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Welcome to Day Two and Session 5: Fireside Chat: Implementing the Plausible Mechanism Pathway: From Concept to Clinic
Session Chair(s)
Hobart Rogers, PHARMD, PHD
Pharmacologist, CDER, FDA, United States
Dan Swerdlow, MD, PHD
Senior Director, Early Clinical Development, GSK, United Kingdom
This session will provide a comprehensive overview of FDA's draft guidance, "Considerations for the Use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause." The presentation will examine regulatory pathways for individualized antisense oligonucleotides (ASOs) under CDER's jurisdiction, with particular emphasis on how the plausible mechanism framework can streamline development and approval processes for these patient-specific therapies. The session will also feature insights from Critical Path Institute (C-Path), which will present their strategic vision for implementing this regulatory pathway and introduce their "One to Millions" Initiative, a collaboration designed to accelerate access to individualized genetic therapies.
Learning Objective : At the conclusion of this session, participants should be able to: - Describe three key FDA plausible mechanism criteria for individualized antisense oligonucleotide (ASO) therapies.
- Explain CDER’s regulatory pathway for individualized ASOs, including two differences from traditional drug development.
- Summarize the C-Path “One to Millions” Initiative, its goals, and two strategies to accelerate access to individualized genetic therapies.
Speaker(s)
Representative Invited
Director, Office of Neuroscience, OND, CDER, FDA, United States
An Overview of the New Plausible Mechanism Guidance from a CDER Perspective
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