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.

• Keywords
• ASPIRE, Carfilzomib, Cost-effectiveness, Multiple myeloma, Real world, Registry of Monoclonal Gammopathies,
• MeSH
• Cost-Benefit Analysis * MeSH
• Dexamethasone pharmacology   MeSH
• Quality-Adjusted Life Years MeSH
• Lenalidomide pharmacology   MeSH
• Humans MeSH
• Neoplasm Recurrence, Local drug therapy   etiology   MeSH
• Multiple Myeloma drug therapy   etiology   mortality   MeSH
• Drug Costs MeSH
• Oligopeptides pharmacology   MeSH
• Disease-Free Survival MeSH
• Antineoplastic Combined Chemotherapy Protocols MeSH
• Registries MeSH
• Check Tag
• Humans MeSH
• Publication type
• Journal Article MeSH
• Randomized Controlled Trial MeSH
• Geographicals
• Czech Republic MeSH
• Names of Substances
• carfilzomib MeSH Browser
• Dexamethasone MeSH
• Lenalidomide MeSH
• Oligopeptides MeSH