Regulatory Science for ATMPs

Overview

Evolving regulatory frameworks for Advanced Therapy Medicinal Products (ATMPs) including gene and cell therapies, covering registration-ready clinical trial design, risk-based approaches to process changes, and international regulatory convergence. Clinical trials for gene therapies should be designed with registration in mind from the outset: phase 1/2 data can become part of the registration dossier if collected consistently using quality systems and the same endpoints throughout development. Both EMA and FDA accepted an integrated analysis combining phase 1/2 and phase 2/3 trial data for a San Raffaele gene therapy, because the data was consistently collected with quality systems using the same endpoints throughout the program. (Segment 2 (05:00-10:00))