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Directions for new developments on statistical design and analysis of small population group trials
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Hilgers, Ralf-Dieter, Roes, Kit and Stallard, Nigel (2016) Directions for new developments on statistical design and analysis of small population group trials. Orphanet Journal of Rare Diseases, 11 (1). 78. doi:10.1186/s13023-016-0464-5
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Official URL: http://dx.doi.org/10.1186/s13023-016-0464-5
Abstract
Most statistical design and analysis methods for clinical trials have been developed and evaluated where at least several hundreds of patients could be recruited. These methods may not be suitable to evaluate therapies if the sample size is unavoidably small, which is usually termed by small populations. The specific sample size cut off, where the standard methods fail, needs to be investigated. In this paper, the authors present their view on new developments for design and analysis of clinical trials in small population groups, where conventional statistical methods may be inappropriate, e.g., because of lack of power or poor adherence to asymptotic approximations due to sample size restrictions.
| Item Type: | Journal Article | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| Subjects: | R Medicine > R Medicine (General) | ||||||||
| Divisions: | Faculty of Medicine > Warwick Medical School > Health Sciences > Statistics and Epidemiology Faculty of Medicine > Warwick Medical School |
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| Library of Congress Subject Headings (LCSH): | Clinical trials -- Statistical methods -- Research, Rare diseases | ||||||||
| Journal or Publication Title: | Orphanet Journal of Rare Diseases | ||||||||
| Publisher: | BioMed Central Ltd. | ||||||||
| ISSN: | 1750-1172 | ||||||||
| Official Date: | 14 June 2016 | ||||||||
| Dates: |
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| Volume: | 11 | ||||||||
| Number: | 1 | ||||||||
| Article Number: | 78 | ||||||||
| DOI: | 10.1186/s13023-016-0464-5 | ||||||||
| Status: | Peer Reviewed | ||||||||
| Publication Status: | Published | ||||||||
| Access rights to Published version: | Open Access | ||||||||
| Funder: | Seventh Framework Programme (European Commission) (FP7) | ||||||||
| Grant number: | Health-F5-2013-603160 Asterix project, 602552 IDEAL project, 602144 InSpiRe project | ||||||||
| Adapted As: |
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