United BioSource Corporation, Bethesda, Maryland 20814, USA.
COPD. 2013 Jun;10(3):393-8. doi: 10.3109/15412555.2013.795423.
The precise assessment of treatment efficacy in clinical trials requires scientific instruments that are not only relevant to the target population and treatment, but have been shown to be reliable, valid, and sensitive to change within the intended context of use. This paper describes the background, procedures, and current status of 2 patient-reported outcome (PRO) instruments developed for use in clinical trials of chronic obstructive pulmonary disease (COPD). The first measure, the EXAcerbations of Chronic pulmonary disease Tool (EXACT), was developed under the EXACT-PRO Initiative, a multi-year, multi-sponsor project involving experts in pulmonary medicine, instrument development, and drug development regulatory issues, dedicated to the development of a single, standardized instrument for evaluating the effects of treatment on acute exacerbations of COPD. The second measure, the EXACT-Respiratory Symptoms (E-RS) scale, is a derivative instrument comprising a subset of EXACT items to test the effect of treatment on the severity of respiratory symptoms in stable COPD. The EXACT-PRO Initiative was the first PRO instrument development consortia, and the EXACT and E-RS are the first PRO measures to undergo qualification review by the United States Food and Drug Administration (FDA).
精确评估临床试验中的治疗效果需要科学的工具，这些工具不仅要与目标人群和治疗方法相关，而且要在预期使用范围内经过验证，具有可靠性、有效性和对变化的敏感性。本文介绍了为慢性阻塞性肺疾病（COPD）临床试验开发的 2 种患者报告结局（PRO）工具的背景、程序和现状。第一个衡量标准是慢性肺病加重工具（EXACT），它是在 EXACT-PRO 计划下开发的，该计划是一个多年多赞助商项目，涉及肺病学、仪器开发和药物开发监管问题方面的专家，致力于开发一种单一的、标准化的仪器，用于评估治疗对 COPD 急性加重的影响。第二个衡量标准是 EXACT-Respiratory Symptoms（E-RS）量表，它是一个衍生工具，由 EXACT 的一部分项目组成，用于测试治疗对稳定 COPD 患者呼吸症状严重程度的影响。EXACT-PRO 计划是第一个 PRO 仪器开发联盟，EXACT 和 E-RS 是第一个接受美国食品和药物管理局（FDA）资格审查的 PRO 措施。
