FDA Announces Progress, Next Steps in Streamlining Orphan Drug Review Process

FDA Commissioner Scott Gottlieb, MD, announced that the agency has achieved its goal of clearing the backlog of orphan drug designation requests pending review. The FDA also plans to implement policies to further streamline the process and to close a loophole that may be hindering pediatric drug research.

In his recent post on the FDA Voice blog, Gottlieb reminded readers of his promise in June that he would implement a plan to eliminate the backlog of orphan drug designation requests and modernize the existing policies to prevent future pile-ups. He had set a deadline of September 21 to have all requests older than 120 days processed, but that goal had been met by August 28.

To avoid backlogs like these going forward, Gottlieb said the FDA would introduce efficiency-boosting strategies like Lean management principles to optimize the distribution of work, measure productivity, and pinpoint areas where the process is held up.

“This new workflow will outline a more efficient process that eliminates redundancies and delays that don’t add value,” Gottlieb explained, adding that the new process map would be released to the public this fall.

The FDA’s plan to streamline the orphan drug designation process includes a review to update its regulatory policies, including the incentives offered to drug makers. This modernization is especially important as new biologics and molecularly targeted drugs present “complex scientific and regulatory issues,” Gottlieb said.

Although orphan drug designation is awarded to products treating a disease affecting fewer than 200,000 Americans, the FDA has emphasized the Orphan Drug Designation Program as a tool to ensure more therapy options become available for the total 25 to 30 million patients with rare diseases, which can include cancers, autoimmune disorders, and more.

However, Gottlieb’s post acknowledges the program has shortcomings that can hinder that goal, including loopholes that can weaken the power of other legislation. For instance, the FDA allows pediatric subpopulations of more common diseases to be designated as orphan conditions, but this designation actually allows pharmaceutical companies to avoid pediatric research requirements set under other laws like the Pediatric Research Equity Act (PREA).

He used the example of inflammatory bowel disease in children. When a drug receives an orphan designation to treat the small population of children with the disease, it actually becomes exempt from the PREA requirements which would have necessitated studies of the drug in children. Essentially, Gottlieb explained, the orphan designation has the opposite effect of what Congress intended by resulting in fewer pediatric studies. The FDA will issue draft guidance to close this loophole shortly.

Keeping a close watch on the efficiency of the Orphan Drug Designation Program and identifying potential loopholes is especially important as the number of requests continues to rise; Gottlieb said it has more than doubled from 2012 to 2016. He pledged that the FDA would continue to utilize the available resources provided by Congress to incentivize the development of innovative therapies for rare diseases, including those affecting children.

“These steps will help us achieve our ultimate goal: to facilitate the development of safe, effective innovations that have the potential to meaningfully impact rare diseases,” Gottlieb concluded.