From the Formation of Conceptual Framework to Regulatory Decision-Making: Considerations for the Developments of Patient-Reported Outcome Instruments

Kai Cao,1,2 Xu-Yuan Quan,1 Yan Hou1,3,4 1Department of Biostatistics, School of Public Health, Peking University, Beijing, 100191, People’s Republic of China; 2Beijing Institute of Ophthalmology, Beijing Tongren Hospital, Capital Medical University, Beijing, 100005, People’s Republic of China; 3Beijing Cancer Hospital, Beijing, 100142, People’s Republic of China; 4Peking University Clinical Research Center, Institute of Advanced Clinical Medicine, Peking University, Beijing, 100191, People’s Republic of ChinaCorrespondence: Yan Hou, Email [email protected]: In recent years, patient-focused drug development (PFDD) has received widespread attention as a new paradigm in clinical trials. The PFDD emphasizes patients are partners in research projects, where patients can participate in research design, implementation, and outcome measurement, rather than just providing data. PFDD has shown great value in the research and development of pharmaceutical products, such as in accelerating the process of patient enrollment and improving the success rate of drug approval. Many countries and regions, including the United States, China, and Europe, have issued relevant regulatory policies and guidelines related to PFDD, covering study design, implementation, and risk–benefit assessment. The core of PFDD implementation is clinical outcome assessment (COA), of which patient-reported outcome (PRO) is most common. As far as the US Food and Drug Administration is concerned, there are numerous COA tools waiting for qualification, but currently all qualified are PROs. This review focuses on PRO and explores the key elements of PRO instruments’ development, application, and inclusion in regulatory decision-making.Keywords: patient-reported outcome, patient-focused, drug, development, clinical trial