FDA Continues to Make Rare Diseases a Priority with Speedy Therapeutic Development

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FDA’s CDER continues to make rare diseases a priority with drug approvals and programming to speed therapeutic development.

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.

The percentage of drugs approved to treat rare or orphan diseases have increased during the past decade. More than half of CDER’s novel drug approvals in 2021 were for orphan diseases.


CDER is continuing to advance drug development for rare diseases by sharing data with the Critical Path Institute and developing the Rare Disease Cures Accelerator-Data and Analytics Platform. The platform provides a database of information on rare diseases, which can aid in standardization and further understanding. CDER is also working to develop publicly available sets of clinical outcome assessments as part of the Standard Core Clinical Outcome Assessment Grant Program. These steps will aid in trial design, endpoint selections, and other important considerations for drug development.

Source: FDA