Bayesian methods for the design and interpretation of clinical trials in very rare diseases.

ABSTRACT: This paper considers the design and interpretation of clinical trials comparing treatments for conditions so rare that worldwide recruitment efforts are likely to yield total sample sizes of 50 or fewer, even when patients are recruited over several years. For such studies, the sample size needed to meet a conventional frequentist power requirement is clearly infeasible. Rather, the expectation of any such trial has to be limited to the generation of an improved understanding of treatment options. We propose a Bayesian approach for the conduct of rare-disease trials comparing an experimental treatment with a control where patient responses are classified as a success or failure. A systematic elicitation from clinicians of their beliefs concerning treatment efficacy is used to establish Bay

SUBMITTER: Hampson LV 

PROVIDER: S-EPMC4260127 | biostudies-literature | 2014 Oct

REPOSITORIES: biostudies-literature