The Advocate

Volume 3, Issue 3

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March 2023

In This Issue:

Attend Two Annual Meeting Sessions on CMC Challenges
CMMI to Assess Three New Payment Models
Biden's FY24 Budget Request Emphasizes Lowering Drug Costs
Policy News


ASGCT Advocacy

Attend Two Annual Meeting Sessions on CMC Challenges 

As the pipeline of cell and gene therapies (CGT) advances to include commercialization in addition to early and mid-phase development, Chemistry, Manufacturing, and Control (CMC) challenges have emerged as a bottleneck in the IND submission and FDA approval process. At this year’s Annual Meeting, two sessions tackle common CMC challenges through case studies and insights from expert presenters. Attendees should come away with knowledge to put them on the road to success with their clinical programs.  

To kick off the conference on Tuesday May 16, an interactive workshop, CMC Development Challenges and How to Avoid Them, will present best practices and case studies from FDA and industry experts. Following an extended Q&A period, attendees will be presented with hypothetical scenarios to discuss courses of action based on what they learned. In-person attendees will split into breakout groups to consider one or more hypothetical scenarios and suggest possible courses of action based on what they learned from the presenters. At the close of the workshop, all attendees will reconvene to hear ideas from each group and help illuminate key themes. 

Later that same day, attendees can attend the Comparability Challenges for CMC symposium. This session will present attendees with information they can use to make timely, informed decisions throughout the development cycle to help them avoid or overcome comparability challenges. Speakers will discuss: 

  • Overview of comparability for CGT products: Considerations for developers 

  • FDA’s assessment of CGT manufacturing changes and comparability data 

  • Case study on comparability issues for cell therapies: Atara's experience 

  • Case study on comparability issues for AAV products: Solid Biosciences’ experience shifting from HSV to transfection platform 

Join your colleagues and register today for the Annual Meeting! An online program is being updated as speakers and sessions are confirmed, so be sure to check back for the most current information.

CMMI to Assess Three New Payment Models

Last month, the Society shared initial information on one of the three new payment models announced by the Department of Health and Human Services (HHS). These models will be tested by the Centers for Medicare and Medicaid Innovation (CMMI) to assess how to best ensure patient access to high-cost prescription drugs. This blog post goes into further detail about each model and its impact on the CGT field.  

The Cell and Gene Therapy Access Model will allow for single point negotiation of outcomes-based arrangements through CMS. Through the model, state Medicaid agencies would assign CMS to coordinate and administer multi-state outcomes-based agreements with manufacturers for certain cell and gene therapies. Rather than 50 different negotiations between states and manufacturers, this approach could give states the option to cede to CMS the authority to negotiate outcomes-based arrangements. Through this model, CMS would also be responsible for reconciling the financial and clinical outcomes of the outcome-based agreements.   

The Accelerating Clinical Evidence Model was also selected for demonstration by HHS Secretary Xavier Becerra. Through this proposal, CMS, in consultation with FDA, is considering developing payment methods for drugs approved through accelerated approval to “encourage manufacturers to complete confirmatory trials,” suggesting differential payment rates for drugs approved through this pathway. Accelerated approval allows FDA to make treatments available earlier based on a surrogate endpoint if they address an unmet medical need for a serious condition, contingent on post-marketing trials that demonstrate the drug offers a clinical benefit and an improved patient outcome. Notably, the 2022 omnibus package included reforms to the accelerated approval pathway that requires greater reporting on post-marketing study progress as well as a refined withdraw pathway. ASGCT is concerned that this approach by CMMI could harm the integrity of the accelerated approval pathway and slow access to the next generation of gene and cell therapies.   

ASGCT President Hans-Peter Kiem, MD, PhD, said: “Many gene therapies have the potential to be approved through the accelerated pathway, as their outcomes may not be possible to assess within the duration of a traditional clinical trial. ASGCT believes post-market surveillance is necessary to ensure approved products remain safe and efficacious. However, efforts to ensure confirmatory trials should not have the unintended consequence of impeding the development of new therapies and patient access.” 

The third modeld, the Medicare $2 Drug List, would select high-value generic drugs to be capped at a max co-payment of $2 for a month's supply for Part D plans. In return, CMS would highlight those plans during open enrollment.  

The report also identified areas for additional research on which CMMI plans to seek feedback. Most relevant to the regenerative medicine field, CMMI is directed to look into alternative payment models for CGT (such as bundled payments) to replace traditional fee-for-service billing during extended care episodes associated with CGT. The report suggests, “a bundled payment approach may increase manufacturer competition, which could lead to reduced manufacturer prices.” ASGCT will be watching for specifics on this potential approach to be released. The additional research section also discussed biosimilar adoption and price transparency. 

Information on these three models, along with the piece on modernizing the fee-for-service approach, is still in the preliminary stages and there are few details about the timeline, implementation, and exemptions from each of these models. The Society's staff will monitor the roll out of these models and share more detail as it becomes available.    

Biden's FY24 Budget Request Emphasizes Lowering Drug Costs 

Last week, the president’s fiscal year 2024 budget request was released detailing the Administration’s priorities for the coming year. This $6.9 trillion budget is just the beginning of federal negotiations over the final appropriations package.  

Of note, this budget request would extend the Inflation Reduction Act’s (IRA) inflation rebates to commercial markets and expands the drugs that will now be subject to government negotiations. Following the IRA expansion, the report includes a proposal that would allow Medicare to double the number of drugs they negotiate beginning in 2026. The request also proposes shortening the negotiation exception timeline to five years. ASGCT will continue to watch as this budget proposal moves through Congress.  


Save the Date for the 2023 Policy Summit 

Mark your calendars! The 2023 Policy Summit has been confirmed for September 18-19 at the Renaissance Washington DC, Downtown Hotel. Be sure to check back for an updated agenda this spring. In the meantime, catch up with recaps of previous Policy Summits

Register Now for the Annual Meeting

Register now for the 26th Annual Meeting, to be held again as a hybrid event from May 16 – 20 in Los Angeles, CA, and virtually. Join your colleagues to discuss and learn about exciting scientific and commercial innovation in the field. Registration and hotel blocks are now open - we hope to see you in LA or online!  

New: Join us on Thursday, May 18 for an engaging conversation with high-level regulators from the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and FDA. During this town hall-style symposium, PDMA's Yoshiaki Maruyama, PhD, and FDA's Peter Marks, MD, PhD, will address key issues in the field including: 

  • Regulatory challenges in the development of gene and cell therapies  

  • Current outlook for clinical development in 2023 and beyond 

  • Guidance documents, new policies, and convergence efforts between regulatory bodies 

Policy News

  • CBER’s Office of Therapeutic Products (formerly the Office of Tissues and Advanced Therapies [OTAT]) will hold a public listening session, Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products, on April 27 to solicit input on methods and approaches (e.g., use of real-world evidence and registries) for capturing post-approval safety and efficacy data. 

  • As a follow up to the September 2022 virtual town hall, CBER's Office of Therapeutic Products will hold an event on gene therapy CMC on April 25. Register now to hear the Agency’s perspective on late-stage clinical studies. 

  • Last month CBER Director Dr. Peter Marks announced a new pilot to support and accelerate rare disease drug development, similar to Operation Warp Speed. Dr. Marks also noted the Agency would like to see a CGT review approach in line with Project Orbis to pool resources and expertise from other regulators to expand approvals to other countries.  

  • A new publication from the American Society for Hematology and ASGCT Board Member Rayne Rouce, MD, examines the influence of household poverty and neighborhood on access and outcomes of young patients treated with certain commercial CAR T products.  

  • Hear from FDA, patient advocates, and industry professionals during a free ASGCT Lunch and Learn later this month, Roadblocks to Developing a Treatment. This session will explore some of the areas where treatments can get stopped while en route to an approval, as well as program halts, regulatory holds, safety and patient enrollment issues, and off-target effects in the pre-clinical setting. 

  • The Alliance for a Stronger FDA will hold a series of six budget webinars to explain FDA’s request within the fiscal year 2024 budget. The April 4 webinar will host Dr. Peter Marks to discuss CBER’s specific requests within the proposed budget. Registration is free and open now. 

  • The Advisory Committee on Heritable Disorders in Newborns and Children met last month and will require additional information before moving Duchenne muscular dystrophy to the next step of inclusion in the Recommended Uniform Screening Process (RUSP). Krabbe disease also failed to be added to the RUSP.  



26th Annual Meeting

May 16-20 | Los Angeles, CA

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