Novel Drug Approvals by the U.S. Food and Drug Administration in Rare Diseases: Findings From 2020–2023

Rare diseases, affecting fewer than 200,000 individuals in the U.S., present significant challenges due to the lack of effective treatments, leading to substantial clinical and humanistic burdens. Novel therapies offer the potential to improve patient outcomes and quality of life. The FDA Center for Drug Evaluation and Research (CDER) has been instrumental in fostering pharmaceutical innovations, with a notable increase in novel drug approvals (NDAs) for rare diseases. This study systematically reviews NDAs granted by the CDER for rare diseases between 2020 and 2023 to understand trends in rare disease drug development. All novel drugs approved during this period were identified using the FDA drug database. Data was collected from drug labels, approval letters, and FDA submission documents, focusing on the number of NDAs, indication types, approval categories, regulatory pathways, and trial endpoints, including patient-reported outcomes (PROs).

This report is a poster presentation of Axtria from ISPOR 2024.

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