Schulman K A, Glick H, Buxton M, Sculpher M, Backhouse M, Bell L, Eisenberg J M
Clinical Economics Research Unit, Georgetown University Medical Center, Washington, D.C. 20007, USA.
Control Clin Trials. 1996 Aug;17(4):304-15. doi: 10.1016/0197-2456(95)00166-2.
Prospective economic assessments of new pharmaceutical therapies are increasingly being incorporated into phase III clinical trials. We report on the design of an economic evaluation integrated into the Flolan International Randomized Survival Trial (FIRST). Economic evaluation was considered a critical component of the assessment of this therapy given the resources required to administer epoprostenol (Flolan), a therapy that would require lifelong continuous intravenous infusion. Economic secondary endpoints were incorporated in the clinical trial. The economic evaluation of the treatment was integrated into all aspects of study development, including study design, implementation, and monitoring. Since this was a multinational trial, special care was required to ensure that the protocol design was appropriate for all study countries. The economic assessment required the development of several methodologic components: a set of background economic concepts to guide protocol development, a set of resource items to be recorded when required for study participants; a set of data collection instruments for assessment of health-related quality of life for study patients; and a protocol for a resource costing exercise for the study. We report the data elements included in the study design, as well as a discussion of some of the issues faced in developing the economic evaluation for this trial.
新药物疗法的前瞻性经济评估越来越多地被纳入III期临床试验。我们报告了一项纳入弗洛兰国际随机生存试验（FIRST）的经济评估设计。鉴于使用依前列醇（弗洛兰）所需的资源，这种疗法需要终身持续静脉输注，经济评估被认为是该疗法评估的关键组成部分。经济次要终点被纳入临床试验。治疗的经济评估被纳入研究开展的各个方面，包括研究设计、实施和监测。由于这是一项跨国试验，需要特别注意确保方案设计适用于所有研究国家。经济评估需要开发几个方法学组件：一套指导方案制定的背景经济概念、一组在研究参与者需要时记录的资源项目、一套用于评估研究患者健康相关生活质量的数据收集工具，以及一项针对该研究的资源成本核算方案。我们报告了研究设计中包含的数据元素，以及对开展该试验经济评估时面临的一些问题的讨论。
