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Hoogendoorn, M.* ; Feenstra, T.* ; Asukai, Y.* ; Briggs, A.* ; Borg, S.* ; dal Negro, R.* ; Hansen, R.N.* ; Jansson, S.A.* ; Leidl, R. ; Risebrough, N.* ; Samyshkin, Y.* ; Wacker, M. ; Rutten van-Mölken, M.*

Patient heterogeneity in health economic decision models for Chronic Obstructive Pulmonary Disease: Are current models suitable to evaluate personalized medicine?.

Value Health 19, 800-810 (2016)
Publ. Version/Full Text DOI PMC
Open Access Green as soon as Postprint is submitted to ZB.
Objectives To assess how suitable current chronic obstructive pulmonary disease (COPD) cost-effectiveness models are to evaluate personalized treatment options for COPD by exploring the type of heterogeneity included in current models and by validating outcomes for subgroups of patients. Methods A consortium of COPD modeling groups completed three tasks. First, they reported all patient characteristics included in the model and provided the level of detail in which the input parameters were specified. Second, groups simulated disease progression, mortality, quality-adjusted life-years (QALYs), and costs for hypothetical subgroups of patients that differed in terms of sex, age, smoking status, and lung function (forced expiratory volume in 1 second [FEV1] % predicted). Finally, model outcomes for exacerbations and mortality for subgroups of patients were validated against published subgroup results of two large COPD trials. Results Nine COPD modeling groups participated. Most models included sex (seven), age (nine), smoking status (six), and FEV1% predicted (nine), mainly to specify disease progression and mortality. Trial results showed higher exacerbation rates for women (found in one model), higher mortality rates for men (two models), lower mortality for younger patients (four models), and higher exacerbation and mortality rates in patients with severe COPD (four models). Conclusions Most currently available COPD cost-effectiveness models are able to evaluate the cost-effectiveness of personalized treatment on the basis of sex, age, smoking, and FEV1% predicted. Treatment in COPD is, however, more likely to be personalized on the basis of clinical parameters. Two models include several clinical patient characteristics and are therefore most suitable to evaluate personalized treatment, although some important clinical parameters are still missing.
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Publication type Article: Journal article
Document type Scientific Article
Corresponding Author
Keywords Copd ; Model ; Patient Heterogeneity ; Validation
ISSN (print) / ISBN 1098-3015
e-ISSN 1524-4733
Journal Value in Health
Quellenangaben Volume: 19, Issue: 6, Pages: 800-810 Article Number: , Supplement: ,
Publisher Elsevier
Publishing Place New York, NY
Non-patent literature Publications
Reviewing status Peer reviewed