JMIR Publications

ABSTRACT

Background:

International health policy and researchers have emphasized the value of evaluating patient-reported outcomes (PROs) in clinical studies, while the characteristics of patient-reported outcomes in tumors in China are not well established.

Objective:

The purpose of this study was to assess the application and characteristics of PROs instruments as primary and/or secondary outcomes in randomized clinical trials of tumors in mainland China.

Methods:

This cross-sectional study used data from tumor-randomized clinical trials in mainland China from January 1, 2010, to June 30, 2022. The ClinicalTrials.gov databases and Chinese Clinical Trial Registry were selected as the databases. Trials were classified according to those that (1) listed PRO instruments as primary outcomes, (2) listed PRO instruments as secondary outcomes, (3) listed PRO instruments as coprimary outcomes, and (4) did not mention any PRO instruments. Data on study phase, setting, regions, center, participant age and gender, target diseases, and names of the PRO instruments were extracted from trials. The target diseases involved in the trials were grouped by the American Joint Committee on Cancer (AJCC) Cancer Staging Manual 8th edition.

Results:

Among a total of 6445 trials, 2429 (37.7%) trials used the PRO instruments in their outcomes. Among them, 27.0% (656/2429) trials listed PRO instruments as primary outcomes, 52.5% (1275/2429) trials used as secondary outcomes, and 20.5% (498/2429) trials used as coprimary outcome in their outcomes. From more than 2.1 million people included in the trials, data on 542 895 (25.2%) patients were collected by PRO instruments as primary or secondary outcomes, and coprimary outcomes were assessed for 75 275 (3.5%) patients. The most common conditions for which used explicitly specified PRO instruments were thorax tumor (18.7%), gastrointestinal tract tumor (13.0%), and lower gastrointestinal tract tumor (12.4%). The most common instruments for PRO measurements were the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30, visual analog scale, numeric rating scale, Traditional Chinese Medicine symptom scale, and Pittsburgh Sleep Quality Index.

Conclusions:

Over the past several years, the use of PROs increased in tumor-randomized clinical trials conducted in mainland China. Nonetheless, the patient's opinion still seems to be rarely measured. Specific PRO instruments should be widely used in different categories of tumor disease and there is room for improvement in standard PROs.