Regulatory Strategy for Novel Modalities

Overview

Approach to IND and approval pathway for unprecedented therapeutics; emphasizes risk-based evolving data packages, proactive regulator education, and orthogonal analytical methods rather than standard data sets. For novel modalities without prior IND precedent, regulatory agencies move away from expected standard data sets toward risk-based decision-making, requiring developers to proactively educate regulators and demonstrate causal links between molecular variability and clinically relevant parameters. Presenting regulators with a rationalized parameter range and an integrated, evolving data set showing structure, function, PKPD, and safety together brings them along as engaged partners rather than placing the developer on the back foot responding to questions about data gaps. (Segment 3 (10:00-15:00))