FDA Commissioner Marty Makary’s proposal to roll out a new drug approval pathway is heightening hope for the rare disease industry, but attorneys say implementation may face challenges for spurring rare drug development.
Makary outlined on the Megyn Kelly Show in April a plan to approve drugs for rare conditions based on a “plausible mechanism,” allowing medicines to be approved without data from a randomized controlled clinical trial. A drug could be eligible for the pathway if it “makes sense physiologically,” and “the mechanism is scientifically plausible,” he said.
“Even though we don’t have a randomized control trial, because it’s ...