推进分散和实用的临床试验,以提高临床试验证据的代表性和更大的普遍性。

IF 3.6 4区 医学 Q2 PHARMACOLOGY & PHARMACY
Caroline Marra, Krisda H Chaiyachati, Vindell Washington, Amy P Abernethy
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引用次数: 0

摘要

尽管努力提高临床试验的研究公平性,但缺乏代表性继续威胁着临床试验证据的普遍性,并导致一些伦理和经济后果。分散临床试验(dct)和实用临床试验(pct)是一种新型临床试验模式,利用技术实现替代数据收集方法并将研究整合到临床护理中,它们在解决代表性挑战方面前景广阔,但也面临一些局限性。利用技术和临床护理环境进行试验访问和收集试验数据,从本质上限制了无法获得可靠技术和一致医疗服务的人的参与。此外,从早期产品开发阶段进行的试验到产品获得批准后进行的试验,需要在整个临床试验范围内提高代表性,但可以提高代表性的DCT和PCT要素更有可能在新医疗产品获得监管部门批准的重要证据已经产生之后才被部署。我们提出了三种解决方案,以帮助确保dct和pct的定义方面增加代表性和在产品生命周期的所有阶段进行的临床试验中产生的证据的普遍性。我们建议,应集中努力与监管机构开展协作,以确定数据标准,并在使用替代数据源和收集方法时验证结果,为开展技术促进的试验创建技术支持资源和透明流程,并将卫生公平原则纳入用于促进dct和pct的基于技术的工具的设计和部署。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Advancing Decentralized and Pragmatic Clinical Trials as a Path Toward Improved Representativeness and Greater Generalizability of Clinical Trial Evidence.

Despite efforts to improve research equity in clinical trials, a lack of representativeness continues to threaten the generalizability of clinical trial evidence and leads to several ethical and economic consequences. Decentralized clinical trials (DCTs) and pragmatic clinical trials (PCTs), novel clinical trial models that use technology to enable alternative data collection methods and integrate studies into clinical care, hold great promise for addressing representativeness challenges but also face several limitations. Leveraging technology and clinical care settings to conduct trial visits and collect trial data inherently limits participation from people without reliable access to technology and consistent medical care. Further, representativeness needs to be improved across the full spectrum of clinical trials, from trials conducted in early product development phases to trials conducted after a product is approved, but the DCT and PCT elements that can improve representativeness are more likely to be deployed after seminal evidence for a new medical product's regulatory approval has already been generated. We propose three solutions to help ensure the defining aspects of DCTs and PCTs increase representativeness and the generalizability of evidence generated in clinical trials conducted at all phases in the product lifecycle. We suggest that efforts should be centered around collaborative work with regulators to define data standards and validate outcomes when alternative data sources and collection methods are used, the creation of technical support resources and transparent processes for the conduct of trials facilitated by technology, and the incorporation of health equity principles into the design and deployment of technology-based tools used to facilitate DCTs and PCTs.

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来源期刊
Clinical therapeutics
Clinical therapeutics 医学-药学
CiteScore
6.00
自引率
3.10%
发文量
154
审稿时长
9 weeks
期刊介绍: Clinical Therapeutics provides peer-reviewed, rapid publication of recent developments in drug and other therapies as well as in diagnostics, pharmacoeconomics, health policy, treatment outcomes, and innovations in drug and biologics research. In addition Clinical Therapeutics features updates on specific topics collated by expert Topic Editors. Clinical Therapeutics is read by a large international audience of scientists and clinicians in a variety of research, academic, and clinical practice settings. Articles are indexed by all major biomedical abstracting databases.
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