FDA Updates Guidance on Rare Disease Drug Development

The U.S. Food and Drug Administration (FDA) released a revised draft guidance for sponsors developing “orphan drugs” for the treatment or prevention of rare diseases. The guidance, which is available for public comment through March, updates the original draft guidance that was published in 2015.

The revised document is intended to assist industry sponsors in conducting more efficient development programs, including updated information about natural history studies and the use of biomarkers as surrogate endpoints.

“We know that developing a drug or biologic for a rare disease can be especially challenging, which is why it’s important that the FDA continues to provide clear information to drug developers so that they can plan modern, efficient drug development programs that will be successful,” said FDA Commissioner Scott Gottlieb, MD. “Many of the updates are in direct response to feedback that we received on our prior draft.”

In its announcement of the draft guidance, the agency noted that it plans to hold a public meeting to obtain patients’ and caregivers’ perspectives on the effects of rare diseases on their daily lives. This information may help the FDA and sponsors better understand the diseases and develop treatments for them through the creation of novel endpoints or trial designs that focus on commonalities across a variety of rare diseases.

Source: FDA news release, January 16, 2019.