• 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Orphanet journal of rare diseases >Critical appraisal of arguments for the delayed-start design proposed as alternative to the parallel-group randomized clinical trial design in the field of rare disease
【24h】

Critical appraisal of arguments for the delayed-start design proposed as alternative to the parallel-group randomized clinical trial design in the field of rare disease

机译：对罕见疾病领域中的平行组随机临床试验设计的替代建议的延迟启动设计的论点的严格评估

获取原文
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

BackgroundA number of papers have proposed or evaluated the delayed-start design as an alternative to the standard two-arm parallel group randomized clinical trial (RCT) design in the field of rare disease. However the discussion is felt to lack a sufficient degree of consideration devoted to the true virtues of the delayed start design and the implications either in terms of required sample-size, overall information, or interpretation of the estimate in the context of small populations. ObjectivesTo evaluate whether there are real advantages of the delayed-start design particularly in terms of overall efficacy and sample size requirements as a proposed alternative to the standard parallel group RCT in the field of rare disease. MethodsWe used a real-life example to compare the delayed-start design with the standard RCT in terms of sample size requirements. Then, based on three scenarios regarding the development of the treatment effect over time, the advantages, limitations and potential costs of the delayed-start design are discussed. ResultsWe clarify that delayed-start design is not suitable for drugs that establish an immediate treatment effect, but for drugs with effects developing over time, instead. In addition, the sample size will always increase as an implication for a reduced time on placebo resulting in a decreased treatment effect. ConclusionsA number of papers have repeated well-known arguments to justify the delayed-start design as appropriate alternative to the standard parallel group RCT in the field of rare disease and do not discuss the specific needs of research methodology in this field. The main point?is that a limited time on placebo will result in an underestimated treatment effect and, in consequence, in larger sample size requirements compared to those expected under a standard parallel-group design. This also impacts on benefit-risk assessment.
机译：背景技术在罕见病领域，许多论文已经提出或评估了延迟启动设计，以替代标准的两臂平行组随机临床试验（RCT）设计。但是，人们认为该讨论缺乏足够的考虑来考虑延迟启动设计的真正优点以及所涉及的影响，无论是所需的样本量，总体信息还是在人口较少的情况下对估计的解释。目的评估延迟启动设计是否真正具有优势，特别是在总体功效和样本量要求方面，作为罕见病领域中标准平行组RCT的替代方案。方法我们使用一个真实的示例，根据样本量要求，将延迟启动设计与标准RCT进行了比较。然后，基于关于随时间推移产生治疗效果的三种情况，讨论了延迟启动设计的优点，局限性和潜在成本。结果我们澄清了延迟启动设计不适用于具有立竿见影疗效的药物，而是适用于随着时间而发展的药物。另外，样本量将总是增加，这意味着减少了对安慰剂的时间，从而导致治疗效果降低。结论许多论文重复了著名的论据，以证明延迟启动设计是罕见病领域中标准平行组RCT的适当替代方案，并且没有讨论该领域研究方法的具体需求。要点是，有限的时间使用安慰剂会导致治疗效果被低估，因此与标准平行组设计所预期的相比，需要更大的样本量。这也影响到利益风险评估。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号