以患者为中心的药物研发策略

介绍美国食品及药物管理局（FDA）以患者为中心的药物开发思路，展示了不同疾病、治疗收益、使用环境分别对应的临床结局评估（COA）工具，整理出5个已获批患者报告结局（PRO）工具的相关信息，并归纳了2010—2015年FDA获批新药中，应用PRO评估工具支持说明书相关声明的药品信息。旨在为业界选择适合的COA类型（PRO、ClinRO、ObsRO或PerfO评估工具）应用于药物研发提供借鉴，并建议将COA工具用于儿科、罕见病、认知障碍相关疾病以及中药的临床疗效评估；同时也为将患者的观点纳入药品监管流程提供参考。

Patient-focused drug development strategies

The FDA''s patient-focused drug development ideas was introduces, showed the clinical outcome assessment (COA) measures for different diseases, treatment benefits, and contexts of use, and summarizes the relevant information of 5 approved patient-reported outcome (PRO) measures, and sums up the drug information of the FDA approved new drugs in 2010-2015, using the PRO measures to support labeling. The aim was to provide the industry with a suitable type of COA (PRO, ClinRO, ObsRO, or PerfO) for drug development, and to recommend the use of COA measures for pediatrics, rare diseases, cognitively impaired, and clinical efficacy evaluation of Traditional Chinese Medicine. It also provides a reference for incorporating patient perspectives into the drug regulatory process.