Photographer: Krisztian Bocsi/Bloomberg

Rare-Disease Drugs Get Help From FDA's Dedicated Swat Team

  • Agency is trying to expedite review of requests for incentives
  • FDA has backlog of about 200 reviews pending, commisioner says

Incentives for drugmakers to develop treatments for the 7,000 rare diseases in the U.S. have helped spur innovation in the once-neglected field in recent years -- in fact they’ve worked so well the Food and Drug Administration hasn’t been able to keep up.

To clear a backlog of about 200 requests, FDA Commissioner Scott Gottlieb is deploying a swat team focused solely on reviewing applications to get experimental treatments designated as orphan drugs. The designation, which applies to rare diseases affecting fewer than 200,000 people, qualifies drugmakers for incentives such as tax credits for research and monopoly status for seven years.

The swat team will eliminate the backlog within 90 days and respond to new designation requests within 90 days as well, the FDA said in a statement Thursday. The agency has faced a sharp increase in applications, which more than doubled in four years to 568 in 2016.

Rare diseases have become a fast-growing pocket in the pharmaceutical industry, helped in part by incentives but also because the drugs’ prices have climbed. For example, Alexion Pharmaceuticals Inc.’s drug Soliris, which typically costs from $500,000 to $700,000 a year, has one of the largest price tags in the world. Globally, sales of treatments for rare diseases are expected almost to double, to $209 billion, by 2022, according to Evaluate Ltd., a life-sciences consulting company.

About two-thirds of the 33 FDA employees in the orphan-drug office are currently involved in the review and administration of the designation program. A few of the senior reviewers will dedicate all of their time to clearing the backlog, the agency said.

“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,” Gottlieb said.

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