Rare Disease Drug Development Guide

Federal Register notice: FDA has issued a draft guidance, Rare Diseases: Common Issues in Drug Development, to assist drug and biological sponsors in conducting more efficient and successful drug development programs. The document revises and replaces an 8/2015 drug guidance by updating the natural history studies section; inclusion of issues for evaluation and validation of surrogate biomarkers; description of nonclinical flexibility; additional information on external controls and early randomization; addition of a safety section; retitled chemistry, manufacturing, and controls section to pharmaceutical quality considerations; additional information on changes to drug substance or manufacturing process with clarification on areas of flexibility; and inclusion of an additional considerations section addressing several topics — participation of patients, caretakers, and advocates, consideration of pediatric issues, and interactions with FDA. To view the notice, click here.