• Best Pract Res Clin Rheumatol · Apr 2014

    Review

    Methodology of clinical trials for rare diseases.

    • Catrin Tudur Smith, Paula R Williamson, and Michael W Beresford.
    • MRC North West Hub for Trials Methodology Research, Department of Biostatistics, Institute of Translational Medicine, University of Liverpool, Liverpool, UK. Electronic address: cat1@liv.ac.uk.
    • Best Pract Res Clin Rheumatol. 2014 Apr 1;28(2):247-62.

    AbstractEvidence 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.Copyright © 2014 Elsevier Ltd. All rights reserved.

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