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Improving the analysis of composite endpoints in rare disease trials

BACKGROUND: Composite endpoints are recommended in rare diseases to increase power and/or to sufficiently capture complexity. Often, they are in the form of responder indices which contain a mixture of continuous and binary components. Analyses of these outcomes typically treat them as binary, thus...

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Bibliographische Detailangaben
Veröffentlicht in:Orphanet J Rare Dis
Hauptverfasser: McMenamin, Martina, Berglind, Anna, Wason, James M. S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2018
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5964664/
https://ncbi.nlm.nih.gov/pubmed/29788976
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0819-1
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