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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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Detalles Bibliográficos
Autores principales: McMenamin, Martina, Berglind, Anna, Wason, James M. S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5964664/
https://www.ncbi.nlm.nih.gov/pubmed/29788976
http://dx.doi.org/10.1186/s13023-018-0819-1