For Industry

The Science of Small Clinical Trials Course

FDA White Oak Campus, Silver Spring, Maryland, November 27–28, 2012

On Wednesday and Thursday, November 27 and 28, 2012, the Food and Drug Administration (FDA) Office of Orphan Products Development along with the National Institutes of Health (NIH), Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), held the Science of Small Clinical Trials Course at the FDA White Oak Campus in Silver Spring, MD. The course covered techniques for clinical studies with limited sample sizes including a focus on the study of rare diseases, and provided examples of how such methods have been implemented. There were over 80 attendees from government, academia, industry, and patient advocacy groups and more than 200 additional participants who joined the event via live webcast.

The first day focused on general principles and included six case studies of small drug, biologic and device trials that supported marketing approvals. Various techniques including Enrichment, Adaptive design and Bayesian statistical methods were highlighted. The second day provided an opportunity to focus on special considerations in the rare disease space, as well as topics including historical controls, the role of patient organizations, and the industry perspective. All sessions were enhanced by active learning methods and panel discussions with audience participation.

The course was well received and attendees noted that it provided a valuable training opportunity and included a breadth of expert knowledge and interesting dialogues.

Speaker presentations and webcast recordings are now available and can be accessed on the FDA_NIH Science of Small Clinical Trials Course website.

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