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 not feasible, we will allow that, and at the same time, monitor everybody who gets it so that we can make inferences as soon as the data speaks,” Makary said.
A new pathway has the potential to open the door for more rare disease drug approvals, which has been a long-standing challenge due to the high costs of development for diverse patient populations that often limit clinical trial designs.
But the proposal hinges on how Makary will move the plan forward, which could range from lawmakers introducing legislation, the agency issuing guidance, or even expanding the FDA’s existing authority.
“It’s a wait-and-see in terms of which mechanism FDA might use and whether they will need a legislative vehicle or not to implement something here,” said Julie Tibbets, chair of Goodwin Procter LLP’s life science regulatory and compliance practice.
“The current access gap that patients face while they’re waiting for new treatments to reach approval in the rare disease space really heightens the need for this flexibility of getting promising new treatments to patients,” Tibbets said.
The FDA has approved hundreds of drugs to treat rare diseases, but developing them remains a challenge due to regulatory requirements sponsors must meet. Some of the obstacles relate to data collection, limited patient pools and trial designs, and limitations to natural history studies that would show the progression of rare diseases…….
