FDA Training Session Set to Focus on Clinical Trials for Rare Disease Populations

Posted 10 October 2012 | By Alexander Gaffney, RAC 

The US Food and Drug Administration (FDA) and the National Institutes of health (NIH) are launching a new training course to instruct sponsors of rare disease therapies on how to best conduct small clinical trials for drugs, biologics and medical devices.

The course, "The Science of Small Clinical Trials," is being coordinated by FDA and NIH's Office of Rare Diseases Research and National Center for Advancing Translational Sciences (NCATS).

Because many diseases, and in particular those classified as "rare" or "orphan" diseases-those affecting fewer than 200,000 patients in the US-have exceptionally small pools of patients, enrolling a sufficient number of patients into clinical trials can be difficult and lead to problems with the statistical significance of the results.

"The course is intended to present an overall framework and provide training in the scientific aspects of designing and analyzing clinical trials based on small study populations," explained FDA in its Federal Register posting. "The course will bring together subject experts and stakeholders to identify when such trials should be conducted, along with strategies and trial designs that are conducive to overcoming the challenges they present."

The course will reportedly involve FDA reviewers, industry, NIH scientists and clinicians in an attempt to, "Build upon their existing knowledge and to obtain a broader contact of what is known about small clinical trials across medical products."

Presentations will include regulatory case studies and panel discussion, FDA said.

The course will be held at FDA's White Oak campus in Silver Spring, Maryland on 27-28 November 2012.

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