Advanced

Patient Heterogeneity in Health Economic Decision Models for Chronic Obstructive Pulmonary Disease : Are Current Models Suitable to Evaluate Personalized Medicine?

Hoogendoorn, Martine; Feenstra, Talitha L.; Asukai, Yumi; Briggs, Andrew H.; Borg, Sixten LU ; Dal Negro, Roberto W.; Hansen, Ryan N.; Jansson, Sven Arne; Leidl, Reiner and Risebrough, Nancy, et al. (2016) In Value in Health 19(6). p.800-810
Abstract

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... (More)

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.

(Less)
Please use this url to cite or link to this publication:
author
, et al. (More)
(Less)
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
COPD, model, patient heterogeneity, validation
in
Value in Health
volume
19
issue
6
pages
11 pages
publisher
Wiley-Blackwell
external identifiers
  • scopus:84969531822
  • wos:000385930500017
ISSN
1098-3015
DOI
10.1016/j.jval.2016.04.002
language
English
LU publication?
yes
id
d3980d87-6687-4483-b684-c0753eb85e46
date added to LUP
2016-11-23 12:52:12
date last changed
2017-11-19 04:35:05
@article{d3980d87-6687-4483-b684-c0753eb85e46,
  abstract     = {<p>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 [FEV<sub>1</sub>] % 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 FEV<sub>1</sub>% 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 FEV<sub>1</sub>% 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.</p>},
  author       = {Hoogendoorn, Martine and Feenstra, Talitha L. and Asukai, Yumi and Briggs, Andrew H. and Borg, Sixten and Dal Negro, Roberto W. and Hansen, Ryan N. and Jansson, Sven Arne and Leidl, Reiner and Risebrough, Nancy and Samyshkin, Yevgeniy and Wacker, Margarethe E. and Rutten-van Mölken, Maureen P M H},
  issn         = {1098-3015},
  keyword      = {COPD,model,patient heterogeneity,validation},
  language     = {eng},
  month        = {09},
  number       = {6},
  pages        = {800--810},
  publisher    = {Wiley-Blackwell},
  series       = {Value in Health},
  title        = {Patient Heterogeneity in Health Economic Decision Models for Chronic Obstructive Pulmonary Disease : Are Current Models Suitable to Evaluate Personalized Medicine?},
  url          = {http://dx.doi.org/10.1016/j.jval.2016.04.002},
  volume       = {19},
  year         = {2016},
}