Medical and Pharmaceutical Statistics Research Unit, Department of Mathematics and Statistics, Lancaster University, Lancaster, UK.
Clin Trials. 2013 Apr;10(2):344-6. doi: 10.1177/1740774512474375. Epub 2013 Feb 1.
In recent years, the success rate of confirmatory studies has been poor resulting in more emphasis on the conduct of exploratory studies. As one possibility to improve decision-making during the early stages of development, adaptive and Bayesian methods have been recommended.
To investigate the current practice in designing early-phase studies in UK public sector research institutions and the use of adaptive and Bayesian methods in particular and to determine factors that hinder the penetration of methodological advances into practice.
A questionnaire was sent to all UK clinical trials units (CTUs) to gauge their involvement in early-phase studies and to learn about the designs used in these studies. Follow-up visits to units conducting early-phase studies with round-table discussions around the methods used and the obstacles faced when using adaptive methods were undertaken.
More than half of the CTUs are involved in early-phase studies, but conservatism in the methods used in these studies is present. Reasons for novel methodology not being used include a lack of expertise, incompatible funding and unit structure, and a lack of software.
Information is collected from UK CTUs, which undertake a large portion (but not all) publicly funded trials.
The use of adaptive and Bayesian methods for early-phase clinical studies in the UK public sector is at present limited. Various different initiatives aim to support and facilitate the use of these methods, however, so that an increased use of these methods can be anticipated in the future.
近年来，验证性研究的成功率一直很低，因此更加重视探索性研究的开展。作为一种改进开发早期决策的可能性，适应性和贝叶斯方法已被推荐使用。
调查英国公共部门研究机构在设计早期阶段研究中的当前实践，特别是使用适应性和贝叶斯方法的情况，并确定阻碍方法学进展渗透到实践中的因素。
向所有英国临床试验单位（CTU）发送了一份问卷，以评估他们在早期阶段研究中的参与情况，并了解这些研究中使用的设计。对正在进行早期阶段研究的单位进行了后续访问，围绕使用的方法进行了圆桌讨论，并探讨了在使用适应性方法时面临的障碍。
超过一半的 CTU 参与了早期阶段的研究，但这些研究中使用的方法仍然比较保守。没有采用新方法的原因包括缺乏专业知识、资金和单位结构不兼容，以及缺乏软件。
信息是从英国 CTU 收集的，这些单位承担了大部分（但不是全部）公共资助的试验。
目前，英国公共部门早期临床研究中使用适应性和贝叶斯方法的情况有限。各种不同的举措旨在支持和促进这些方法的使用，因此可以预期未来会更多地使用这些方法。
