Use of external evidence for design and Bayesian analysis of clinical trials: a qualitative study of trialists' views.
Use of external evidence for design and Bayesian analysis of clinical trials: a qualitative study of trialists' views.
复制标题
DOI：
10.1186/s13063-021-05759-8
复制
发表时间：
2021-11-08
期刊：
影响因子：
2.5
通讯作者：
Jones HE
中科院分区：
文献类型：
--
作者：
Clayton GL;Elliott D;Higgins JPT;Jones HE
在临床试验的设计中，以前研究的证据通常被相对非正式地使用：例如，一项系统的审查，以表明当前证据基础中的差距是否有理由进行新的试验。外部证据可以在试验设计和分析中更正式地使用，明确地将其合成纳入贝叶斯框架。然而，目前还不清楚这种做法在实践中有多普遍，也不清楚它被认为有多大的争议性。在这项定性研究中，我们通过试验的贝叶斯设计或分析，探索了对试验者的态度和在纳入综合外部证据方面的经验。对16名试验者进行了半结构化访谈：13名统计学家和3名临床医生。参与者是通过滚雪球和有目的的抽样在英国的几所大学和试验单位招募的。使用主题分析和恒定比较技术对数据进行分析。试验者在试验设计中以多种方式使用现有证据，例如，证明证据基础中的差距是合理的，并在样本量计算中提供参数。然而，在我们的样本中，没有人报告在贝叶斯框架中使用了这些证据。参与者倾向于将贝叶斯分析等同于纳入关于干预效果的先前信息，而较少意识到纳入关于其他参数的数据的可能性。当被引入这些概念时，许多试验者认为他们可以更多地利用现有数据来为特定情况下的试验设计和分析提供信息。例如，一些人认为现有的数据可以更正式地用于告知背景不良事件发生率，而不是依赖于临床意见是否存在潜在的安全问题。然而，已查明在实践中实施这些方法的几个障碍，包括担心外部数据的相关性、贝叶斯方法的可接受性、对贝叶斯方法和软件缺乏信心以及实际问题，如获取相关数据的困难。尽管试验者认识到，在某些领域，更正式地使用外部证据可能比目前的方法更有利，并有助于敏感性分析，但在实践中使用这种方法仍然存在障碍。网上版载有补充材料，可在10.1186/s13063-021-05759-8查阅。
Evidence from previous studies is often used relatively informally in the design of clinical trials: for example, a systematic review to indicate whether a gap in the current evidence base justifies a new trial. External evidence can be used more formally in both trial design and analysis, by explicitly incorporating a synthesis of it in a Bayesian framework. However, it is unclear how common this is in practice or the extent to which it is considered controversial. In this qualitative study, we explored attitudes towards, and experiences of, trialists in incorporating synthesised external evidence through the Bayesian design or analysis of a trial. Semi-structured interviews were conducted with 16 trialists: 13 statisticians and three clinicians. Participants were recruited across several universities and trials units in the United Kingdom using snowball and purposeful sampling. Data were analysed using thematic analysis and techniques of constant comparison. Trialists used existing evidence in many ways in trial design, for example, to justify a gap in the evidence base and inform parameters in sample size calculations. However, no one in our sample reported using such evidence in a Bayesian framework. Participants tended to equate Bayesian analysis with the incorporation of prior information on the intervention effect and were less aware of the potential to incorporate data on other parameters. When introduced to the concepts, many trialists felt they could be making more use of existing data to inform the design and analysis of a trial in particular scenarios. For example, some felt existing data could be used more formally to inform background adverse event rates, rather than relying on clinical opinion as to whether there are potential safety concerns. However, several barriers to implementing these methods in practice were identified, including concerns about the relevance of external data, acceptability of Bayesian methods, lack of confidence in Bayesian methods and software, and practical issues, such as difficulties accessing relevant data. Despite trialists recognising that more formal use of external evidence could be advantageous over current approaches in some areas and useful as sensitivity analyses, there are still barriers to such use in practice. The online version contains supplementary material available at 10.1186/s13063-021-05759-8.
登录
查看更多内容
影响因子：
2
作者：
Hampson, Lisa V.;Whitehead, John;Eleftheriou, Despina;Brogan, Paul
通讯作者：
Brogan, Paul
影响因子：
1.2
作者：
Connelly, Lynne M.;Peltzer, Jill N.
通讯作者：
Peltzer, Jill N.
影响因子：
3.7
作者：
Page MJ;Higgins JP;Clayton G;Sterne JA;Hróbjartsson A;Savović J
通讯作者：
Savović J
影响因子：
2
作者：
SANDELOWSKI, M
通讯作者：
SANDELOWSKI, M
影响因子：
--
作者：
Lilford, RJ
通讯作者：
Lilford, RJ