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

Abstract

IMPORTANCE 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. OBJECTIVETo evaluate the application and characteristics of PRO instruments as primary and secondary outcomes in randomized clinical trials in China. DESIGN, SETTING, AND PARTICIPANTSA 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. MAIN OUTCOMES AND MEASURESTrials 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. RESULTS 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. CONCLUSIONS AND RELEVANCEIn 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.

Patient-Reported Outcome Measures—Challenges and Opportunities for China

“…As such, the discovery of new insights reported by Zhou et al 6 requires more widespread and consistent deployment of well-constructed PROMs that provide the best understanding of which…”

“…Author affiliations and article information are listed at the end of this article. The benefit of this well-done study by Zhou et al 6 is that it exposes both challenges and opportunities for the all-important future of PROM development and implementation-not just for the PRC, but for the entire international field. Fortunately, several reliable and publicly available resources and repositories for PROMs now exist widely, such as Patient-Reported Outcomes Measurement Information System, Patient-Reported Outcomes and Quality-of-Life Instrument database, and Online Guide to Quality-of-Life Assessment.…”

“…Fortunately, several reliable and publicly available resources and repositories for PROMs now exist widely, such as Patient-Reported Outcomes Measurement Information System, Patient-Reported Outcomes and Quality-of-Life Instrument database, and Online Guide to Quality-of-Life Assessment. 5,6 The joint initiative between the Consensus-Based Standards for the Selection of Health Measurement Instruments initiative and the Core Outcome Measures in Effectiveness Trials initiative helps facilitate agreement among PROMsfocused stakeholders with regard to the selection of outcomes (eg, constructs or domains) and outcome measurement instruments for clinical trials. 7 Measure development and evaluation standards are also in use by consensus-driven groups (eg, the National Quality Forum) for the various and complex design domains of true PROMs, including content validity (including face validity), structural validity, internal consistency, reliability, measurement error, hypotheses testing, cross-cultural validity, criterion validity, and responsiveness.…”

“…Using a cross-sectional survey of interventional clinical trials using data from the Chinese Clinical Trial Registry and the ClinicalTrials.gov databases, Chinese researchers have evaluated the current applications of PROMs in clinical trials in the PRC . As the authors note, this study documents the major increase from 2010 to 2020 in the number of clinical trials originating in the PRC that include the application and characteristics of PRO instruments and PROMs as primary and secondary outcomes in clinical trials across China.…”

“…Much is required to generate better quality of evidence and measurement of cost-effectiveness for guideline-directed medical therapies indicated for major chronic conditions and other, less common diseases. As such, the discovery of new insights reported by Zhou et al requires more widespread and consistent deployment of well-constructed PROMs that provide the best understanding of which PROs matter most to patients and other subsequent relevant stakeholders interested in the resultant data.…”