Systematic reviews in paediatric multiple sclerosis and Creutzfeldt-Jakob disease exemplify shortcomings in methods used to evaluate therapies in rare conditions

BACKGROUND: Randomized controlled trials (RCTs) are the gold standard design of clinical research to assess interventions. However, RCTs cannot always be applied for practical or ethical reasons. To investigate the current practices in rare diseases, we review evaluations of therapeutic intervention...

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Bibliografiske detaljer
Udgivet i:Orphanet J Rare Dis
Main Authors: Unkel, Steffen, Röver, Christian, Stallard, Nigel, Benda, Norbert, Posch, Martin, Zohar, Sarah, Friede, Tim
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2016
Fag:
Research
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4761188/
https://ncbi.nlm.nih.gov/pubmed/26897367
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-016-0402-6
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