FDA’s Center for Drug Evaluation and Research (CDER) continues to make rare diseases a priority with teams and programs dedicated to speeding up therapeutic development. Both government incentives and scientific advancements have allowed for drug development for rare diseases.
According to CDER Director, Patrizia Cavazzoni, M.D., “A rare disease is any disease that affects less than 200,000 people in the United States. Drug development for the approximately 7000 rare diseases can be complex for many reasons.” There are approximately 30 million people living in the US who have a rare disease. Many of these diseases have little to no available treatments, because of a lack of participants in rare disease clinical trials and limited understanding of the diseases.
