Methodology and results of real-world cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone in relapsed multiple myeloma using registry data

Abstract

Objective To predict the real-world (RW) cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone (KRd) versus lenalidomide and dexamethasone (Rd) in relapsed multiple myeloma (MM) patients after one to three prior therapies. Methods A partitioned survival model that included three health states (progression-free, progressed disease and death) was built. Progression-free survival (PFS), overall survival (OS) and time to discontinuation (TTD) data for the Rd arm were derived using the Registry of Monoclonal Gammopathies in the Czech Republic; the relative treatment effects of KRd versus Rd were estimated from the phase 3, randomised, ASPIRE trial, and were used to predict PFS, OS and TTD for KRd. The model was developed from the payer perspective and included drug costs, administration costs, monitoring costs, palliative care costs and adverse-event related costs collected from Czech sources. Results The base case incremental cost effectiveness ratio for KRd compared with Rd was €73,156 per quality-adjusted life year (QALY) gained. Patients on KRd incurred costs of €117,534 over their lifetime compared with €53,165 for patients on Rd. The QALYs gained were 2.63 and 1.75 for patients on KRd and Rd, respectively. Conclusions Combining the strengths of randomised controlled trials and observational databases in cost-effectiveness models can generate policy-relevant results to allow well-informed decision-making. The current model showed that KRd is likely to be cost-effective versus Rd in the RW and, therefore, the reimbursement of KRd represents an efficient allocation of resources within the healthcare system.

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

“…Comparisons between regimens containing two novel agents resulted in 0.02 to 1.10 LYs and 0.01 to 0.91 QALYs gained. This comes with high costs: lifetime healthcare costs ranging from USD 60,413 to USD 1,434,937 per patient and incremental costs compared to backbone therapies ranging from dominated to USD 535,530 per patient [25][26][27][28][29][30][31][32][34][35][36][37][38] The ICERs we found were in only 12 (out of 32) comparisons beneath the generally accepted willingness-to-pay (WTP) threshold of USD 150,000 per QALY gained in the USA [25][26][27][28][29][30][31][32][33][34][35][36][37][38]56]. Three of these were comparisons between two novel treatment; thus, only nine comparisons were between a backbone therapy combined with a novel agent and a backbone therapy only.…”

“…However, higher WTP thresholds are reported by Health Technology Assessment (HTA) agencies (e.g., up to USD 95,072 in the Netherlands) [58]. Nevertheless, none of the ICERs per QALY gained (except for dominating regimens, i.e., Pano-Vd and comparisons between Pom-d and daratumumab monotherapy and Kd) fell below the WTP threshold of USD 34,097 [25][26][27][28][29][30][31][32][33][34][35][36][37][38]57] With the WTP threshold of USD 150,000 per QALY gained taken in account, compared with backbone therapies Vd and Rd, carfilzomib and panobinostat are below the WTP in most cases [29,31,[33][34][35]. The ICER per QALY gained of pomalidomide is below the WTP threshold against Kd, daratumumab monotherapy and HiDex [25,26,30].…”

“…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.…”

“…We used the CHEERS checklist to assess quality and completeness of reporting of the studies. Most studies (12) scored well regarding reporting quality [26][27][28][29][30][31][32][33][34][35][36][37][38]. One study (Gong et al) was of a low quality [25].…”