Small clinical trials may be appropriate and even a necessity in various contexts that include, but are not limited, to rare diseases. Being able to conduct them with scientific rigor is of increasing importance in the current regulatory and scientific environment. The Food and Drug Administration (FDA), in collaboration with the National Institutes of Health (NIH) Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), conducted a 2-day public course titled The Science of Small Clinical Trials. This course presented an overall framework and provided training in the scientific aspects of designing and analyzing clinical trials based on small study populations.

This event brought together subject experts and stakeholders to discuss examples of small clinical trials and to identify strategies and trial designs that are conducive to overcoming the problem of executing clinical trials using small study populations. The meeting was broadcast via a live interactive webcast.

The format consisted of didactic lectures provided by representatives from various FDA Centers, NIH, and non-government organizations, along with interactive panel discussion sessions for questions from on-site participants as well as FDA web participants.
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