical Trial

Tune in on Oct. 30 to learn from patients, care partners, and advocates who have direct experience building and managing support networks while navigating and participating in clinical trials.

Nov. 14: FDA Workshop on Integration Site Analysis for Gene Therapies

On Nov. 14, CBER’s Office of Therapeutic Products (OTP) is hosting a virtual scientific public workshop on the clinical use of integration site analysis (ISA) during long term follow-up following administration of gene therapies with integrating viral vectors. In this workshop, FDA will convene a panel of external experts to discuss the risk of insertional mutagenesis and best practices for ISA method design, data analysis, and clinical interpretation. Registration is now open.

Policy News

The National Academies provided new recommendations to the FDA and Congress on the topic of rare disease regulatory policy. The report was called for by Congress in a 2022 law, and addresses topics such as global alignment on evidence-based approaches and the inclusion of pediatric population.
Patient uptake for the newly approved gene therapies for sickle cell disease has been slower than anticipated due to barriers such as limited access to specialized treatment centers and the need for more comprehensive patient education to help individuals fully understand the risks and benefits of these complex treatments.
FDA-approved gene therapies continue to see access issues with full coverage to the labeled indication. Research has found state Medicaid programs are often covering therapies to the clinical trial population, rather than the expanded population identified by FDA.
The HHS has issued a final rule revising research misconduct regulations for the first time in nearly 20 years, requiring institutions receiving U.S. Public Health Service funding to update their policies by April 2026. Key changes clarify definitions of misconduct, address record-keeping responsibilities, and introduce new procedures for handling witness testimony and research records.