A New Toolkit for Conducting Clinical Trials in Rare Disorders

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Lusine Abrahamyan
Ivan R Diamond
Sindhu R Johnson
Brian M Feldman


Evidence-based medicine, observational studies, rare disorders, NNT, adaptive trial design, Bayesian analysis


Evidence based medicine requires strong scientific evidence upon which to base treatment. Because the available study populations for rare diseases are small, this evidence is difficult to accrue. Investigators need to consider a flexible toolkit of methods to deal with the problems inherent in the study of rare disease. We present some potential solutions in this paper.

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