In the modern pharmaceutical and biotechnology landscape, regulatory authorities across the globe are putting greater emphasis on the patient experience in treatment development. As a recent example of this global impetus, China’s National Medical Products Administration (NMPA) published new technical guidance in January 2025 on the application of Patient-Reported Outcomes (PROs) in clinical trials for rheumatic diseases (RD).
Issued in January 2025, the "Technical Guidelines for the Application of Patient-Reported Outcomes in Clinical Trials on Rheumatic Diseases" provides pharmaceutical companies and researchers with specific directions on implementing PRO measures in rheumatic disease studies. The issuance of this guideline is significant given that as recently as 2022, fewer than 10% of clinical trials conducted in China included PROs in their outcomes. In fact, from more than 32 million participants, scientifically collected PRO data was available for fewer than 5% of patients.[1]
[1] Zhou H, Yao M, Gu X, Liu M, Zeng R, Li Q, Chen T, He W, Chen X, Yuan G. Application of Patient-Reported Outcome Measurements in Clinical Trials in China. JAMA Netw Open. 2022 May 2;5(5):e2211644. doi: 10.1001/jamanetworkopen.2022.11644. PMID: 35544134; PMCID: PMC9096600.*
A big step forward for patient-centered trials in China
NMPA’s guidance identifies five dimensions where PROs add significant value in rheumatic disease trials:
1. Disease symptom severity
Due to the frequency of symptoms such as pain and swelling experienced by patients suffering from RD, the guidance highlights that PROs can be instrumental in capturing subjective feelings of these symptoms. As an example, the Visual Analogue Scale (VAS) is a common PRO used to assess pain.
2. Physical function evaluation
The loss of physical function is an irreversible symptom of RD. NMPA’s guidance specifically references the Health Assessment Questionnaire Disability Index (HAQ-DI) for rheumatoid arthritis and the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) for osteoarthritis as validated tools.
3. Impact of symptoms on quality of daily life
The guidance emphasizes capturing how rheumatic diseases affect patients' daily activities and social functioning across different disease stages, as these may be entirely subjective and only knowable from a patient’s perspective.
4. Patient satisfaction with treatment
The NMPA recognizes the importance of PROs in evaluating patients’ subjective views on whether a given treatment is having an impact.
5. Medication benefits and risks
Lastly, the guidance emphasizes that due to the limitations of study cycles, some adverse events may go unreported. PROs can be instrumental in building more comprehensive safety and efficacy profiles for treatments.
Validated COAs for rheumatic diseases
In addition to well-known tools like the HAQ-DI and WOMAC mentioned above, several other COAs provide valuable insights in rheumatic disease trials. For example, the MultiDimensional Health Assessment Questionnaire (MDHAQ) is used to assess physical function and fatigue, pain, and psychological well-being—capturing a more complete picture of disease burden. Another COA, the QUOTE-Rheumatic-Patients instrument, helps evaluate patients’ satisfaction with the quality of care received, giving insight into treatment responsiveness. Another example is the SWE-RES-23 scale, which gauges patient empowerment, reflecting how informed and in control patients feel about managing their condition. These tools, when adapted and validated for use in China, can support the NMPA’s goal of making PRO data more mainstream within Chinese clinical trials.
Implementation considerations specific to the Chinese market
The NMPA provides important practical considerations for researchers implementing PROs within their trials. Most notably, the guidance emphasizes the need for researchers to confirm the validity of a PRO for the Chinese population, given that the majority of PRO instruments available today are translations of foreign language scales. If any further cultural adaptation is required, the guidance recommends developing localized scales specific to the Chinese population.
Finally, the guidance highlights that while PROs are instrumental for gathering patients’ subjective feelings regarding treatment, they should be sparingly used as primary endpoints due to the variability with subjective data.
State-of-the-art COA platform, ePROVIDE™, available in China
The release of this new NMPA guidance marks a clear opportunity for sponsors to embrace more patient-centered research strategies that leverage scientifically-sound and culturally-relevant PROs.
Mapi Research Trust, a leading authority on the use of clinical outcome assessments in trials, is proud to announce that the ePROVIDE™ platform is now available in Chinese.
The availability of eProvide™ within the Chinese market marks a major step forward in our commitment to enabling patient-centered research worldwide. With ePROVIDE™, sponsors conducting studies in China can:
- Ensure regulatory alignment with NMPA expectations
- Access validated Chinese-language PRO instruments
- Facilitate culturally appropriate implementation of COAs in clinical trials
Explore ePROVIDE™ today to access our library of validated assessment tools, including all the tools described within this document, and implementation best practices in Chinese.