A New Toolkit for Conducting Clinical Trials in Rare Disorders: Will This Dog Hunt?

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Robert G Peterson
Stuart M MacLeod
Agnes V Klein


Personalized medicine, clinical trials, NNT benefit/harm, drug regulation, cost - effectiveness


Abrahamyan et al 1 have provided a valuable summary of innovative methods that may be applied in the design of clinical trials for small populations such as those suffering from rare disorders. In that context they point out the many limitations encountered in relying upon traditional randomized trial designs, both with respect to the challenges in providing power to assess small treatment effects, and the consequent dilemma for payers in determining economic and/or social value from outcomes measure d in such studies.

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