介紹美國(guó)食品及藥物管理局（FDA）以患者為中心的藥物開(kāi)發(fā)思路，展示了不同疾病、治療收益、使用環(huán)境分別對(duì)應(yīng)的臨床結(jié)局評(píng)估（COA）工具，整理出5個(gè)已獲批患者報(bào)告結(jié)局（PRO）工具的相關(guān)信息，并歸納了2010—2015年FDA獲批新藥中，應(yīng)用PRO評(píng)估工具支持說(shuō)明書相關(guān)聲明的藥品信息。旨在為業(yè)界選擇適合的COA類型（PRO、ClinRO、ObsRO或PerfO評(píng)估工具）應(yīng)用于藥物研發(fā)提供借鑒，并建議將COA工具用于兒科、罕見(jiàn)病、認(rèn)知障礙相關(guān)疾病以及中藥的臨床療效評(píng)估；同時(shí)也為將患者的觀點(diǎn)納入藥品監(jiān)管流程提供參考。

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.

國(guó)家自然科學(xué)基金資助項(xiàng)目（81503347，81503068）