Removing the Orphan Drug Backlog a Top Priority of the FDA

James Radke

In 2016, the U.S. Food and Drug Administration (FDA) received 568 new requests for Orphan Drug Designation and there are still currently an estimated 200 requests under review for the special designation.
The numbers reflect the pharmaceutical industry’s interests in developing orphan drugs for rare disease, and as a champion for the rare disease community, new FDA Commissioner Scott Gottlieb, MD, is making efforts to ensure that those drugs are reviewed in a more efficient and timely manner.

Today, Gottlieb announced a 2-part plan that will remove the backlog of requests waiting for review and expedite responses moving forward.
An orphan drug designation means that the FDA has determined that the drug in development is for a rare disease and the designation provides various development incentives, including tax credits for clinical trial costs, relief from prescription drug user fee, and eligibility for 7 years of marketing exclusivity upon approval. 
First, in the coming 90 days, the FDA will complete the review of all Orphan Drug Designation requests that are currently backlogged (requests older than 120 days).
Next, the FDA plans to have a strategy in place to ensure that all new Orphan Drug Designation requests will have received a response from the agency within 90 days of receipt.
In a statement, Gottlieb said, “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.” Dr. Gottlieb, added, “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.”
1. To achieve the first goal of addressing the backlog of orphan drug designation requests in the next 90 days, the agency has put in place the following procedures:
2. To achieve the second goal of making sure all future orphan drug designations requests will receive a response within 90 days, the agency will take the following steps:
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