Genetically Enriched Clinical Trials for Precision Development of Noncancer Therapeutics: A Scoping Review.

IF 11.2 1区 医学 Q1 PHARMACOLOGY & PHARMACY Annual review of pharmacology and toxicology Pub Date : 2024-09-30 DOI:10.1146/annurev-pharmtox-031524-021631
Benoît Delabays, Chiara De Paoli, Andrea Miller-Nesbitt, Vincent Mooser
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Abstract

Genetically driven clinical trial enrichment has been proposed to accelerate and reduce the cost of developing new therapeutics. Usage of this approach has not been comprehensively reviewed. We searched Ovid MEDLINE, Embase, Web of Science, Cochrane Library, ClinicalTrials.gov, and WHO ICTRP for articles published between 2010 and 2023. Excluding absorption, distribution, metabolism, and elimination pharmacogenetic studies and anti-infectives, we found 95 completed, 4 terminated, and 22 ongoing prospective genetically enriched trials on 110 drugs for 48 nononcology, nonrare syndromic indications. Trial sizes ranged from 4 to 6,147 participants (median 72) and covered numerous disease areas, particularly neurology (30), metabolism (22), and psychiatry (17). Fifty-six completed studies (60%) met their primary end point. Overall, this scoping review demonstrates that genetically enriched trials are feasible and scalable across disease areas and provide critical information for further development, or attrition, of investigational drugs. Large, appropriately designed disease-, hospital-, or population-based biobanks will undoubtedly facilitate this type of precision drug development approach.

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用于非癌症治疗药物精准开发的基因丰富临床试验:范围综述》。
有人提出通过基因驱动的临床试验富集来加快新疗法的开发并降低其成本。目前尚未对这种方法的使用情况进行全面审查。我们检索了 Ovid MEDLINE、Embase、Web of Science、Cochrane Library、ClinicalTrials.gov 和 WHO ICTRP 中 2010 年至 2023 年间发表的文章。除去吸收、分布、代谢和消除药物遗传学研究以及抗感染药物,我们找到了 95 项已完成、4 项已终止和 22 项正在进行的前瞻性基因富集试验,涉及 110 种药物,用于 48 种非肿瘤、非罕见综合症适应症。试验规模从 4 人到 6,147 人不等(中位数为 72 人),涉及众多疾病领域,尤其是神经病学(30 项)、新陈代谢(22 项)和精神病学(17 项)。56项已完成的研究(60%)达到了主要终点。总之,本次范围界定综述表明,基因富集试验在各个疾病领域都是可行的、可扩展的,并为研究药物的进一步开发或损耗提供了重要信息。设计合理的大型疾病、医院或人群生物库无疑将促进这类精准药物开发方法的发展。
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来源期刊
CiteScore
27.80
自引率
0.00%
发文量
53
期刊介绍: Since 1961, the Annual Review of Pharmacology and Toxicology has been a comprehensive resource covering significant developments in pharmacology and toxicology. The journal encompasses various aspects, including receptors, transporters, enzymes, chemical agents, drug development science, and systems like the immune, nervous, gastrointestinal, cardiovascular, endocrine, and pulmonary systems. Special topics are also featured in this annual review.
期刊最新文献
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