Published Mar 2015
Ramsey SD, Willke RJ, Glick HA, et al. Cost-effectiveness analysis alongside clinical trials II: an ISPOR Good Research Practices Task Force Report. Value Health. 2015;18(2):161-172.
Clinical trials evaluating medicines, medical devices, and procedures
now commonly assess the economic value of these interventions. The
growing number of prospective clinical/economic trials reflects both
widespread interest in economic information for new technologies
and the regulatory and reimbursement requirements of many countries
that now consider evidence of economic value along with clinical
efficacy. As decision makers increasingly demand evidence of economic
value for health care interventions, conducting high-quality
economic analyses alongside clinical studies is desirable because they
broaden the scope of information available on a particular intervention,
and can efficiently provide timely information with high internal
and, when designed and analyzed properly, reasonable external
In 2005, ISPOR published the Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials: The ISPOR RCTCEA Task Force report. ISPOR initiated an update of the report in 2014 to include the methodological developments over the last 9 years. This report provides updated recommendations reflecting advances in several areas related to trial design, selecting data elements, database design and management, analysis, and reporting of results. Task force members note that trials should be designed to evaluate effectiveness (rather than efficacy) when possible, should include clinical outcome measures, and should obtain health resource use and health state utilities directly from study subjects. Collection of economic data should be fully integrated into the study. An incremental analysis should be conducted with an intention-to-treat approach, complemented by relevant subgroup analyses. Uncertainty should be characterized. Articles should adhere to established standards for reporting results of cost-effectiveness analyses. Economic studies alongside trials are complementary to other evaluations (e.g., modeling studies) as information for decision makers who consider evidence of economic value along with clinical efficacy when making resource allocation decisions.
Keywords: clinical trial, cost-effectiveness, economic, guidelines.
Copyright © 2015, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc.