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Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials.gov
BACKGROUND: Clinical trials are typically designed using the classical frequentist framework to constrain type I and II error rates. Sample sizes required in such designs typically range from hundreds to thousands of patients which can be challenging for rare diseases. It has been shown that rare di...
Autores principales: | Hee, Siew Wan, Willis, Adrian, Tudur Smith, Catrin, Day, Simon, Miller, Frank, Madan, Jason, Posch, Martin, Zohar, Sarah, Stallard, Nigel |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5335492/ https://www.ncbi.nlm.nih.gov/pubmed/28253932 http://dx.doi.org/10.1186/s13023-017-0597-1 |
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