Clinical and Economic Burden of Diseases with Accelerated Approval Therapies: Evidence of Unmet Patient Need.

BACKGROUND: Some US payers delay coverage for non-oncology treatments approved via the Food and Drug Administration (FDA)'s accelerated approval (AA) pathway. OBJECTIVES: To estimate the economic burden and characteristics of diseases with therapies approved via the AA pathway compared to diseases with no AA therapies. METHODS: The study identified the initial FDA-approved AA indications for all non-oncology therapies from January 2015 to February 2025. For comparison, 100 non-oncology diseases with the most recent FDA-approved non-AA therapies were identified. Annual economic burden and disease characteristics were extracted from peer-reviewed literature, with costs inflated to 2025 USD. Economic burden studies that used a societal perspective were prioritized. Comparative analyses were conducted using the Mann-Whitney U Test. RESULTS: Thirty-two non-oncology AA treatments were identified representing 16 unique diseases. Average annual incremental societal costs for AA diseases were 1.4 times higher than for non-AA diseases ($91,857 vs. $65,468, p = 0.012). Additionally, the annual average productivity loss and caregiver burden were 1.8 times ($17,635 vs. $9,620, p = 0.092) and 2.4 times ($20,268 vs. $8,294, p = 0.055) greater in the AA diseases, respectively. Diseases with newly-approved AA treatments were characterized by shorter life expectancy (59.3 vs 65.1 years), lower average quality of life (0.64 vs 0.69), and more likely to be rare diseases (68.8% vs 53.1%) compared to non-AA diseases. CONCLUSION: Non-oncology diseases with treatments approved via the AA pathway imposed a higher economic burden and had more severe disease characteristics compared to non-AA diseases. These findings should be considered alongside AA coverage policy decision-making.