主要使用适应性设计,但在早期药物试验阶段报告不足
Yuning Wang1,2,3, Minghong Yao1,2,3, Jiali Liu1,2,3
1Institute of Integrated Traditional Chinese and Western Medicine, West China Hospital, Chinese Evidence-based Medicine Center and Chinese Cochrane Center, Sichuan University, Chengdu, 610041, China.
BMC medical research methodology
|June 5, 2024
概括
适应性设计在药物试验中越来越多地用于提高效率,但报告质量仍然不理想. 需要进一步努力,以提高适应性试验报告的透明度和完整性,以改善药物开发.
科学领域:
- 临床试验方法论 临床试验方法论
- 药学研究 药学研究
- 生物统计学 生物统计学
背景情况:
- 经典的随机对照试验 (RCT) 面临成本和效率方面的挑战.
- 适应性设计为加速药物开发提供了潜在的解决方案.
- 在药物RCT中适应性设计的应用和报告质量需要调查.
研究的目的:
- 总结适应性药物试验的流行病学特征.
- 评估适应性药物试验的报告质量,使用适应性设计CONSORT扩展 (ACE) 检查清单.
主要方法:
- 在主要数据库 (MEDLINE,EMBASE,CENTRAL,ClinicalTrials.gov) 进行系统的文献搜索,截至2020年1月.
- 包括明确使用自适应设计的药物RCT.
- 使用ACE检查清单提取流行病学特征和评估报告质量;回归分析以确定与报告质量相关的因素.
主要成果:
- 包括108项适应性试验,显示应用越来越多,主要是在II期试验中.
- 适应性设计使用的关键原因包括加速试验,决策,参与者利益和样本大小减少.
- 报告质量低于最佳,在报告关键适应性试验要素 (如统计分析计划和保密措施) 中存在重大差距.
结论:
- 适应性设计在早期药物试验中越来越多地被采用.
- 适应性试验的总体报告质量目前不够理想.
- 报告的实质性改进是必要的,以充分利用适应性试验设计的好处.
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