FDA Revises Guidance for Sponsors of Rare Disease Drug Trials

The FDA issued a revised draft guidance to help sponsors of rare disease treatments conduct more effective clinical trials.

The revision includes a drug safety section, more information on external controls and early trial randomization and additional information on changes to the drug substance or the manufacturing process.

The newly added drug safety section offers approaches to expanding a safety assessment — such as paying close attention to dose selection. It also calls for the use of data from alternative sources, such as trials that use the drug for other indications or studies of similar treatments.