FDA Issues Recommendations on Pre-IND Meetings for Rare Disease Drugs

The FDA released draft guidance on how sponsors of treatments of rare diseases can get more from their pre-investigational new drug meetings with the agency.

Sponsors should be ready to discuss the prevalence of the disease in adult and pediatric populations worldwide, the currently approved therapies and standard of care, its rate and pattern of progression and the availability of patients for clinical trials, the agency said.

“Sponsors developing drugs for rare diseases face many challenges,” the guidance states. These may include the low number of patients with the disorder, a lack of understanding about the disorder’s history, the need to conduct pediatric trials and a lack of precedent for drug development.