Ultra-Rare Disease Approvals By US FDA Could Take More ‘Mechanistic,’ Less ‘Empirical’ Approach

Clinical trial sponsors in the rare disease space face “big safety demands” from regulators, which can be challenging for those with limited resources. A CRO founder makes the case for “embedding automation” into the clinical trial process to keep pace with regulatory requirements.

The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.

FDA regulation was painted as an obstacle to US dominance in the cell and gene therapy space even as panelists at an agency event praised the Office of Therapeutic Products' track record under Nicole Verdun.

Expanded funding for cost recovery could dovetail with FDA Commissioner Martin Makary’s idea for a “conditional approval” pathway based on a plausible mechanism of action.