一个未实现的承诺:需要改变药物审批程序,使个性化医疗成为现实。

IF 0.3 4区 医学 Q4 FOOD SCIENCE & TECHNOLOGY
Food and drug law journal Pub Date : 2015-01-01
Margaret Foster Riley
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引用次数: 0

摘要

在人类基因组测序之前,个性化医疗药物的广泛使用一直是人们的愿望。最近,人们重新燃起了兴趣;个性化医疗经常出现在新闻中。立法被认为是为了平滑、缩短和激励治疗产品的批准过程。奥巴马总统在国情咨文中提到需要采取新的举措来实现这些目标。但这些举措大多没有考虑到个性化医疗所需要的根本改变。它需要为开发适用于小群体的产品而设计的法定结构,这与我们目前为开发面向大量人口的产品而设计的模型非常不同。目前的审批程序故意不考虑个人疗效。它的目的是鼓励减少临床试验中的变化,而不是拥抱变化。此外,它是基于20世纪的疾病概念,侧重于表型而不是病理生理途径。目前对伴随诊断、孤儿药和批准后研究的关注很重要,但还不够。FDA没有权力要求标准化类型、临床试验设计和广泛的数据报告和共享。是实现个性化医疗目标所必需的。此外,FDA目前的药物审批程序过于冗长和繁琐,无法处理个性化医疗所需的反复反应。如果我们真的想要实现这些目标,我们就需要对权威进行这种根本性的变革。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
An Unfulfilled Promise: Changes Needed to the Drug Approval Process to Make Personalized Medicine a Reality.

The widespread availability of drugs for personalized medicine has been an aspiration since before the human genome was sequenced. Recently, there is renewed interest; personalized medicine is much in the news. Legislation has been considered with the goal of smoothing, shortening and incentivizing the approval process for therapeutic products. President Obama mentioned the need for new initiatives to achieve such goals in the State of the Union address. But most of these initiatives do not consider the fundamental changes that personalized medicine demands. It requires a statutory structure designed for the development of products applicable for small subpopulations that is very different from our current model which is designed for the development of products for large populations. The current approval process is purposely not designed to consider individual efficacy. It is designed to incentivize reduced variation in clinical trials rather than embracing variation. In addition, it is based on twentieth-century notions of disease focused on phenotype rather than on pathophysiologic pathways. Current foci on the development of companion diagnostics, orphan drugs and post-approval study are important but insufficient. FDA does not have the authority to require the type of standardization, clinical trial design and extensive data reporting and sharing that. is needed to achieve the goals for personalized medicine. In addition, FDA's current drug approval process is too lengthy and cumbersome to deal with the iterative responses personalized medicine entails. If we are serious about wanting to achieve these goals, we will need to entertain such fundamental changes in authority.

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来源期刊
Food and drug law journal
Food and drug law journal 医学-食品科技
CiteScore
0.20
自引率
50.00%
发文量
0
审稿时长
>36 weeks
期刊介绍: The Food and Drug Law Journal is a peer-reviewed quarterly devoted to the analysis of legislation, regulations, court decisions, and public policies affecting industries regulated by the U.S. Food and Drug Administration (FDA) and related agencies and authorities, including the development, manufacture, marketing, and use of drugs, medical devices, biologics, food, dietary supplements, cosmetics, veterinary, tobacco, and cannabis-derived products. Building on more than 70 years of scholarly discourse, since 2015, the Journal is published in partnership with the Georgetown University Law Center and the O’Neill Institute for National & Global Health Law. All members can access the Journal online. Each member organization and most individual memberships (except for government, student, and Emeritus members) receive one subscription to the print Journal.
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