Bakker Jessie P, Izmailova Elena S, Clement Aude, Hoffmann Steven, Leptak Christopher, Menetski Joseph P, Wagner John A
Koneksa Health, New York, New York, USA.
F.Hoffmann-La Roche Ltd, Basel, Switzerland.
Clin Pharmacol Ther. 2025 Jan;117(1):56-72. doi: 10.1002/cpt.3398. Epub 2024 Aug 15.
Despite widespread interest and substantial investment in the adoption of sensor-based digital health technologies (sDHTs) for remote data capture in drug development trials, no drug has been approved based on an sDHT-derived primary endpoint in the United States (US). One reason for this lack of advancement is the complexity of obtaining regulatory endorsement for those endpoints within current US regulatory pathways. The goal of our review is to describe the two choices currently available to pharmaceutical study Sponsors: (i) they may navigate the traditional route of compiling the evidence to support the sDHT-derived endpoint in their investigational new drug (IND) application, requiring specific expertise and substantial resources; or (ii) they may navigate the drug development tool (DDT) pathway with the goal of qualifying their sDHT-derived endpoint as a biomarker or clinical outcome assessment applicable to a broader context of use (COU), either alone or as part of a partnership or consortium. We describe the nuances of each pathway; the evidentiary requirements for supporting an sDHT-derived endpoint and the technology used to capture it; and the impact that an sDHT's regulatory status may have on a Sponsor's decision to use it for data capture. By systematically comparing the IND and DDT pathways, our over-arching goals are to support the increasing deployment of sDHTs within the clinical research setting and help advance regulatory science in the field of digital medicine.
尽管人们对在药物研发试验中采用基于传感器的数字健康技术（sDHTs）进行远程数据采集有着广泛兴趣并投入了大量资金，但在美国，尚无药物基于sDHT衍生的主要终点获得批准。缺乏进展的一个原因是在美国当前的监管途径中，获得这些终点的监管认可存在复杂性。我们综述的目的是描述制药研究申办方目前可用的两种选择：（i）他们可以遵循传统途径，在其研究性新药（IND）申请中收集证据以支持sDHT衍生的终点，这需要特定的专业知识和大量资源；或者（ii）他们可以遵循药物研发工具（DDT）途径，目标是将其sDHT衍生的终点鉴定为适用于更广泛使用背景（COU）的生物标志物或临床结局评估，无论是单独使用，还是作为合作伙伴关系或联盟的一部分。我们描述了每种途径的细微差别；支持sDHT衍生终点的证据要求以及用于捕获该终点的技术；以及sDHT的监管状态可能对申办方决定使用它进行数据采集产生的影响。通过系统地比较IND和DDT途径，我们的总体目标是支持sDHT在临床研究环境中的更多应用，并帮助推进数字医学领域的监管科学。
