Saville Benjamin R, Burkhoff Daniel, Abraham William T
Berry Consultants, LLC Austin TX USA.
Department of Biostatistics Vanderbilt University Medical Center Nashville TN USA.
J Am Heart Assoc. 2024 Feb 6;13(3):e033255. doi: 10.1161/JAHA.123.033255. Epub 2024 Jan 23.
The Breakthrough Devices Program of the US Food and Drug Administration has accelerated the development and evaluation of medical devices for patients with heart failure. One such device is the Optimizer Smart System, which the US Food and Drug Administration approved in 2019.
The Optimizer device was evaluated in a pivotal randomized clinical trial (FIX-HF-5C [Confirmatory Randomized Trial Evaluating the Optimizer System]) that leveraged Bayesian borrowing of external data to reduce the sample size and determine therapeutic device benefit versus continued medical therapy. Bayesian borrowing is explained in the context of the FIX-HF-5C trial, including an overview of the statistical methodologies, regulatory considerations, and interpretations of trial results.
The US Food and Drug Administration Breakthrough Devices Program and novel Bayesian statistical methodology accelerated the path to regulatory approval and patient access to a potentially lifesaving device and may serve as a model for future clinical trials.
美国食品药品监督管理局的突破性设备计划加速了心力衰竭患者医疗设备的开发和评估。其中一种设备是Optimizer智能系统，美国食品药品监督管理局于2019年批准了该系统。
在一项关键的随机临床试验（FIX-HF-5C [评估Optimizer系统的验证性随机试验]）中对Optimizer设备进行了评估，该试验利用外部数据的贝叶斯借用方法来减少样本量，并确定治疗设备与持续药物治疗相比的益处。在FIX-HF-5C试验的背景下对贝叶斯借用进行了解释，包括统计方法、监管考虑因素以及试验结果的解读概述。
美国食品药品监督管理局的突破性设备计划和新颖的贝叶斯统计方法加速了监管批准的进程以及患者获得潜在救命设备的机会，并且可能成为未来临床试验的典范。
