Design Issues for Conducting Cost-Effectiveness Analyses Alongside Clinical Trials
- 1 May 2001
- journal article
- review article
- Published by Annual Reviews in Annual Review of Public Health
- Vol. 22 (1), 129-141
- https://doi.org/10.1146/annurev.publhealth.22.1.129
Abstract
▪ Abstract In response to rising demands for timely economic data on new medical technologies, cost-effectiveness studies are increasingly being conducted alongside clinical trials. Because of the historical differences in perspective and methods between cost-effectiveness studies and clinical trials, the design phase of these hybrid trials requires special consideration. Cost-effectiveness studies require more comprehensive evaluations of outcomes than the endpoints typically measured in clinical trials. Often, these comprehensive outcome measures (such as quality of life) prove useful for interpreting the other endpoints measured in the trial, as well as for estimating the cost-effectiveness of the intervention. In this manuscript, we discuss several aspects related to the design of joint clinical/economic trials, including study perspective, hypothesis testing, sample size estimation, and methods for collecting cost and outcome data. We also discuss issues that may limit the external validity of the cost-effectiveness results of these trials. Many potential threats to external validity can be successfully addressed if they are identified and accounted for in the design phase of the study.Keywords
This publication has 21 references indexed in Scilit:
- Rationale and Design of the National Emphysema Treatment Trial: A Prospective Randomized Trial of Lung Volume Reduction SurgeryChest, 1999
- Economic foundations of cost-effectiveness analysisJournal of Health Economics, 1997
- Guidelines for authors and peer reviewers of economic submissions to the BMJBMJ, 1996
- Confidence intervals for cost-effectiveness ratios: An application of Fieller's theoremHealth Economics, 1996
- A Strategy for Collecting Pharmacoeconomic Data During Phase II/III Clinical TrialsPharmacoEconomics, 1996
- In Search of Power and Significance: Issues in the Design and Analysis of Stochastic Cost-Effectiveness Studies in Health CareMedical Care, 1994
- Economic Analysis in Randomized Control TrialsMedical Care, 1992
- Assumptions of the QALY procedureSocial Science & Medicine, 1989
- Selecting Technologies for AssessmentInternational Journal of Technology Assessment in Health Care, 1989
- Economic analysis and clinical trialsControlled Clinical Trials, 1984