FDA Adjusts Review Timeline to Keep Up with Orphan Drug Demand

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The agency says the increasing requests for orphan drug designation has resulted in a change in FDA’s review goals.

In a July 18, 2016 FDAVoice blog post, Gayatri Rao, MD, director of FDA’s Office of Orphan Products Development (OOPD), stated the agency will be revising its goals in regards to the review of orphan drug applications in order for FDA to meet growing demand from the industry for orphan drug designation. FDA’s new timeline goal will be to review appropriate orphan drug applications on an average of 75% of requests within 120 days of receipt.

According to Rao, there was a 30% increase in orphan drug designation applications in 2014, compared to 2013. The interest continued to increase in 2015 when the agency received 470 requests. Rao says that based on requests received so far in 2016, this year could see another 30% increase. Due to the continued increase in requests, the agency may have to reevaluate its review times again in the future.

“We strive to review these requests in an efficient and timely manner because we understand how critical designation can be for companies to move forward with their drug development plans. At the same time, we endeavor to safeguard the intent of the Orphan Drug Act by conducting a thorough review to ensure that the drugs we designate fully satisfy the criteria for designation and the financial incentives associated with designation," Rao says in the blog post.

Rao stresses that companies can help FDA stick to the review timeline by reducing the number of review cycles needed and providing all the necessary information for OOPD to complete its review. FDA recommends companies review information on www.fda.gov/orphan when developing their application.


Orphan drugs are treatments developed to treat rare diseases. The Orphan Drug Act was passed in 1983 and included incentives for companies to develop drugs for rare diseases, including tax credits and market exclusivity.

Source: FDA