An FDA Perspective on Rare Diseases

The FDA Office of Orphan Products Development seeks to advance the evaluation of drugs and diagnostics to treat rare diseases. A growing toolkit of incentives has helped drive the development of new products for rare diseases, but the need remains great. We spoke to Gayatri Rao, director of the FDA’s Office of Orphan Products Development, about the rare disease landscape, how scientific developments are reshaping clinical trials and the use of biomarkers, and what the agency is doing to better incorporate patients’ perspectives into the drug review process.