Abstract

Purpose
High-cost novel therapies for multiple myeloma (MM) require evaluation of efficacy and cost-effectiveness.
Materials and Methods
This study developed a methodology to assess cost-effectiveness using nationwide data from 11,450 newly diagnosed MM patients. A novel algorithm was applied to identify lines of therapy (LoT).
Results
The number of newly diagnosed MM patients increased significantly, from 873 in 2010 to 1,464 in 2019 (p<0.001). Advancing LoT was associated with shorter time to next treatment (TTNT) and overall survival (OS) (p<0.001), while all-cause medical costs increased with each LoT (p<0.001). bortezomib-melphalan-prednisolone was the most common frontline regimen for transplant-ineligible patients (29.2%), while bortezomib-thalidomide-dexamethasone was most used for transplant-eligible patients (11.3%). Daratumumab monotherapy demonstrated superior second TTNT (7.8 vs. 5.2 months) and OS (8.5 vs. 5.3 months) compared to standard care in heavily treated MM patients, with statistical significance maintained after cost adjustment. For subsequent therapies following daratumumab, a methodology was developed to estimate required medical costs using the incremental cost-effectiveness ratio (ICER): Expected cost, $ = ICER × (Expected life expectancy – 0.567) + 35,601.
Conclusion
This study provides a novel cost-effectiveness framework linking treatment efficacy and real-world costs, supporting predictions of societal costs for future MM therapies.

• Keywords: Daratumumab, Cost-effectiveness, Multiple myeloma, CAR T-Cell Therapy, Bispecific antibodies