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.

Amgen s r o Prague Czech Republic

Department of Haematooncology University Hospital Ostrava and Faculty of Medicine University of Ostrava 17 listopadu 1790 708 52 Ostrava Czech Republic

Department of Hemato Oncology University Hospital Olomouc and Faculty of Medicine and Dentistry Palacky University Olomouc 1 P Pavlova 185 6 779 00 Olomouc Czech Republic

Department of Internal Medicine Charles University Prague 1st Faculty of Medicine and General Teaching Hospital Katerinska 32 121 08 Prague Czech Republic

Department of Internal Medicine Hematology and Oncology University Hospital Brno and Faculty of Medicine Masaryk Universit Jihlavská 340 20 625 00 Brno Czech Republic

Economic Modeling Center of Excellence Global Health Economics Amgen GmbH Zug Switzerland

Global Health Economics Amgen GmbH Zug Switzerland

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