Clinical trials in rare diseases: a review of practice
نویسندگان
چکیده
Background The evaluation of treatments for rare diseases presents a number of challenges for trial practitioners, regulators and policy makers. Small sample sizes mean that ‘standard’ approaches to trial design and analysis may not be appropriate and alternatives such as Bayesian trial designs have been recommended [Lilford et al 1995]. However, little is known about the design and analysis approaches that have been implemented in practice.
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