Division of Nephrology, Department of Medicine, Duke University School of Medicine, Durham, NC; Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC.
Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC.
Am J Kidney Dis. 2020 Dec;76(6):842-850. doi: 10.1053/j.ajkd.2020.05.026. Epub 2020 Aug 5.
Occasional bursts of discovery and innovation have appeared during the otherwise stagnant past several decades of drug development in nephrology. Among other recent drug discoveries, the unexpected kidney benefits observed with sodium/glucose cotransporter 2 inhibitors may herald a renaissance of drug development in kidney disease. This recent progress highlights the need to further promote and stimulate research and development of promising therapies that may ameliorate the morbidity and mortality associated with kidney disease. To help identify and address barriers to drug development in nephrology, the Duke Clinical Research Institute convened a conference in April 2019 that included stakeholders from academia, industry, government agencies, and patient advocacy. From these discussions, several opportunities were identified to improve every stage of drug development for kidney disease from early discovery to implementation into practice. Key topics reviewed in this article are the utility of interconnected data and site research networks, surrogate end points, pragmatic and adaptive trial designs, the promising uses of real-world data, and methods to improve the generalizability of trial results and uptake of approved drugs for kidney-related diseases.
在过去几十年相对停滞的肾脏病药物研发中，偶尔会出现一些突破性发现和创新。在最近的其他药物发现中，钠/葡萄糖协同转运蛋白 2 抑制剂观察到的出人意料的肾脏益处，可能预示着肾脏病药物研发的复兴。这一最新进展凸显了需要进一步推动和激励有希望的治疗方法的研究和开发，这些方法可能改善与肾脏病相关的发病率和死亡率。为了帮助确定和解决肾脏病药物研发中的障碍，杜克临床研究所于 2019 年 4 月召开了一次会议，会议邀请了来自学术界、工业界、政府机构和患者权益倡导者的利益相关者。从这些讨论中，确定了几个机会，可以改善肾脏病药物研发的各个阶段，从早期发现到实际应用。本文回顾了关键主题，包括相互关联的数据和站点研究网络、替代终点、实用和适应性试验设计、真实世界数据的应用前景，以及改善试验结果的普遍性和批准用于肾脏相关疾病的药物的采用率的方法。
