Cost-effectiveness of once weekly carfilzomib 70 mg/m2 plus dexamethasone in patients with relapsed and refractory multiple myeloma in the United States

Efficacy and safety of weekly carfilzomib (70 mg/m2), dexamethasone, and daratumumab (KdD70) is comparable to twice-weekly KdD56 while being a more convenient dosing option: a cross-study comparison of the CANDOR and EQUULEUS studies

“…Previously, once-weekly carfilzomib dosing with Kd70 was shown to have improved patients' quality of life with improved clinical outcomes relative to twiceweekly carfilzomib dosing [21][22][23]. In line with these results for the once-weekly Kd70 doublet regimen, we expect that the once-weekly KdD70 triplet regimen will provide patients a more convenient lenalidomidefree treatment option, thus fostering adherence to the treatment schedule, dose, and treatment duration.…”

“…The KdD regimen is recommended by the National Comprehensive Cancer Network (NCCN 2020, version 3) guidelines for patients with previously treated multiple myeloma [4]. As weekly carfilzomib dosing can be more convenient and improve the quality of life [21,22], we undertook rigorous efficacy analyses in this cross-study comparison to support the comparability of twice-weekly KdD56 to once-weekly KdD70. We found that the corresponding groups of the CANDOR and EQUULEUS studies showed similar efficacy in terms of ORR and PFS, both before and after IPTW adjustment, including in patients who were refractory to lenalidomide and independent of past stem cell transplant status.…”

“…study showed delayed disease symptom worsening as well as greater convenience and satisfaction with onceweekly Kd70 as compared with twice-weekly Kd27. Improved clinical outcomes for the once-weekly Kd regimen translated into prolonged health-related quality of life in a cost-effective manner [21,22]. Thus, as observed with the Kd regimen, once-weekly carfilzomib dosing with KdD is expected to improve patients' quality of life by allowing patients to maintain a social role and other meaningful activities, which is key from a patient's perspective [23].…”

A Systematic Review of Cost-Effectiveness Analyses of Novel Agents in the Treatment of Multiple Myeloma

“…All studies declared to have used empirical data for cost estimations (e.g., costs of adverse events, monitoring, administration and medicines), although the sources were not specified in the study of Gong et al [25][26][27][28][29][30][31][32][33][34][35][36][37][38] A lifetime horizon was reported in 10 studies with varying definitions with a maximum of 40 years [25,27,[29][30][31][32][33][34][35][36][37][38]. In contrast, Pelligra et al used a three-year time horizon because, according to them, it reflects a typical US payer's budget horizon and allows enough time to model clinically relevant outcomes appropriately [26].…”

“…Zhang et al used a 10-year time horizon, without giving a rationale [28]. Most studies (10) received funding from a commercial party (e.g., pharmaceutical industry) [25,26,[29][30][31][33][34][35][36]38]. All horizons and funding types are presented in Table 1.…”