Policy Summit

Policy Summit Speakers Explore Boundless Opportunities, Significant Challenges

Chiagbanwe (ChiChi) Enwere, MPH - October 13, 2022

The need for proactive communication was repeatedly stressed during the fourth annual Policy Summit. Speakers said this could decrease issues that might otherwise stall development or impede access.

ASGCT welcomed nearly 300 guests to this year’s hybrid Policy Summit to discuss the issues that impact the regulatory and legislative aspects of cell and gene therapy. You can watch all of the Policy Summit sessions on demand through October 27 – and if you aren’t yet registered, there’s still time to do so!

Looking to the Future of Development for CGTs

The Policy Summit kicked off with a discussion on varied approaches to support innovative cell and gene therapy (CGT) development. Wilson Bryan, MD, from FDA’s Office of Tissues and Advanced Therapies (OTAT), set the tone for a discussion around CGT development possibilities with a presentation on the Regenerative Medicine Advanced Therapy (RMAT) designation. Meanwhile Health Canada’s Emily Griffiths, PhD, reflected on Canada's oversight of CGT and the regulatory innovations supporting current and future gene and cell therapy advances, highlighting their accelerated review process for unmet medical needs or life-threatening diseases. BridgeBio’s Adora Ndu, PharmD, JD, stressed the need for clarity on the use of reasonably likely surrogate endpoints and primary disease activity biomarkers in CGT development. Finally, Courtney Silverthorn, PhD, from the Foundation for the National Institutes of Health, discussed the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC), which aims to promote a platform approach to develop solutions for AAV-based gene therapies. Dr. Silverthorn explained that the AMP BGTC can help the CGT field better understand AAV biology and standardize manufacturing leading into the clinical trial process, which can in turn feed into the regulatory framework.

The next panel delved into PDUFA VII reauthorization, which was signed into law as part of the broader FDA user fee package on October 1. Dr. Bryan participated in this panel as well, joined by Accumulus Synergy’s Khushboo Sharma and BioMarin’s Brad Glasscock, PharmD, both of whom were involved in negotiating last year’s FDA commitment letter on the industry side. As Ms. Sharma outlined, PDUFA VII will play a role in advancing science-based regulatory programs. It aims to strengthen review fundamentals, enhance accountability and transparency, and ensure stable growth for the program through the use of digital tech, remote inspections, and decentralized clinical trials. Critically, PDUFA VII aims to advance the next wave of biologics therapies, the gene and therapy program, and the manufacturing portfolio for new drugs through significantly increased resources for staffing within the Center for Biologics Evaluation and Research (CBER), much of which will be devoted to OTAT. As Dr. Bryan noted, workforce challenges have been one of the key roadblocks in managing a hugely increased number of Investigational New Drug (IND) applications.

ASGCT Board Member Rayne Rouce, MD, a researcher and clinician at Texas Children’s Hospital and Baylor College of Medicine, moderated an extremely timely panel on the need for diversity in CGT, addressing the needs of underrepresented populations and how developers and clinical trialists can better include them at all stages of the therapeutic development process. FDA’s Asha Das, MD, pointed to the Agency’s recent draft guidance on diversity in clinical trials, stating that certain racial groups with a disproportional disease burden are underrepresented in research and clinical trials. She also noted that existing health inequities may increase as personalized and precision medicine advance. Fellow panelists Alex Helman, PhD, from the National Academy of Sciences, Engineering, and Medicine, and Aisling McDonough, from the Office of Subcommittee Chairwoman Anna G. Eshoo, examined some of the potential policy and institutional solutions to improve representation. Dr. Rouce stressed the importance of intentionality and open communication with affected communities from the beginning of any development program.

CGT Coverage and Reimbursement

This year's keynote speaker was John Coster, PhD, from CMS’ Center for Medicaid and CHIP Services, who explained CMS' role in ensuring that states implement Medicaid statutes and regulations to ensure patient access. He dived into Medicaid coverage for CGTs, noting the critical distinction between state plans that cover drugs under a fee-for-service (FFS) model versus the Medicaid managed care model used by some states. He also provided an overview of the Medicaid rebate system and explained how value-based purchasing (VBP) might be used by states to manage therapies with high upfront costs and an expectation for durable effect. He pointed out that it is helpful to have clear and early communication with sponsors to keep CMS and state Medicaid programs informed of possibilities in the pipeline, so that they can be prepared to respond at the point of approval.

The conversation continued with a panel discussion on coverage and reimbursement for cell and gene therapies, exploring ways to manage high-cost, high-value therapies. This engaging conversation allowed participants to understand better how public and private payers can address upfront costs while protecting access to cell and gene therapies. Mark Trusheim of the Tufts NEWDIGS Project summarized the range of tools available to payers and the challenges that might come with each approach. Terry Cothran of the Oklahoma Health Care Authority provided a state Medicaid perspective on budget challenges and opportunities for VBP; Orchard Therapeutics’ Andrew Olaye, PhD, provided context on the process involved in establishing cost and access for high-priced therapies in Europe. During these conversations, Kollet Koulianos of the National Hemophilia Foundation reflected on the realities of working with rare, high-cost populations such as hemophilia, sharing examples of self-insured payers carving out specialty drugs or entire diagnoses for coverage exclusion. The panel discussed opportunities to include the patient voice in valuation and reimbursement decisions and agreed that engagement with state Medicaid offices remains essential to drive the conversation on the value of these products.

Regulatory Essentials

The Policy Summit also featured reviews of a few of today’s most widely debated regulatory issues. In a session on accelerated approval, panelists discussed the need to bring patients, payers, advocacy groups, and product manufacturers together to find sustainable solutions. BGR Group’s Remy Brim, PhD, helped the audience understand recent conversations in Congress on reforming the pathway, following years of FDA approvals in rare diseases and oncology and with the spotlight of FDA’s user fee renewal elevating the issue. She noted that accelerated approval has been wrapped up in broader debates between efficacy data and speed of approval, and in discussions of who can access therapies after approval related to pricing. Matt Salo, formerly with the National Association of Medicaid Directors, relayed the kinds of issues states are considering in providing services to the roughly 90 million Americans covered by state Medicaid programs. Sarepta’s Diane Berry, PhD, and Annie Kennedy of the EveryLife Foundation for Rare Diseases, meanwhile, discussed the role of accelerated approval in catalyzing investment in rare genetic diseases and the ways in which the pathway might be improved and preserved.

The Policy Summit wrapped up with a conversation on CMC applications and hurdles for CGTs. FDA’s Zhaohui Ye, PhD, opened with a presentation on the Agency’s current CMC expectations for gene therapy products incorporating genome editing, followed by a talk from Jan Thirkettle, PhD, of Transine Therapeutics to summarize ASGCT’s recommendations to FDA on CMC considerations, presented at the Society’s 2021 FDA Liaison Meeting. Stacey Frisk of Sarepta Therapeutics next took up the patient and policy considerations of applying benefit/risk assessments to CMC; during her presentation, Ms. Frisk drew attention to Dr. Silverthorn’s earlier presentation on the AMP Bespoke Gene Therapy Consortium, highlighting the fact that there is no business case for commercializing drugs for many rare diseases. Finally, Novo Nordisk’s Andrew Chang, PhD, focused in on comparability for Advanced Therapy Medicinal Products (ATMPs) that undergo changes in their manufacturing process.  

Key Takeaways

While Policy Summit presenters tackled a number of issues over the 1.5-day event, several common themes emerged. First was the need for a wide range of stakeholders to be informed and involved throughout the development process. Speakers repeatedly stressed that proactive communication can head off issues that might otherwise stall development or impede access. Regulators, patients, researchers, policymakers, manufacturers, and payers each have a key role to play in ensuring that the product pipeline will eventually result in safe, effective, accessible therapies. Within each of those groups, remarkable diversity exists that should be recognized and given space to contribute.

The second key takeaway was that the CGT field is simultaneously faced with near-boundless opportunities and significant challenges. In order to harness areas of positive momentum and resolve dilemmas in development and access, creative solutions are required. Panelists illuminated that it is often more productive to view other stakeholders as partners with many shared values, rather than as adversaries—and to seek innovative solutions on that basis. As the field continues to grow and develop, a collective effort will be needed to ensure these transformative products can achieve their full potential.

Ready to watch the full discussions summarized here? Catch every session on-demand through October 27 if you registered, or register through that date to get access.

Ms. Enwere is ASGCT's manager of regulatory affairs.

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