FDA Meeting Orphan Drug Goals: Gottlieb

FDA commissioner Scott Gottlieb says reviews of all orphan drug designation requests older than 120 days were completed 8/28, well ahead of the 9/21 deadline the agency had set for itself. The reviews were part of a comprehensive orphan drug modernization plan Gottlieb announced 6/29.

Writing in an FDA Voice blog post, Gottlieb says the early completion of reviews “is hopeful news for those with a rare disease, defined as a disease that generally affects fewer than 200,000 people in the U.S.”

He writes that the agency is putting in place new policies to improve the efficiency of its review process to ensure that it meets a new 90-day mandate to prevent new backlogs. Among the process improvements, he says, will be a reorganization of the review staff to improve workload efficiencies and to better leverage the expertise across FDA’s medical product centers. The agency also plans to use “lean management principles” to design a new process map based on an assessment of sources of delay or redundancy and metrics for measuring success. “This new workflow will outline a more efficient process that eliminates redundancies and delays that don’t add value.” He says the map will be shared in the fall.

Finally, according to Gottlieb, FDA will take new policy steps to ensure that the incentives offered by the Orphan Drug Act are granted by the agency in a way that is consistent with the manner Congress intended. FDA will soon hold a public meeting to get input on complex scientific and regulatory issues such as those raised by molecularly targeted drugs and biologics and the appropriate application of orphan incentives in that paradigm, he says.

Gottlieb says the agency will be addressing criticism that some sponsors are using orphan drug designations as a way to sidestep other important public health goals set out by Congress, such as an obligation to study drugs in pediatric indications.