Overview of cost-effectiveness analysis and health state utilities in multiple myeloma and estimations of health state utilities from real-world macedonian data

Abstract

Multiple myeloma (MM) is a hematological neoplastic plasma-cell disorder in the bone marrow. Nowadays, the survival of MM patients has improved by using novel therapies (e.g., bortezomib, thalidomide). Decision-analytic models (DAM) are known to be very useful in guiding clinical and health policy decisions by systematically evaluating expected outcomes of alternative treatments. When conducting DAM, it is recommended by National Institute for Clinical Excellence (NICE) as the preferred framework to use qualityadjusted life years as the main measure for health outcomes derived from generic preference-based instruments. The objective of our study was to identify and analyze the structural and methodological approaches of published studies that evaluated: (1) DAM for various treatment strategies in MM and (2) published health utility values (HUVs) after MM treatment. The searches were developed in the electronic literature database PubMed/MEDLINE. Additionally, we aimed to derive real-world data for HUVs from the current patients’ treatment landscape in the Hematology Clinical Center in Skopje. The survey collecting national HUVs was conducted during the period January-November 2016, using the generic preference-based instrument EQ-5D-5L, administered at one-time point in 20 MM patients. Only five studies reported on using a DAM. Seven studies were included in the extraction of already published HUVs. The calculated national health utility score of 0.723 was comparable to the published data in the previously identified studies reporting on similar MM treatment protocols. Several modeling approaches and HUVs, developed for different treatment strategies and target groups in MM were identified. Those data could be used to parameterize a DAM for MM treatment.

Cost-effectiveness analysis of treating transplant-eligible multiple myeloma patients in Macedonia

“…This decision was based on the literature review published before that evaluated the most recent decision-analytic models in MM. 23 In the 1-year model, it is assumed that none of the patients die; however, that is not the case in the 10- and 20-year models, as based on the nature of the disease and the overall survival it should be taken into consideration that a patient could die during that period.…”