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Session 5: Post-authorisation evidence generation for ATMPs
Session Chair(s)
Keith Wonnacott, PhD
Vice President, Regulatory Affairs, Lexeo Therapeutics, United States
ATMPs have several unique considerations such as: they are often developed for rare diseases, they have unique long-term safety concerns, and they often show compelling efficacy early in development. Therefore, there is a significant need to gather data and/or follow patients outside of the clinical trial setting. Increasingly, we are hearing discussions of how registries and real-world evidence can be used to address some of the regulatory and payer expectations post-authorisation. This session will discuss expectations and experience in generating and utilising these innovative approaches.
Topics explored in this Session:
- Registries Supporting RWE | Big Data.
- Resolving HTA/Payer Uncertainties with RWE
- RWE for decision making for PLEG and for approval.
Speaker(s)
Nuria Semis-Costa, MSc
Scientific Specialist (Risk Management), European Medicines Agency, Netherlands
Post-authorisation evidence generation for ATMPs
Ian Winburn
Vice President, Global Medical Lead Haemophilia, Endocrine and IEM, Pfizer Biopharmaceuticals Group, United States
Registries enabling RWE generation and regulatory/payer discussions
Yanni Hao, PhD
Executive Director Cell & Gene, Global RWE & Data Science, Novartis, United States
Experience with post-authorisation data generation, registries, and the use of RWE
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