Ouma Luke O, Al-Ashmori Sarah, Sarkodie Samuel, Whitehead Lou, Konkoth Ann Breeze, Hiu Shaun, Bigirumurame Theophile, Kareithi Dorcas Njeri, Lozano-Kuehne Jingky, Shahmandi Marzieh, Wason James M S
Biostatistics research group, Newcastle University Faculty of Medical Sciences, Newcastle upon Tyne, UK
Biostatistics research group, Newcastle University Faculty of Medical Sciences, Newcastle upon Tyne, UK.
BMJ Glob Health. 2025 Jul 25;10(7):e018561. doi: 10.1136/bmjgh-2024-018561.
BACKGROUND: Master protocols-umbrella, basket and platform trials, study multiple therapies, multiple diseases or both, offering many advantages, most profoundly that they answer multiple treatment-related questions, which would otherwise take multiple trials. We conducted a review of clinical trial registries to characterise their use in advancing precision medicine in low and middle-income countries (LMICs). METHODS: We searched trial records available in 20 trial registries globally, including ClinicalTrials.gov and WHO ICTRP, to identify umbrella, basket and platform trials launched until 30 September 2023. RESULTS: We identified 102 master protocols-29 umbrella trials, 31 basket trials, 36 platform trials, as well as six other designs that partially aligned with the working definition of master protocols, run in 54 different LMICs. Most trials were pharmaceutical industry-sponsored studies (60/102, 58.8%), conducted in oncology settings (56/102, 54.9%), currently ongoing (69/102, 67.6%) in early phase (phase I and II) settings (70/102, 68.6%) and have been planned or launched in the last 5 years (93/102, 91.2%), mainly with international collaborations in high-income countries. China was a site to more than half of all master protocols (53/102, 52%), and only a small proportion of trials (5/102, 4.9%) launched exclusively in LMICs excluding China and European middle-income countries. For most studies, aspects of trial design and trial documentation (including study protocols and analysis plans) were not publicly accessible. CONCLUSION: Unlike high-income countries, where several hundreds of master protocols are ongoing or completed, there is limited use of master protocols in LMICs, partly owing to low penetration of precision medicine research and limited clinical trial infrastructure in most LMICs. The evidence presented here creates a case for supporting precision medicine initiatives in LMICs (especially Africa) and training and capacity building initiatives focused on innovative clinical trial designs like master protocols, especially in therapeutic areas outside oncology.
背景：主方案——伞形试验、篮子试验和平台试验，研究多种疗法、多种疾病或两者兼而有之，具有诸多优势，其中最显著的是它们能回答多个与治疗相关的问题，否则需要进行多项试验。我们对临床试验注册库进行了回顾，以描述它们在中低收入国家（LMICs）推进精准医学方面的应用情况。 方法：我们检索了全球20个试验注册库（包括ClinicalTrials.gov和WHO ICTRP）中可获取的试验记录，以识别截至2023年9月30日启动的伞形试验、篮子试验和平台试验。 结果：我们识别出102个主方案——29个伞形试验、31个篮子试验、36个平台试验，以及6个其他部分符合主方案工作定义的设计，这些试验在54个不同的中低收入国家开展。大多数试验是由制药行业赞助的研究（60/102，58.8%），在肿瘤学环境中进行（56/102，54.9%），目前处于早期阶段（I期和II期）的试验正在进行中（69/102，67.6%），且是在过去5年中计划或启动的（93/102，91.2%），主要是与高收入国家进行国际合作。中国是所有主方案一半以上试验的开展地点（53/102，52%）, 并且仅有一小部分试验（5/102，4.9%）专门在不包括中国和欧洲中等收入国家的中低收入国家开展。对于大多数研究，试验设计和试验文档（包括研究方案和分析计划）的相关内容无法公开获取。 结论：与有数百个主方案正在进行或已完成的高收入国家不同，中低收入国家对主方案的使用有限，部分原因是精准医学研究的渗透率较低，且大多数中低收入国家的临床试验基础设施有限。此处提供的证据表明有必要支持中低收入国家（特别是非洲）的精准医学倡议，以及针对创新临床试验设计（如主方案）的培训和能力建设倡议，尤其是在肿瘤学以外的治疗领域。
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