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...

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Detalles Bibliográficos
Autores principales: Hee, Siew Wan, Willis, Adrian, Tudur Smith, Catrin, Day, Simon, Miller, Frank, Madan, Jason, Posch, Martin, Zohar, Sarah, Stallard, Nigel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
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

Internet

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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