Broadening the perspective of cost-effectiveness modeling in COPD: a new patient-level simulation model suitable to evaluate stratified medicine.
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 patientlevel 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.
|Keywords||chronic obstructive pulmonary disease, discrete event simulation model, costs, personalized medicine, quality-adjusted lifeyears|
|Persistent URL||dx.doi.org/10.1016/j.jval.2018.10.008, hdl.handle.net/1765/122615|
|Journal||Value in Health|
Hoogendoorn, M, Corro Ramos, I, Baldwin, M, Gonzalez-Rojas Guiz, N, & Rutten-van Mölken, M.P.M.H. (2019). Broadening the perspective of cost-effectiveness modeling in COPD: a new patient-level simulation model suitable to evaluate stratified medicine. Value in Health, 22, 313–332. doi:10.1016/j.jval.2018.10.008