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Please use this identifier to cite or link to this item: http://hdl.handle.net/1942/25210

Title: Design and analysis of clinical trials for small rare disease populations
Authors: Hilgers, Ralf-Dieter
König, Franz
Molenberghs, Geert
Senn, Stephen
Issue Date: 2016
Citation: Journal of Rare Diseases Research & Treatment, 1(3), p. 53-60
Abstract: The mostly unmet need as well as the pressure to show efficacy of new therapies to treat rare diseases contrasts with the limited possibility to use traditional statistical methods to design and analyse clinical trials in this setting. Within this paper, we will refer to the current state of design and analysis methods, as well as practical conditions to be considered when conducting a clinical trial for rare diseases. We will embed the research of the IDeAl project within this setting and give some first recommendations to improve the methodology for clinical trials in rare diseases.
URI: http://hdl.handle.net/1942/25210
ISSN: 2572-9411
Category: A2
Type: Journal Contribution
Appears in Collections: Research publications

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