Phase I Clinical Trial Center, the First Affiliated Hospital, Sun Yat-sen University, Guangzhou, China.
Department of Pharmacy, the First Affiliated Hospital, Sun Yat-sen University, Guangzhou, China.
JAMA Netw Open. 2022 May 2;5(5):e2211644. doi: 10.1001/jamanetworkopen.2022.11644.
Regulatory authorities, industry peers, and international health policies have emphasized the value of assessing patient-reported outcomes (PROs) in clinical studies. Despite the increase in the number of clinical studies in the last decade in China, little is known about the extent of the use of PROs.
To evaluate the application and characteristics of PRO instruments as primary and secondary outcomes in randomized clinical trials in China.
DESIGN, SETTING, AND PARTICIPANTS: A cross-sectional study of interventional clinical trials conducted in China from January 1, 2010, to December 31, 2020, was performed. Data obtained from the Chinese Clinical Trial Registry and ClinicalTrials.gov databases were evaluated.
Trials were categorized according to those that (1) precisely listed PRO tools as outcomes, (2) mentioned patient subjective feelings in outcomes but did not clarify which tools were used for assessment, and (3) did not mention any PRO measurements. Data on study phase, setting, participant age, and sex were extracted from trials that considered patient feelings, along with the target diseases and names of the PRO tools.
Among a total of 34 033 trials, 6915 (20.3%) listed the explicit PRO instruments used and 3178 (9.3%) included PRO in their outcomes but did not include the names of the assessment tools. From more than 32 million people included in the registered trials, data on 1.5 million (4.7%) patients were scientifically collected by PRO instruments, and subjective feelings were assessed for 693 867 (2.1%) participants. Pain (16.8%), cancer (15.6%), and musculoskeletal symptoms (13.3%) were the most common conditions for which PROs were precisely collected by tools. The most common tools for PRO measurements were the visual analog scale, Short-Form 36, and Hamilton Depression Scale.
In this cross-sectional study, the use of PROs increased during the study period in clinical trials conducted in China. However, patient opinion appears to still be rarely measured. The application of PRO is geographically unevenly distributed. Development of PRO instruments, especially those suitable for the Chinese population, may be useful. Further expansion of PROs with respect to the scope of diseases is needed to avoid missing important data.
监管机构、行业同行和国际卫生政策都强调了在临床研究中评估患者报告的结果（PRO）的价值。尽管过去十年中国的临床研究数量有所增加，但对于 PRO 的使用程度知之甚少。
评估在中国进行的随机临床试验中，将 PRO 作为主要和次要结局的应用和特点。
设计、设置和参与者：对 2010 年 1 月 1 日至 2020 年 12 月 31 日在中国进行的干预性临床试验进行了一项横断面研究。评估了从中国临床试验注册中心和 ClinicalTrials.gov 数据库中获得的数据。
根据（1）明确列出 PRO 工具作为结局，（2）在结局中提到患者的主观感受但未明确使用哪些工具进行评估，以及（3）未提及任何 PRO 测量的试验进行分类。从考虑患者感受的试验中提取了研究阶段、设置、参与者年龄和性别数据，以及目标疾病和 PRO 工具名称。
在总共 34033 项试验中，有 6915 项（20.3%）明确列出了使用的明确 PRO 工具，3178 项（9.3%）将 PRO 纳入其结局但未包括评估工具的名称。在注册试验中纳入的 3200 多万人中，有 150 万人（4.7%）的数据通过 PRO 工具进行了科学收集，有 693867 名（2.1%）参与者的主观感受得到了评估。疼痛（16.8%）、癌症（15.6%）和肌肉骨骼症状（13.3%）是通过工具精确收集 PRO 的最常见病症。用于 PRO 测量的最常见工具是视觉模拟量表、简短 36 项健康调查量表和汉密尔顿抑郁量表。
在这项横断面研究中，在中国进行的临床试验中，PRO 的使用在研究期间有所增加。然而，患者的意见似乎仍然很少被测量。PRO 的应用在地理上分布不均。开发 PRO 工具，特别是适合中国人群的工具，可能会有所帮助。需要进一步扩大 PRO 的疾病范围，以避免遗漏重要数据。
