Objectives: To study the impact of novel treatments for elderly (≥66 yr) patients with multiple myeloma (MM) in daily practice by comparing real-world effects [overall survival (OS) and quality-adjusted life years (QALYs)] and costs over time. Also, we calculate cost-effectiveness of treatment sequences commonly prescribed to predict effects and costs if patients had received a different treatment sequence.
Methods: Real-world data including patient and disease characteristics, treatment information and resource use were collected from 1054 elderly patients with MM. Patients received first-line treatment during 2004-2007 (cohort 1) and 2008-2013 (cohort 2). The two cohorts were compared using a patient-level simulation (PLS) model comprising regression models which used patient and disease characteristics to estimate time to next treatment and death. Effects and costs from cohort 2 were compared to 4 commonly prescribed real-world sequences.
Results: Utilisation of novel agents was higher for cohort 2 compared to cohort 1. Modelled average OS for cohort 1 was 38 months (median 25) and total costs €44 200. OS for cohort 2 was 42 months (median 28) and total costs €69 017. The model identified potential OS gains if all patients were to be treated using combinations containing thalidomide, lenalidomide and bortezomib in that particular order. This sequence had, compared to real-world treatment, the most favourable incremental cost-effectiveness ratio, €24 618 per life year gained and €34 875 per QALY.
Conclusions: Our patient-level model enabled to study the effects and costs of entire treatment sequences and to compare real-world treatment patterns over time. Increased utilisation of novel agents improved survival and increased costs for real-world patients with MM in the Netherlands.

Bortezomib, Cost-effectiveness analysis, Lenalidomide, Multiple myeloma, Real-world data, Registries, Thalidomide
dx.doi.org/10.1111/ejh.12571, hdl.handle.net/1765/91660
European Journal of Haematology
Erasmus MC: University Medical Center Rotterdam

Blommestein, H.M, Verelst, S.G.R, de Groot, S, Huijgens, P.C, Sonneveld, P, & Uyl-de Groot, C.A. (2016). A cost-effectiveness analysis of real-world treatment for elderly patients with multiple myeloma using a full disease model. European Journal of Haematology, 96(2), 198–208. doi:10.1111/ejh.12571