FDA Fast-Tracks Orphan Drugs


The agency is trying to deliver on its goal of a 90-day response time.

The FDA is trying to finally clear its backlog of orphan drug applications and ensure that the backlog does not grow in the future.

The FDA is promising to complete review of every orphan drug request older than 120 days by September 21. After that, the agency says that it will review every new request within 90 days of its receipt.

Currently, the agency has around 200 orphan drug applications pending review. The number of applications the FDA receives is increasing every year. In 2016 the agency received 568 orphan drug requests, more than twice the number they received in 2012. The FDA said that this increase is a positive thing for those with rare diseases, but the agency has struggled to keep up with the program.

To help catch up, the FDA is creating what it calls a “backlog SWAT team,” a group of experienced researchers who have worked extensively on orphan drug applications in the past. They will begin with the oldest requests and work their way through the backlog and use a new-reportedly streamlined-designation review template to speed along the process.

Related article: Will Gottlieb’s Faster Drug Approvals at FDA Help or Harm?

“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,” said FDA Commissioner Scott Gottlieb, M.D., in press 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.”

In addition to the SWAT team, the FDA is also making changes such as reorganizing staff, increasing the efficiency of medical product centers, and creating a new FDA Orphan Products Council designed to help provide consistency to the review process.

You can read the whole “Orphan Drug Modernization Plan” here

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