Drug Approval Package에서의 환자자기평가결과 사용 현황 분석: FDA 2017-2023 허가 의약품을 중심으로

Analysis of Patient-Reported Outcome use status in Drug Approval Package: Focusing on novel drugs approved by FDA 2017-2023

The aim of this study is to assess the current utilization of Patient-Reported Outcomes (PROs) as efficacy endpoints in clinical trials for approving new drugs. To achieve this goal, the study focused on products where PROs played a significant role in demonstrating efficacy. According to the FDA’s official annual report on new drug approvals, which spans from July 2017 to May 2023, 21.9% of the 288 approved drugs utilized PRO as a main endpoint to demonstrate their efficacy. At the time of product approval, we identified 128 clinical trials that served as the primary evidence supporting the efficacy of drugs, utilizing a total of 199 PRO measures across these trials. When PRO measures were categorized based on measurement concepts, such as symptoms, function, quality of life, and others, it was discovered that measures capturing symptoms accounted for almost half of the total. On the other hand, the highest percentage, approximately 63%, comprised of measures included in the drug label. Furthermore, disease-specific PRO measures accounted for approximately 66% of the total and encompassed around 80% of the PRO measures included in the drug label. To ensure effective utilization of PRO in clinical trials, it is necessary to develop diverse training programs for reviewers and researchers. These programs will facilitate the review of PRO by regulatory agencies and promote the utilization of PROs in clinical trials.