Cost-Effectiveness of Novel Agents in Medicare Patients with Multiple Myeloma: Findings from a U.S. Payer's Perspective.

BACKGROUND: Since multiple myeloma (MM) incurs a substantial economic burden in care management, more and more discussion has been generated in recent years about the costs of novel antimyeloma drugs and their associated value. Because of these costs, economic assessment that quantifies value of care over the long-term is essential.

OBJECTIVE: To determine the cost-effectiveness (measured as cost per life-year saved) of front-line novel agent-based therapy use among a cohort of elderly patients with MM in a real-world setting.

METHODS: We identified 2,551 elderly patients with advanced MM from 2000 to 2009 who initiated novel agent-based therapy (bortezomib, lenalidomide, or thalidomide) or chemotherapy from the Surveillance, Epidemiology, and End Results-Medicare linked data. Patients were characterized according to age at diagnosis, sex, race, geographic region, marital status, socioeconomic status, comorbidities, and receipt of novel agents. Twenty-month cost of care and overall survival related to MM were compared between patients treated with novel agent-based therapy and patients treated with chemotherapy. A net monetary benefit approach and corresponding cost-effectiveness acceptability curves were used to evaluate the cost-effectiveness of novel agent-based therapy.

RESULTS: Overall, average 12-month MM total costs were 2.03 times higher for novel agent-based therapy ($144,665) than for chemotherapy ($47,750). Antimyeloma pharmacy costs represented about 31% ($45,095) of total MM costs for patients treated with novel agents but represented about 19% ($8,921) of total MM costs for patients treated with chemotherapy. Twelve-month survival rates increased significantly among patients receiving novel agents compared with patients receiving chemotherapy. In the incremental net monetary benefit analysis, after adjusting for potential covariates, patient use of novel agents was only cost-effective compared with chemotherapy when the willingness-to-pay thresholds were high, at about $230,000.

CONCLUSIONS: Given the most common treatment practices in the United States, the use of novel agent-based therapy is not cost-effective at its current level of cost and effectiveness. Future studies should evaluate the generalizability of these results by evaluating cost-effectiveness of novel agent-based therapy use in different patient populations.

DISCLOSURES: Funding for this study was contributed by the Agency for Healthcare Research and Quality (R01-H5018956). The authors have nothing to disclose. Study concept and design were contributed by Chen, Lairson, and Du, along with Chan. Chen and Du took the lead in data collection, along with Lairson and Huo. Data interpretation was performed by Chen, Chan, and Du, along with Lairson and Huo. The manuscript was written by Chen, along with Du, Lairson, Chan, and Huo, and revised by primarily by Du, along with Lairson, Huo, Chen, and Chan.