患者報告アウトカムを臨床研究に組み込むためのガイドラインの整備現状

抄録

The incorporation of patient-reported outcomes (PROs) into clinical research has gained increasing attention in parallel with the global shift toward patient-focused drug development. PROs are essential for evaluating outcomes that are subjective in nature and not adequately captured by clinician-reported assessments or objective clinical indicators, such as symptoms, treatment-related adverse events, health-related quality of life, and patient experience. When collected using well-validated patient-reported outcome measures (PROMs) within appropriately designed studies, PRO data may inform benefit-risk assessment and support regulatory decision-making.

The scientific and ethical use of PROs requires methodological rigor across all stages of clinical research, including study planning, outcome selection, data collection and management, statistical analysis, interpretation, and reporting. Over the past three decades, international regulatory authorities, academic societies, and expert consortia have developed a comprehensive set of PRO-related guidelines. These include guidance for protocol development (SPIRIT-PRO), conceptual frameworks for outcome selection (ISPOR Clinical Outcome Assessment framework), standards for evaluating the measurement properties of PROMs (COSMIN guidelines), ethical guidance for PRO use, recommendations for electronic PRO implementation and data management, statistical analysis recommendation (SISAQOL), and reporting guidance (CONSORT-PRO).

This short report summarizes the current landscape of PRO-related guidelines and highlights recent developments in Japan. The dissemination and practical implementation of these guidelines in the Japanese clinical research context are critical to ensuring high-quality, ethically sound, and patient-centered use of PROs in both clinical trials.