As more drug makers race to win orphan designations, a new report finds the Food and Drug Administration fails to consistently record and evaluate needed information, which suggests the agency may not always include critical data for granting the coveted orphan status.

Specifically, the FDA granted the designation to 26 applications that were missing required information, such as verified estimated patient populations, and 102 of 148 so-called review templates were missing background information. Moreover, FDA guidance was not always clear in instructing agency staff on how to use the recorded information, according to the U.S. Government Accountability Office.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!


What is it?

STAT Plus is a premium subscription that delivers daily market-moving biopharma coverage and in-depth science reporting from a team with decades of industry experience.

What's included?

  • Authoritative biopharma coverage and analysis, interviews with industry pioneers, policy analysis, and first looks at cutting edge laboratories and early stage research
  • Subscriber-only networking events and panel discussions across the country
  • Monthly subscriber-only live chats with our reporters and experts in the field
  • Discounted tickets to industry events and early-bird access to industry reports

Leave a Comment

Please enter your name.
Please enter a comment.

Sign up for our Daily Recap newsletter

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy