期刊
BEST PRACTICE & RESEARCH IN CLINICAL RHEUMATOLOGY
卷 28, 期 2, 页码 247-262出版社
ELSEVIER SCI LTD
DOI: 10.1016/j.berh.2014.03.004
关键词
Rare disease trials; Paediatric rheumatology; Trial design
类别
资金
- Medical Research Council - North West Hub for Trials Methodology Research at the University of Liverpool [G0800792]
- Medical Research Council [G0800792, MR/K025635/1] Funding Source: researchfish
- MRC [G0800792, MR/K025635/1] Funding Source: UKRI
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. (C) 2014 Elsevier Ltd. All rights reserved.
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