Economic evaluation of lipid lowering with PCSK9 inhibitors in patients with familial hypercholesterolemia: Methodological aspects.

Autor: Wisløff T; Department of Health Management and Health Economics, University of Oslo, Oslo, Norway; Department of Infectious Disease Epidemiology and Modelling, Norwegian Institute of Public Health, Oslo, Norway. Electronic address: torbjorn.wisloff@medisin.uio.no., Mundal LJ; The Lipid Clinic, Department of Endocrinology, Morbid Obesity and Preventive Medicine, Oslo University Hospital, Oslo, Norway; Department of Nutrition, Institute of Basic Medical Sciences, University of Oslo, Oslo, Norway., Retterstøl K; The Lipid Clinic, Department of Endocrinology, Morbid Obesity and Preventive Medicine, Oslo University Hospital, Oslo, Norway; Department of Nutrition, Institute of Basic Medical Sciences, University of Oslo, Oslo, Norway., Igland J; Department of Global Public Health and Primary Care, University of Bergen, Bergen, Norway., Kristiansen IS; Department of Health Management and Health Economics, University of Oslo, Oslo, Norway.
Jazyk: angličtina
Zdroj: Atherosclerosis [Atherosclerosis] 2019 Aug; Vol. 287, pp. 140-146. Date of Electronic Publication: 2019 Jun 14.
DOI: 10.1016/j.atherosclerosis.2019.06.900
Abstrakt: Background and Aims: Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors have proved to reduce low density lipoprotein cholesterol levels in numerous clinical trials. In two large clinical trials, PCSK9 inhibitor treatment reduced the risk of cardiovascular disease. Our aim was to explore the impact of varying assumptions about clinical effectiveness on health and economic outcomes for patients with familial hypercholesterolemia.
Methods: We used a previously published and validated Norwegian model for cardiovascular disease. The model was updated with recent data from the world's second largest registry of patients with genetically confirmed familial hypercholesterolemia. We performed analyses for 24 different subgroups of patients based on age, gender, statin tolerance and previous history of cardiovascular disease.
Results: In 1 out of 24 subgroups, PCSK9 inhibitors were cost-effective when effectiveness was modelled using direct relative efficacy as reported in the FOURIER trial. When using assumptions, as suggested in a recent consensus statement from the European Atherosclerosis Society, 14 subgroups were cost-effective.
Conclusions: Cost-effectiveness of PCSK9 inhibitors depends highly on assumptions regarding effectiveness. Basing assumptions only on randomised controlled trials, and not taking into account varying effects based on baseline cholesterol level, results in much fewer groups being cost-effective.
(Copyright © 2019 Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
Externí odkaz: