Martine Hoogendoorn1, Isaac Corro Ramos2, Michael Baldwin3, Nuria Gonzalez-Rojas Guix3, Maureen P M H Rutten-van Mölken2. 1. Institute for Medical Technology Assessment/Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, The Netherlands. Electronic address: hoogendoorn@imta.eur.nl. 2. Institute for Medical Technology Assessment/Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, The Netherlands. 3. Boehringer Ingelheim International GmbH, Ingelheim, Germany.
Abstract
OBJECTIVES: To develop a health economic model that included a great diversity of patient characteristics and outcomes for chronic obstructive pulmonary disease (COPD), which can be used to inform decisions about stratified medicine in COPD. METHODS: The choice of patient characteristics and outcomes to include in the model was based on 3 literature reviews on multidimensional prognostic COPD indices, COPD phenotypes, and treatment effects in subgroups. A conceptual model was constructed including 14 patient characteristics, 7 intermediate outcomes (lung function, physical activity, exercise capacity, symptoms, disease-specific quality of life, exacerbations, and pneumonias), and 3 final outcomes (mortality, quality-adjusted life-years [QALYs], and costs). Regression equations describing the statistical associations between the patient characteristics and intermediate and final outcomes were estimated using the longitudinal data of 5 large COPD trials (19,378 patients). A patient-level simulation model was developed in which individual patients from the baseline population of the 5 trials are sampled and their outcomes over lifetime are predicted based on the regression equations. RESULTS: The base-case analysis (single-arm simulation representing treatment with tiotropium) showed that patients had a mean lung function decline of 43 mL/year, 0.62 exacerbations/year, a worsening of their physical activity and quality of life with 1.48 and 1.10 points/year, a life expectancy of 11.2 years, 7.25 QALYs, and total lifetime costs of £24,891. Results for a selection of treatment scenarios and subgroups were shown to demonstrate the potential of the model. CONCLUSIONS: We developed a unique patient-level simulation model that can be used to evaluate COPD treatment options for a variety of subgroups.
OBJECTIVES: To develop a health economic model that included a great diversity of patient characteristics and outcomes for chronic obstructive pulmonary disease (COPD), which can be used to inform decisions about stratified medicine in COPD. METHODS: The choice of patient characteristics and outcomes to include in the model was based on 3 literature reviews on multidimensional prognostic COPD indices, COPD phenotypes, and treatment effects in subgroups. A conceptual model was constructed including 14 patient characteristics, 7 intermediate outcomes (lung function, physical activity, exercise capacity, symptoms, disease-specific quality of life, exacerbations, and pneumonias), and 3 final outcomes (mortality, quality-adjusted life-years [QALYs], and costs). Regression equations describing the statistical associations between the patient characteristics and intermediate and final outcomes were estimated using the longitudinal data of 5 large COPD trials (19,378 patients). A patient-level simulation model was developed in which individual patients from the baseline population of the 5 trials are sampled and their outcomes over lifetime are predicted based on the regression equations. RESULTS: The base-case analysis (single-arm simulation representing treatment with tiotropium) showed that patients had a mean lung function decline of 43 mL/year, 0.62 exacerbations/year, a worsening of their physical activity and quality of life with 1.48 and 1.10 points/year, a life expectancy of 11.2 years, 7.25 QALYs, and total lifetime costs of £24,891. Results for a selection of treatment scenarios and subgroups were shown to demonstrate the potential of the model. CONCLUSIONS: We developed a unique patient-level simulation model that can be used to evaluate COPD treatment options for a variety of subgroups.
Authors: Deborah A Marshall; Luiza R Grazziotin; Dean A Regier; Sarah Wordsworth; James Buchanan; Kathryn Phillips; Maarten Ijzerman Journal: Value Health Date: 2020-03-26 Impact factor: 5.725
Authors: Frits Me Franssen; Peter Alter; Nadav Bar; Birke J Benedikter; Stella Iurato; Dieter Maier; Michael Maxheim; Fabienne K Roessler; Martijn A Spruit; Claus F Vogelmeier; Emiel Fm Wouters; Bernd Schmeck Journal: Int J Chron Obstruct Pulmon Dis Date: 2019-07-09