ABSTRACT
Background: We assessed the economic value of carfilzomib 56 mg/m2 and dexamethasone (Kd56) vs. bortezomib and dexamethasone (Vd) for relapsed/refractory multiple myeloma (R/RMM) using ENDEAVOR trial results.
Methods: Cost-effectiveness of Kd56 vs. Vd was assessed using a partitioned survival model by estimating progression-free survival, overall survival, and direct costs over a lifetime horizon. Surveillance Epidemiology and End Results (SEER) survival data were extrapolated after matching registry and ENDEAVOR patients. Utilities were sourced from the literature and mapped from patient-reported quality of life in ENDEAVOR to estimate quality-adjusted life-years (QALYs) from life-years (LYs).
Results: The model predicted an average gain of 1.66 LYs and 1.50 QALYs with Kd56 vs. Vd, and lifetime additional costs of $182,699, resulting in an incremental cost-effectiveness ratio (ICER) of $121,828/QALY gained. The ICER was $114,793/QALY in patients with 1 prior treatment; $99,263/QALY in those not transplanted, and <$150,000/QALY up to an 85% discount in bortezomib price.
Conclusions: Kd56 is cost-effective for patients with R/RMM at a willingness-to-pay threshold of $150,000/QALY. Trial data in the model may limit generalizability; however, SEER registry data mitigates this challenge. Kd56 provides additional value in key subgroups, and remains cost-effective after steep comparator discounts.
Declaration of interest
AJ has served as an advisory board member and consultant for Amgen, BMS, Celgene, Janssen, Karyopharm Therapeutics, Millennium Takeda, Sanofi, and Skyline Diagnostics; has received sponsorships/grants from Amgen, Celgene, BMS, Karyopharm, and Millennium/Takeda; and has received honoraria from Amgen, BMS, Celgene, Jansen, Karyopharm, Millennium/Takeda, Sanofi, and Skyline Diagnostics. SA has served as an advisory board member for Takeda, Pharmacyclics, Amgen, and Novartis. MC, IM, and SP are employed by and own stock in Amgen Inc. AB and IH are employees of Evidera, Inc., and this analysis was developed in collaboration with Evidera and sponsored by Amgen. The authors would like to thank Julie Gegner, employee of Amgen, for writing and editorial support. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. A reviewer on this manuscript has disclosed advisory board and speakers’ bureau membership for Amgen, Janssen-Janssen, Bristol-Myer Squibb, Takeda, and Celgene but have no other relevant financial relationships to disclose.