MRC North West Hub for Trials Methodology Research, Department of Biostatistics, Institute of Translational Medicine, University of Liverpool, Liverpool, UK.
Department of Women's and Children's Health, Institute of Translational Medicine, University of Liverpool, Liverpool, UK; Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust, Liverpool, UK.
Best Pract Res Clin Rheumatol. 2014 Apr;28(2):247-62. doi: 10.1016/j.berh.2014.03.004.
Evidence from clinical trials, ideally using randomisation and allocation concealment, is essential for informing clinical decisions regarding the benefits and harms of treatments for patients. Where diseases are rare, such as in paediatric rheumatic diseases, patient recruitment into clinical trials can be a major obstacle, leading to an absence of evidence and patients receiving treatments based on anecdotal evidence. There are numerous trial designs and modifications that can be made to improve efficiency and maximise what little data may be available in a rare disease clinical trial. These are discussed and illustrated with examples from paediatric rheumatology. Regulatory incentives and support from research networks have helped to deliver these trials, but more can be done to continue this important research.
来自临床试验的证据，最好采用随机化和分配隐藏，对于告知临床医生有关治疗患者的益处和危害的决策至关重要。在儿科风湿病等罕见疾病中，患者招募到临床试验可能是一个主要障碍，导致缺乏证据，患者接受基于传闻证据的治疗。有许多试验设计和修改可以提高效率，并最大限度地利用罕见病临床试验中可能获得的有限数据。本文讨论了这些设计和修改，并结合儿科风湿病的实例进行了说明。监管激励和研究网络的支持有助于开展这些试验，但还可以做更多工作来继续这一重要研究。
