Please use this identifier to cite or link to this item:
http://hdl.handle.net/1942/25210
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DC Field | Value | Language |
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dc.contributor.author | Hilgers, Ralf-Dieter | - |
dc.contributor.author | König, Franz | - |
dc.contributor.author | MOLENBERGHS, Geert | - |
dc.contributor.author | Senn, Stephen | - |
dc.date.accessioned | 2017-11-21T14:46:47Z | - |
dc.date.available | 2017-11-21T14:46:47Z | - |
dc.date.issued | 2016 | - |
dc.identifier.citation | Journal of Rare Diseases Research & Treatment, 1(3), p. 53-60 | - |
dc.identifier.issn | 2572-9411 | - |
dc.identifier.uri | http://hdl.handle.net/1942/25210 | - |
dc.description.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. | - |
dc.language.iso | en | - |
dc.rights | © 2016 Ralf‐Dieter Hilgers. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License. | - |
dc.subject.other | clinical trials; rare disease populations; IDeAl consortium | - |
dc.title | Design and analysis of clinical trials for small rare disease populations | - |
dc.type | Journal Contribution | - |
dc.identifier.epage | 60 | - |
dc.identifier.issue | 3 | - |
dc.identifier.spage | 53 | - |
dc.identifier.volume | 1 | - |
local.bibliographicCitation.jcat | A1 | - |
local.type.refereed | Refereed | - |
local.type.specified | Review | - |
local.identifier.vabb | c:vabb:437909 | - |
item.fullcitation | Hilgers, Ralf-Dieter; König, Franz; MOLENBERGHS, Geert & Senn, Stephen (2016) Design and analysis of clinical trials for small rare disease populations. In: Journal of Rare Diseases Research & Treatment, 1(3), p. 53-60. | - |
item.fulltext | With Fulltext | - |
item.validation | vabb 2019 | - |
item.contributor | Hilgers, Ralf-Dieter | - |
item.contributor | König, Franz | - |
item.contributor | MOLENBERGHS, Geert | - |
item.contributor | Senn, Stephen | - |
item.accessRights | Open Access | - |
crisitem.journal.issn | 2572-9411 | - |
Appears in Collections: | Research publications |
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