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Design and analysis features used in small population and rare disease trials: A targeted review

Giles Partington,Suzie Cro,Alexina Mason,Rachel Phillips,Victoria Cornelius

Journal of Clinical Epidemiology（2022）

Cited 4|Views8
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Abstract
Bayesian trials required lower sample size through use of informative priors. Most frequentists didn't achieve their target sample size. Bayesian methods offer promising solutions for such trials but are underutilised.
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Key words
Rare diseases,Small populations,Bayesian methods,Randomized trials,Trial design
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