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.