New Strategic FDA Plan Seeks to Eliminate Orphan Drug Designation Request Backlogs


An estimated 200 orphan drug designation requests are pending FDA review.

The FDA today released the Orphan Drug Modernization Plan to address the backlog of orphan drug designation requests, according to a press release.

The Orphan Drug Act was first enacted in the United States in 1983 to encourage the development of drugs to treat patients with rare diseases and conditions, such as Huntington’s disease, myoclonus, Amyotrophic lateral sclerosis, Tourette syndrome, and muscular dystrophy.

Over the past 5 years, the number of orphan drug designation requests have steadily increased. The Office of Orphan Products Development (OOPD) received 568 new requests for designation in 2016 alone—–more than double the amount of requests received in 2012.

“People who suffer with rare diseases are too often faced with no, or limited, treatment options, and what treatment options they have may be quite expensive due in part to significant costs of developing therapies for smaller populations,” Scott Gottlieb, MD, FDA commissioner, said in a release. “Congress gave us tools to incentivize the development of novel therapies for rare diseases and we intend to use these resources to their fullest extent in order to ensure Americans get the safe and effective medicines they need, and that the process for developing these innovations is as modern and efficient as possible.”

Approximately 200 orphan drug designation requests are pending review by the FDA, and interest in the orphan drug space is only increasing, with 9 of 22 approved medications last year being orphan drugs.

Part of the strategic plan involves eliminating the backlog of requests within 90 days and responding to new requests within 90 days.

“By September 21, 2017, [the] FDA will complete reviews of all orphan drug designations that are older than 120 days,” the plan states. “Following that 90-day period, the agency is committing to respond to 100% of all new orphan drug designation requests within 90 days of their receipt by FDA.”

Some of the steps the agency will take to achieve its goals include the creation of a Backlog SWAT team comprised of senior, experienced OOPD reviewers to focus on designation requests in the order that the agency receives them; the creation and implementation of a new streamlined “Designation Review Template” to increase consistency and efficacy of reviews; and collaborate with the FDA’s medical product centers to increase efficiency, including conducting joint reviews with the Office of Pediatric Therapeutics for rare pediatric disease designation requests.

“To ensure all future requests review a response within 90 days of receipt, the agency will take a multifaceted approach,” the press release stated. “These efforts include, among other new steps: reorganizing the review staff to maximize expertise and improve workload efficiencies; better leveraging the expertise across the FDA’s medical product centers’ and establishing a new FDA Orphan Products Council that will help address scientific and regulator issues to ensure the agency is applying a consistent approach to regulating orphan drug products and reviewing designation requests.”

Click here to learn more details about the Orphan Drug Designation Plan.

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