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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Foilsithe in:Orphanet J Rare Dis
Main Authors: Unkel, Steffen, Röver, Christian, Stallard, Nigel, Benda, Norbert, Posch, Martin, Zohar, Sarah, Friede, Tim
Formáid: Artigo
Teanga:Inglês
Foilsithe: BioMed Central 2016
Ábhair:
Research
Rochtain Ar Líne: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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