Cost-effectiveness analysis for multinational clinical trials

Stat Med. 2005 Jul 15;24(13):1965-82. 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.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Clinical Trials as Topic / economics*
  • Cost-Benefit Analysis / statistics & numerical data*
  • Humans
  • Internationality*
  • Myocardial Infarction / drug therapy*
  • Ontario
  • Thrombolytic Therapy