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Cost-effectiveness analysis for multinational clinical trials
Eleanor M Pinto1, Andrew R Willan, Bernie J O'Brien
1Program in Population Health Sciences, Research Institute, Hospital for Sick Children, 555 University Avenue, Toronto, Ontario, Canada M5G 1X8.
Statistics in Medicine
|April 2, 2005
Summary
Shrinkage estimation offers a balanced approach for multinational cost-effectiveness studies. This method provides precise, country-specific results, improving upon pooled or stratified analyses for healthcare policy decisions.
Area of Science:
- Health economics
- Clinical trial methodology
- Biostatistics
Background:
- Multinational clinical trials present challenges in cost-effectiveness analysis due to potential between-country heterogeneity in costs.
- Traditional methods like pooled estimates can obscure country-specific nuances, while country-specific analyses may lack statistical precision.
- Policy decisions are often national, necessitating reliable country-level cost-effectiveness data.
Purpose of the Study:
- To propose and evaluate shrinkage estimation as a method to balance precision and country-specificity in multinational cost-effectiveness analyses.
- To compare univariate and multivariate shrinkage estimators against traditional pooled and stratified methods.
- To illustrate the application of shrinkage estimation using real-world data from a multinational trial.
Main Methods:
- Development of univariate and multivariate shrinkage estimators for costs and effects in multinational settings.
- A simulation study to compare the performance of shrinkage estimators with pooled and stratified subgroup analyses.
- Application of the proposed methods to data from a multinational trial on thrombolytic drugs for acute myocardial infarction.
Main Results:
- Shrinkage estimators provide country-specific results that are less variable than those from traditional subgroup analyses.
- Simulation results demonstrate the advantages of shrinkage estimation in achieving a balance between precision and interpretability.
- The methods are effectively illustrated using data from a trial on thrombolytic drug regimens.
Conclusions:
- Shrinkage estimation represents a valuable compromise for analyzing cost-effectiveness data from multinational clinical trials.
- This approach enhances the reliability of country-specific estimates, supporting informed national policy decisions.
- Shrinkage methods offer improved precision and interpretability compared to conventional pooled or stratified analyses.
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