FDA Unveils New Framework to Speed Ultra-Rare Drug Approvals (1)

The FDA formally introduced on Monday a pathway that seeks to accelerate the development of drugs for ultra-rare diseases, opening the door for treatments that often struggle to meet the agency’s rigorous approval process.

The framework outlines a “plausible mechanism” pathway that would allow drugmakers to bypass certain clinical trial requirements for drugs treating ultra-rare conditions. It would target products where a randomized clinical trial is not feasible in order to meet the Food and Drug Administration’s approval requirements, according to the agency’s draft guidance.

“It’s common sense,” FDA Commissioner Marty Makary said at an event at the US ...