Presentation (CTI)

OBJECTIVES: Drugs for autoimmune diseases are often approved for multiple indications, for which their cost-effectiveness varies. Due to the current pricing and reimbursement models, US healthcare payers struggle with aligning a single price to each drug’s differing value. This study examines the clinical and economic value, price, and launch strategies of multi-indication immunotherapies for the treatment of autoimmune diseases in the US.
METHODS: Twelve multi-indication ISTs, with no available generic or biosimilar, across 10 indications were identified (US Food and Drug Administration-approved 2000-2024). A targeted literature review in Medline (1966-2024) and Embase (1974-2024) was performed to obtain data on economic evaluations and disease prevalence. Wholesale Acquisition Costs (WAC) were obtained from Micromedex RED BOOK. Total quality-adjusted life years (QALYs), disease prevalence, and WACs were compared across indications.
RESULTS: First approved indications provided numerically higher clinical benefits as measured by mean total QALYs (8.13; 95% CI: −1.00, 17.27) compared to the second (5.61; 95% CI: 2.52, 8.70; P=0.62) and third (5.52; 95% CI: 5.31, 5.74; P=0.76). Mean US disease prevalence per 100,000 individuals was 409.16 (95% CI: 136.69, 681.63) for first compared to 1571.84 (95% CI: −225.76, 3,369.44) for second and 13.07 (95% CI: −0.78, 26.92) for third approved indications. Average total WAC increased with each subsequent indication compared to the first indication (range: 7.72% [second indication]-138.33% [sixth indication]).
CONCLUSIONS: The study suggests that in the US, the first indication launch of multi-indication ISTs is prioritized based on clinical value and unmet need, as reflected in higher QALYs and disease prevalence. The US price-value misalignment suggests the need for a value-based, indication-specific pricing policy to better align drug prices with their clinical benefits. Broader evaluation is necessary to support this approach.