Application of Patient-Reported Outcome Measurements in Clinical Trials in China

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.

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.