Catch Policy & Advocacy-Focused Sessions at #ASGCT23
Caitlin McCombs, MBA - March 03, 2023
If you're interested in policy and advocacy, here are some sessions not to miss at the ASGCT Annual Meeting. All times are in PT.
View speakers and more details on each session's agenda page.
Tuesday, May 16 | 8 a.m. - 12 p.m.
This workshop will present attendees with information they can use to make timely, informed decisions throughout the development cycle to help them avoid or overcome common CMC challenges.
*** Interactive Portion: Following level-setting presentations, 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. Finally, discussion leaders will reconvene to share ideas from each group and help illuminate key themes.
Tuesday, May 16 | 1:30 -3:15 p.m.
This session will provide a CMC comparability playbook that can be utilized by CGT developers to support the incorporation of manufacturing changes throughout the stages of drug product development. Speakers will focus on:
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Overview of comparability for CGT products: Considerations for developers
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FDA’s assessment of comparability data in CGT
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Case study on comparability issues for cell therapies: Atara's experience
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Case study on comparability issues for AAV products: Solid Biosciences’ experience
Tuesday, May 16 | 1:30 – 3:15 p.m.
This symposium will first provide an overview of the core ethical questions at play, then engage a series of stakeholders to provide their views on the U.S. healthcare system at large, unique development and pricing considerations for CGTs, and the question of privilege in patient access. Speakers will focus on:
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Setting the stage: Outlining the ethical dilemmas involved in pricing and access
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ICER’s valuation framework: Assessing cell and gene therapies
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Pricing valuation of CGTs from the developer perspective
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The promise and challenges of accessing innovative high-cost therapies: Angelman syndrome
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Assessing the American health care system: How did we get here?
Wednesday, May 17 | 8:00 – 9:45 a.m.
This session will first address what accelerated approval is, including its scientific underpinnings and how it has been used in the CGT space. The session will then turn to forward-looking and in-depth panel discussion on the future of accelerated approval. Speakers will focus on:
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Level-setting accelerated approval for CGTs: What sponsors should know
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Bluebird’s experience with accelerated approval and efficacy data - discussion on the Skysona (eli-cel) experience
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The scientific underpinnings of accelerated approval: Validity of biomarkers and clinical data to demonstrate relevance
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Novel endpoints and biomarkers in supporting CGT development
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CBER’s views on the past, present, and future of accelerated approval for CGT products
Thursday, May 18 | 8:00 – 9:45 a.m.
This symposium will bring together senior leaders from global health authorities (FDA and PDMA) to discuss important aspects of their regulatory frameworks for cell and gene therapy products. Speakers will focus on:
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Upcoming guidance documents, new policies, and convergence efforts between regulatory bodies
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Discussion of the current outlook for cell and gene therapy development in 2023 and beyond
Friday, May 19 | 8:00 – 9:45 a.m.
This session will be tailored to help the meeting's audience understand current access issues within the Medicaid system and explore solutions to better accommodate the exponential pipeline of gene and cell therapies. Speakers will focus on:
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Current challenges in patient access and the implications for scientists
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The role of Medicaid in ensuring timely, equitable access to care
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Payer perspective on outcomes-based arrangements: State Medicaid programs
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Payer perspective on outcomes-based arrangements: Managed care organizations
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Payer perspective on outcomes based arrangements: Reinsurance for states and MCOs
Caitlin is ASGCT's senior advocacy program specialist.
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