Abstract:
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The 21st Century Cures Act recognizes that an overemphasis on product development can sometimes cause a neglect of individual patients and calls for patient-focused drug development. N-of-1 trials, where a single patient represents the entire trial, solve this problem by giving drug development organizations permission to focus on individual patients, which is especially relevant for government organizations like the US Army where the mission is to support the warfighter rather than specific product development. We discuss how n-of-1 trials can be effectively used in heterogeneous diseases like bacterial infection, dengue, and malaria, in early phase trials, and show how their use may even be possible in outbreak events like Ebola and Zika. We demonstrate how a properly established framework for n-of-1 trials can make it possible to leverage Bayesian methods to combine data across trials to accomplish drug development objectives more efficiently and at lower cost to assist in winning regulatory approval.
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