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Recommendations for the design of small population clinical trials

BACKGROUND: Orphan drug development faces numerous challenges, including low disease prevalence, patient population heterogeneity, and strong presence of paediatric patient populations. Consequently, clinical trials for orphan drugs are often smaller than those of non-orphan drugs, and they require...

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Detalles Bibliográficos
Autores principales:	Day, Simon, Jonker, Anneliene Hechtelt, Lau, Lilian Pek Lian, Hilgers, Ralf-Dieter, Irony, Ilan, Larsson, Kristina, Roes, Kit CB, Stallard, Nigel
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2018
Materias:	Position Statement
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6219020/ https://www.ncbi.nlm.nih.gov/pubmed/30400970 http://dx.doi.org/10.1186/s13023-018-0931-2

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