FDA Eliminates Orphan Drug Designation Backlog, But Sets Stage for Controversial Changes

Rare Daily Staff

U.S. Food and Drug Administration Commissioner Scott Gottlieb said the agency has eliminated a backlog of about 200 applications for orphan drug designations, delivering on a promise he made to a U.S. Senate subcommittee to respond to the requests and implement a policy to require the agency to act on new designation requests within 90 days of receiving them.

Gottlieb made the announcement in a September 12 blog post that discussed the Orphan Drug Modernization Plan, which the agency unveiled at the end of June. The plan is intended to better advance the goals of the Orphan Drug Act. It comes as there has been a steady increase in the number of orphan drug designation requests over the past five years. In 2016, the FDA’s Office of Orphan Products Development received 568 new requests for designation–more than double the number of requests received in 2012. The increased interest has been hailed by the FDA as a positive development for those with rare diseases.

Companies that receive orphan drug designation for their product qualify for various incentives including tax credits for clinical trial costs, relief from prescription drug user fees and the potential for seven years of marketing exclusivity after the drug is approved. To be eligible for orphan drug designation, Drugs and biologics must be developed for a rare disease indication that affects fewer than 200,000 people in the United States.

While the Orphan Drug Act has played a critical role in enticing pharmaceutical companies to develop drugs for the small patient populations that define rare diseases, there has long been outcry about abuses of the designation as companies have profited from it in ways that were not envisioned when lawmakers drafted the act. An analysis by Kaiser Health News found that since the passage of passage of the Orphan Drug Act more than 70 drugs that have been approved with the designation were first approved for indications with broader markets including several drugs that have captured billion markets.

Gottlieb said that the 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. “As part of this process, the FDA will also be examining aspects of how the agency grants designations,” he said, “to make sure they continue to reflect current science and drug development and the goals intended by Congress.”

The agency is expected to issue guidance document and policies to address the issues. Gottlieb highlighted one loophole the agency will target, which allows sponsors to avoid an obligation to study drugs in pediatric indications if sponsors received an orphan designation for a pediatric subtype of an otherwise common and non-orphaned adult disease. He noted this use pre-dated the implementation of the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act, both of which were aimed at promoting more pediatric studies. But by letting sponsors designate pediatric subpopulations of drugs intended to treat adult diseases, he said the drugmakers receive an unintended “free pass” from having to study drugs in these or other pediatric uses.

“For all the success of the ODA, there’s been criticism that some sponsors are using designations as a way to sidestep other important public health goals set out by Congress,” he said. “We need to make sure our policies take notice of all of these new challenges and opportunities.”

September 12, 2017