title: Patient Heterogeneity in Health Economic Decision Models for Chronic Obstructive Pulmonary Disease: Are Current Models Suitable to Evaluate Personalized Medicine?
abstract: OBJECTIVE
  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.
authors.affiliation: Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, The Netherlands. Electronic address: hoogendoorn@imta.eur.nl.
authors.forename: Martine
authors.initials: M
authors.lastname: Hoogendoorn
authors.affiliation: Department for Prevention and Health Services Research, National Institute for Public Health and the Environment (RIVM), Bilthoven, The Netherlands; Department of Epidemiology, University Medical Centre Groningen, Groningen, The Netherlands.
authors.forename: Talitha L
authors.initials: TL
authors.lastname: Feenstra
authors.affiliation: IMS Health, Health Economics and Outcomes Research and Real-World Evidence Solutions, London, UK.
authors.forename: Yumi
authors.initials: Y
authors.lastname: Asukai
authors.affiliation: Institute of Health & Wellbeing, University of Glasgow, Glasgow, UK.
authors.forename: Andrew H
authors.initials: AH
authors.lastname: Briggs
authors.affiliation: The Swedish Institute for Health Economics, Lund, Sweden; Health Economics Unit, Department of Clinical Sciences in Malmö, Lund University, Lund, Sweden; Evidera, London, UK.
authors.forename: Sixten
authors.initials: S
authors.lastname: Borg
authors.affiliation: National Center for Respiratory Pharmacoeconomics and Pharmacoepidemiology (CESFAR), Verona, Italy.
authors.forename: Roberto W
authors.initials: RW
authors.lastname: Dal Negro
authors.affiliation: Pharmaceutical Outcomes Research and Policy Program, School of Pharmacy, University of Washington, Seattle, WA, USA.
authors.forename: Ryan N
authors.initials: RN
authors.lastname: Hansen
authors.affiliation: Department of Public Health and Clinical Medicine, Occupational and Environmental Medicine, The OLIN Unit, Umeå University, Umeå, Sweden.
authors.forename: Sven-Arne
authors.initials: SA
authors.lastname: Jansson
authors.affiliation: Helmholtz Zentrum München, Institute of Health Economics and Health Care Management, Member of the German Center for Lung Research, Comprehensive Pneumology Center Munich, Neuherberg, Germany.
authors.forename: Reiner
authors.initials: R
authors.lastname: Leidl
authors.affiliation: ICON Health Economics, Toronto, Ontario, Canada.
authors.forename: Nancy
authors.initials: N
authors.lastname: Risebrough
authors.affiliation: IMS Health, Health Economics and Outcomes Research and Real-World Evidence Solutions, London, UK.
authors.forename: Yevgeniy
authors.initials: Y
authors.lastname: Samyshkin
authors.affiliation: Helmholtz Zentrum München, Institute of Health Economics and Health Care Management, Member of the German Center for Lung Research, Comprehensive Pneumology Center Munich, Neuherberg, Germany.
authors.forename: Margarethe E
authors.initials: ME
authors.lastname: Wacker
authors.affiliation: Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, The Netherlands.
authors.forename: Maureen P M H
authors.initials: MPMH
authors.lastname: Rutten-van Mölken
mesh_terms: D000368:Aged; D003657:Decision Making; D004470:Economics, Medical; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D008962:Models, Theoretical; D057285:Precision Medicine; D029424:Pulmonary Disease, Chronic Obstructive / Q000628:therapy; D019057:Quality-Adjusted Life Years
keywords: COPD; model; patient heterogeneity; validation