(209) Does delaying coverage for accelerated approval treatments disproportionately impact patients with high unmet needs?

Background: : Payers are increasingly considering delaying coverage for new non-oncology treatments approved via the Food and Drug Administration (FDA)’s accelerated approval (AA) pathway. It is unclear to what extent this decision would impact patients with high unmet needs.

Objective: : To compare the economic burden and disease characteristics of non-oncology conditions targeted by AA medicines and those treated with non-AA medicines.

Methods: : All conditions for which there was an FDA-approval via AA between Jan 2015 and Feb 2025 were identified; the comparison group was comprised of the 100 diseases with the most recent non-AA medicines approved prior to Feb 2025. Data on economic burden and disease characteristics were sourced from published literature. The economic burden was estimated from the U.S. societal perspective, including direct medical costs, direct non-medical costs, indirect costs from patient productivity loss, and indirect costs from informal caregiving if available. Costs were inflated to 2025 USD using the Medical Care Component of the BLS’ Consumer Price Index. Disease characteristics measures included life years, quality of life, prevalence, and age of onset. Mann-Whitney U Test were applied to test whether the differences between AA and non-AA conditions were statistically significant.

Results: : The 34 non-oncology AA treatments represented n=18 unique diseases and were compared against n=100 non-AA diseases. Average annual societal costs for AA diseases were 1.50 times higher than for non-AA conditions ($94,857 vs. $63,219, p< 0.01). Compared with non-AA conditions, the annual average productivity loss and caregiver burden were 1.35 ($21,538 vs. $9,177) and 1.59 times ($19,846 vs. $7,678) greater in the AA conditions, respectively. On average, AA conditions were characterized by shorter post-diagnosis life expectancy (25.9 vs. 31.0 years), lower quality of life (0.651 vs. 0.688), and lower prevalence (277 vs. 2,250 per 100,000). Diseases treated by AAs also disproportionately affect children ( < 18 years: 29.4% vs. 24.2%) and older adults (>65 years: 27.5% vs. 23.1%).

Conclusions: : Diseases targeted by AA treatments imposed greater economic burden and reflect more severe disease characteristics than non-AA conditions. Delaying coverage of AA treatments would disproportionately impact patients with high unmet needs.

Sponsorship:: National Pharmaceutical Council