The Advocate

February 2024: Volume 4, Issue 2


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In This Issue:

New CGT Payment Model Could Expand Access to Therapies
FDA Launches Global Pilot for GT Application Review
Events
Policy News

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ASGCT Advocacy


New CGT Payment Model Could Expand Access to Therapies

The Biden-Harris Administration unveiled details regarding the timeline and implementation of the Cell and Gene Therapy (CGT) Access Model. Announced on January 30, the model would enable the Centers for Medicare and Medicaid Services (CMS) to negotiate outcomes-based agreements (OBAs) with manufacturers, with a focus on CGT treatments for sickle cell disease (SCD). Notably, this follows FDA's recent approval of two gene therapies, Casgevy and Lyfgenia, for SCD patients aged 12 and older. SCD disproportionately affects Black Americans and has significant associated healthcare costs for the estimated 100,000 affected individuals in the United States. ASGCT is tracking the initiative closely; if implemented properly, it could help expand access to innovative, life-saving gene therapies.  

The model is structured to involve CMS, states, and manufacturers, and it is voluntary. CMS has indicated that states and select territories can begin submitting their non-binding letters of intent to participate in the model as early as April. In March, CMS will release a request for application, and manufacturers will have the option to participate. Negotiations with manufacturers on outcomes-based agreements are expected to begin in May and go through November, with an expected launch in January 2025. 

As stakeholders navigate the implementation process, ongoing engagement and collaboration will be essential to realize the model's full potential to advance patient care and address unmet medical needs. 

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FDA Launches Global Pilot for GT Application Review

Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER) outlined plans for a pilot program to explore the possibility of a collaborative review process for new gene therapy applications with other global regulators. Marks shared that the Collaboration on Gene Therapies Global Pilot (CoGenT) would initially include regulatory members of the International Council for Harmonisation. The goal of the pilot is to increase global harmonization by increasing efficiency in reviews of gene therapies and reducing delays in bringing these therapies to market. 

Events


Register for #ASGCT2024 Before Rates Increase Next Week

Next Thursday, Feb. 29, is the final day to receive early-bird rates when you register for the hybrid ASGCT 27th Annual Meeting May 7-11 in Baltimore, Maryland. Join your colleagues to discuss and learn about exciting scientific and commercial innovation in the field - we hope to see you in Baltimore or online!  

Government relations and regulatory professionals won’t want to miss our policy focused sessions that will focus on topic like value/outcomes-based payment efforts, companion diagnostics, enabling CMC analytics, decentralized manufacturing, global regulatory convergence, and more.  

Policy News


  • The FDA is seeking industry organizations to join a work group to select a nonvoting industry representative for the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC). They are also accepting direct nominations for nonvoting industry representatives, who can be self-nominated or nominated by an organization.  
  • The Foundation for the National Institutes of Health has released a regulatory playbook to advance AAV gene therapies for rare disease. ASGCT has been a member of the Bespoke Gene Therapy Consortium since 2021 and is excited to see the collaborative efforts towards addressing rare disease drug development. 

  • The House Committee on Education and the Workforce recently released an RFI centered on the 50th Anniversary of ERISA, the Employee Retirement Income Security Act. Several questions in the RFI focus on specialty drugs, and call out cell and gene therapies. ASGCT plans to submit a response. 

  • CMS issued new guidance to Medicare Advantage plans relating to how they implement coverage decisions for benefits and services. The guidance discussed artificial intelligence and hit on important questions of how Medicare Advantage plans cover a wealth of medical services, including inpatient stays and post-acute care.  

  • FDA has extended its call for nominations to join its new Genetic Metabolic Diseases Advisory Committee (GeMDAC) as voting members to March 12. The committee was announced in December 2023.

 

2024

Register for the 27th Annual Meeting

May 7-11, 2024 | Baltimore, MD

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