Recommendations for the design of small population clinical trials

Recommendations for the design of small population clinical trials

Abstract 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...

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Bibliographic Details
Main Authors: Simon Day, Anneliene Hechtelt Jonker, Lilian Pek Lian Lau, Ralf-Dieter Hilgers, Ilan Irony, Kristina Larsson, Kit CB Roes, Nigel Stallard
Format: Article
Language:English
Published: BMC 2018-11-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Small populations
Small population studies
Clinical trials
Statistical methods
Rare diseases
Recommendations
Online Access:http://link.springer.com/article/10.1186/s13023-018-0931-2

Internet

http://link.springer.com/article/10.1186/s13023-018-0931-2

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