适应性设计的特点和法规,从2008年到2020年:欧洲药品管理局批准概述。

IF 0.9 4区 医学 Q4 PHARMACOLOGY & PHARMACY International journal of clinical pharmacology and therapeutics Pub Date : 2023-10-01 DOI:10.5414/CP204422
Xiaowei Huang, Jianbin Ma, Zhenzhen Lu, Lihong Huang
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引用次数: 0

摘要

目的:本研究的目的是确定和表征欧洲药品管理局(EMA)在临床试验中从适应性设计中获得的所有批准,并提供这些药物的最新状态。材料和方法:使用与自适应设计相关的合适关键词列表,在2008年至2020年期间的年度报告EMA数据库中确定了相关文件。我们记录了药物批准的试验特征,并使用Fisher精确测试来比较这些特征。结果:共确定了1054项EMA批准,计划进行适应性试验设计的EMA批准比例从2008-2012年的1.85%增加到2017-2020年的6.19%。在91份包含自适应设计的原始EMA文件中,共确定了41项批准。2017年后,临床试验中使用的自适应设计类型有所增加,其中最常见的类型是最常见的(17/41)。大多数批准(32/41)包括关键试验,大多数评估没有加快(38/41)。在计划进行的32项适应性设计验证性试验中,进行额外监测(AM)的比例显著增加(p结论:2008年至2020年期间,观察到与适应性设计相关的EMA批准数量增加的趋势。可以鼓励采用不同类型的适应性试验设计来指定临床试验,特别是抗肿瘤药物;同时,对正在进行的具有适应性设计的抗肿瘤药物的试验似乎进行了更严格的监测规定。
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The characteristics and regulations of adaptive designs from 2008 to 2020: An overview of European Medicines Agency approvals.

Objective: The aim of this study was to identify and characterize all European Medicines Agency (EMA) approvals derived from adaptive designs in clinical trials and to provide an update of the current status of these drugs.

Materials and methods: Relevant files were identified in the EMA database for annual reports for the period between 2008 and 2020 using a list of suitable keywords related to adaptive designs. We recorded trial characteristics from drug approvals and used Fisher exact test to compare the characteristics.

Results: A total of 1,054 EMA approvals were identified, and the percentage of EMA approvals planned with adaptive trial designs increased from 1.85% in the period 2008 - 2012 to 6.19% in between 2017 - 2020. A total of 41 approvals were identified among 91 original EMA files that contained adaptive designs. The types of adaptive designs used in clinical trials increased after 2017 where the most common type used was the most common (17/41). Most approvals (32/41) comprised pivotal trials, and most assessments had not been accelerated (38/41). Of 32 confirmatory trials planned with adaptive designs, the proportion of those with additional monitoring (AM) increased significantly (p < 0.0001) from 0% in the 2008 - 2012 period to 90.48% in the 2017 - 2020 period. The percentage of approved antitumor drugs in approved drugs in ongoing clinical trials was 82.35%, compared to 20.83% in trials that were completed (p = 0.0001). The proportion of drug approved but where clinical trials were still ongoing in companies requiring post-authorization safety studies (PASSs) or post-authorization efficacy studies (PAESs) or who were granted conditional marketing authorization (CMA) significantly differed from the group of drugs approved where clinical trials were completed (p = 0.0230).

Conclusion: A trend showing an increased number of EMA approvals related to adaptive designs was observed for the period from 2008 to 2020. Different types of adaptive trial designs could be encouraged for the designation of clinical trials, especially for antitumor drugs; meanwhile, more stringent monitoring regulations seemed to be conducted for ongoing trials of antitumor drugs with adaptive design.

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来源期刊
CiteScore
1.70
自引率
12.50%
发文量
116
审稿时长
4-8 weeks
期刊介绍: The International Journal of Clinical Pharmacology and Therapeutics appears monthly and publishes manuscripts containing original material with emphasis on the following topics: Clinical trials, Pharmacoepidemiology - Pharmacovigilance, Pharmacodynamics, Drug disposition and Pharmacokinetics, Quality assurance, Pharmacogenetics, Biotechnological drugs such as cytokines and recombinant antibiotics. Case reports on adverse reactions are also of interest.
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