Patient-Reported Outcomes in Clinical Trials of Rare Diseases
Patient-Reported Outcomes in Clinical Trials of Rare Diseases
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DOI：
10.1007/s11606-014-2892-z
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发表时间：
2014-08-01
影响因子：
5.7
通讯作者：
Bennett, Antonia V.
中科院分区：
文献类型：
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作者：
Basch, Ethan;Bennett, Antonia V.
近几十年来，测量患者报告结果 (PRO) 的科学取得了长足的进步，可以在临床研究中评估患者的感受和功能。当采用仔细的规划和严格的方法时，可以使用 PRO 措施成功地评估罕见疾病人群的患者体验。在罕见疾病背景下设计和实施 PRO 分析时存在许多挑战，包括结果的异质性、合适措施的可用性、招募和选择适当的数据收集方法。解决这些问题的策略是存在的，并且已经在过去的临床研究中得到应用，特别是在儿科人群中。通过研究人员、PRO 方法学家和患者组织之间的合作，罕见疾病临床试验中的 PRO 评估特别成功。 PRO 测量的总体目标是了解患者体验，它是评估疾病和治疗影响的重要组成部分。
The science of measuring patient-reported outcomes (PROs) has advanced substantially in recent decades, allowing evaluation of how patients feel and function in clinical research. Assessment of the patient experience in populations with rare diseases can be successfully achieved using PRO measures when careful planning and rigorous methods are employed. A number of challenges exist when designing and implementing PRO analyses in rare disease contexts, including heterogeneity of outcomes, availability of suitable measures, recruitment, and selection of appropriate data collection methods. Strategies to address these exist and have been employed in past clinical research, particularly in pediatric populations. PRO assessments in rare disease clinical trials have been particularly successful through partnerships between investigators, PRO methodologists, and patient organizations. The overall goal of PRO measurement is to understand the patient experience and it provides an essential part of evaluating the impact of disease and treatment.