Cost-effectiveness analysis for multinational clinical trials

• Published in: Statistics in medicine Vol. 24; no. 13; pp. 1965 - 1982
• Main Authors: Pinto, Eleanor M., Willan, Andrew R., O'Brien, Bernie J.
• Format: Journal Article
• Language: English
• Published: Chichester, UK John Wiley & Sons, Ltd 15-07-2005; Wiley Subscription Services, Inc
• Subjects: Clinical trials; Clinical Trials as Topic - economics; Comparative analysis; Cost-Benefit Analysis - statistics & numerical data; cost-effectiveness; Costs; Effectiveness studies; Humans; Internationality; multinational clinical trials; Myocardial Infarction - drug therapy; Ontario; Pharmaceuticals; Shrinkage; shrinkage estimators; Statistical analysis; Thrombolytic Therapy; Ontario
• ISSN: 0277-6715; 1097-0258
• DOI: 10.1002/sim.2078

Clinical trials of cost–effectiveness are often conducted in more than one country. The two most common ways of dealing with the multinational nature of the data are either to calculate a pooled estimate or to stratify results by country. Since the between‐country heterogeneity in costs is potentially substantial, pooled estimates may be difficult to interpret for any one country. Policy decisions are often made at a national level, and so country‐specific results are important. However, country‐specific analyses will be based on fewer patients and will often fail to provide adequate precision for statistical analyses.
Shrinkage estimation is a compromise between these two methods and has been used successfully in other fields. These estimates are country‐specific yet less variable than those derived through a subgroup approach. Univariate and multivariate shrinkage estimators for costs and effects are proposed, then compared with one another and to the traditional methods in a simulation study. The methods are illustrated using data from a multinational trial evaluating the cost–effectiveness of three thrombolytic drug regimens in patients with acute myocardial infarction. Copyright © 2005 John Wiley & Sons, Ltd.