Centre for Tropical Medicine and Global Health, Nuffield Department of Medicine, University of Oxford, Oxford, United Kingdom.
Department of Epidemiology, Center for Public Health, Medical University of Vienna, Vienna, Austria.
PLoS Negl Trop Dis. 2021 Jul 28;15(7):e0009582. doi: 10.1371/journal.pntd.0009582. eCollection 2021 Jul.
Clinical trials are often perceived as being expensive, difficult and beyond the capacity of healthcare workers in low-resource settings. However, in order to improve healthcare coverage, the World Health Organization (WHO) World Health Report 2013 stated that all countries need to become generators as well as recipients of data. This study is a methodological examination of the steps and processes involved in setting up the Gojjam Lymphoedema Best Practice Trial (GoLBeT; ISRCTN67805210), a highly pragmatic clinical trial conducted in northern Ethiopia. Challenges to the trial and strategies used to deal with them were explored, together with the reasons for delays.
Qualitative research methods were used to analyse emails and reports from the period between trial inception and recruitment. This analysis was complemented by interviews with key informants from the trial operational team. The Global Health Research Process Map was used as a framework against which to compare the steps involved in setting up the trial. A mini-group discussion was conducted with the trial operational team after study completion for reflection and further recommendations. This study showed that the key areas of difficulty in setting up and planning this trial were: the study design, that is, deciding on the study endpoint, where and how best to measure it, and assuring statistical power; recruitment and appropriate training of staff; planning for data quality; and gaining regulatory approvals. Collaboration, for example with statisticians, the trial steering committee, the study monitors, and members of the local community was essential to successfully setting up the trial.
Lessons learnt from this trial might guide others planning pragmatic trials in settings where research is not common, allowing them to anticipate possible challenges and address them through trial design, planning and operational delivery. We also hope that this example might encourage similar pragmatic studies to be undertaken. Such studies are rarely undertaken or locally led, but are an accessible and efficient way to drive improved outcomes in public health.
临床试验通常被认为是昂贵、困难的，并且超出了资源匮乏环境中医疗保健工作者的能力范围。然而，为了改善医疗保健覆盖范围，世界卫生组织（WHO）2013 年世界卫生报告指出，所有国家都需要成为数据的生成者和接收者。本研究是对在埃塞俄比亚北部进行的戈贾姆淋巴水肿最佳实践试验（GoLBeT；ISRCTN67805210）的步骤和流程进行的方法学检查。该试验面临挑战，并探讨了应对这些挑战的策略，以及延迟的原因。
使用定性研究方法分析了试验启动和招募期间的电子邮件和报告。对试验运营团队的主要知情者进行访谈，对这一分析进行了补充。使用全球卫生研究过程图作为框架，对试验设置所涉及的步骤进行了比较。在研究完成后，与试验运营团队进行了小型小组讨论，进行反思并提出进一步建议。本研究表明，在试验的设立和规划中，困难的主要领域是：研究设计，即决定研究终点、在哪里以及如何最好地测量终点，以及确保统计学功效；人员招聘和适当培训；数据质量规划；以及获得监管批准。合作，例如与统计学家、试验指导委员会、研究监测员和当地社区成员合作，对于成功设立试验至关重要。
从这项试验中吸取的经验教训可能会为计划在研究不常见的环境中进行务实试验的其他人提供指导，使他们能够通过试验设计、规划和运营交付来预测可能出现的挑战并解决这些挑战。我们还希望这个例子能够鼓励进行类似的务实研究。这类研究很少在当地进行或由当地人领导，但却是提高公共卫生成果的一种可行且高效的方式。
