Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:dateCreated
2010-9-8
pubmed:abstractText
Whenever possible, standard methodological approaches should be applied in the design and analysis of a clinical trial that warrant adequate informative value. However, there are circumstances when the number of experimental subjects is unavoidably small. In such circumstances it is justified to consider abandoning standard statistical methodology in place of alternative approaches. Performing a small clinical trial however it should be pointed out, that a such trial can never be as meaningful and provide as much evidence as a larger trial. In the present text, basic concepts are presented, that apply to small clinical trials in general. Moreover, several specific methodological approaches are presented, that either enhance the efficiency of standard statistical procedures or evolve from the idea of abandoning classical paradigms in the design and analysis of clinical trials. Within the scope of the former approach, (Bayesian) adaptive randomisation, group sequential (adaptive) designs, repeated measurement designs for longitudinal data, and meta-analyses are illustrated and discussed. The latter approach comprises alternative strategies such as (non-randomised) risk-based allocation designs, statistical prediction designs, ranking and selection designs, as well as the application of Bayesian statistics.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:status
MEDLINE
pubmed:issn
0065-2598
pubmed:author
pubmed:issnType
Print
pubmed:volume
686
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
173-90
pubmed:meshHeading
pubmed:year
2010
pubmed:articleTitle
Clinical trials and rare diseases.
pubmed:affiliation
Department of Medical Informatics and Biomathematics, Westfälische Wilhelms-Universität Münster, Münster, Germany. joachim.gerss@ukmuenster.de
pubmed:publicationType
Journal Article, Review