Cost-Effectiveness Analysis Alongside Clinical Trials II-An ISPOR Good Research Practices Task Force Report
Cost-Effectiveness Analysis Alongside Clinical Trials II-An ISPOR Good Research Practices Task Force Report
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DOI：
10.1016/j.jval.2015.02.001
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发表时间：
2015-03-01
期刊：
影响因子：
4.5
通讯作者：
Sullivan, Sean D.
中科院分区：
文献类型：
--
作者：
Ramsey, Scott D.;Willke, Richard J.;Sullivan, Sean D.
评估药物、医疗设备和程序的临床试验现在通常评估这些干预措施的经济价值。前瞻性临床/经济试验数量的不断增加反映了人们对新技术经济信息的广泛兴趣，以及许多国家现在考虑经济价值证据和临床疗效的监管和报销要求。随着决策者越来越需要医疗保健干预措施的经济价值证据，在临床研究的同时进行高质量的经济分析是可取的，因为它们扩大了特定干预措施的可用信息范围，并且可以有效地提供及时的信息，具有较高的内部有效性，并且在正确设计和分析时，可以提供合理的外部有效性。 2005 年，ISPOR 发布了《与临床试验一起进行成本效益分析的良好研究实践：ISPOR RCTCEA 'I'ask Force 报告》 ISPOR 于 2014 年启动了该报告的更新，以纳入过去 9 年的方法学进展。该报告提供了更新的建议，反映了与试验设计、选择数据元素、数据库设计和管理、分析以及结果报告相关的几个领域的进展。特别工作组成员指出，试验的设计应尽可能评估有效性（而不是功效），应包括临床结果指标，并应直接从研究对象那里获取卫生资源的使用情况和健康状况的效用。经济数据的收集应充分纳入研究。应采用意向治疗方法进行增量分析，并辅以相关亚组分析。应表征不确定性。文章应遵守报告成本效益分析结果的既定标准。经济研究和试验是对其他评估（例如建模研究）的补充，为决策者提供信息，决策者在做出资源分配决策时考虑经济价值证据和临床疗效。
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 RCTCEA 'I'ask 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.