title: Cost-effectiveness analysis alongside clinical trials II-An ISPOR Good Research Practices Task Force report.
abstract: 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 validity. In 2005, ISPOR published the Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials: The ISPOR RCT-CEA 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.
authors.affiliation: Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Schools of Medicine and Pharmacy, University of Washington, Seattle, WA, USA. Electronic address: sramsey@fredhutch.org.
authors.forename: Scott D
authors.initials: SD
authors.lastname: Ramsey
authors.affiliation: Outcomes & Evidence Lead, CV/Metabolic, Pain, Urology, Gender Health, Global Health & Value, Pfizer, Inc., New York, NY, USA.
authors.forename: Richard J
authors.initials: RJ
authors.lastname: Willke
authors.affiliation: Division of General Internal Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Henry
authors.initials: H
authors.lastname: Glick
authors.affiliation: Duke Clinical Research Institute, Duke University, Durham, NC, USA.
authors.forename: Shelby D
authors.initials: SD
authors.lastname: Reed
authors.affiliation: Institute for Clinical Effectiveness and Health Policy (IECS), University of Buenos Aires, Buenos Aires, Argentina.
authors.forename: Federico
authors.initials: F
authors.lastname: Augustovski
authors.affiliation: Department of Economics, Stockholm School of Economics, Stockholm, Sweden.
authors.forename: Bengt
authors.initials: B
authors.lastname: Jonsson
authors.affiliation: William R. Lindsay Chair of Health Economics, University of Glasgow, Glasgow, Scotland, UK.
authors.forename: Andrew
authors.initials: A
authors.lastname: Briggs
authors.affiliation: Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Schools of Medicine and Pharmacy, University of Washington, Seattle, WA, USA.
authors.forename: Sean D
authors.initials: SD
authors.lastname: Sullivan
mesh_terms: D026683:Advisory Committees; D003362:Cost-Benefit Analysis / Q000379:methods*; D006291:Health Policy; D006302:Health Services Research / Q000191:economics*; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000191:economics* / Q000379:methods; D058028:Research Report
keywords: clinical trial; cost-effectiveness; economic; guidelines