June 21, 2017: NCATS-FDA Community Engagement Helping Advance Translational Success

“Begin with the end in mind.” This business leadership mantra also is critically important in translation, but defining “the end” correctly can be difficult due to the complexity and diversity of the translational process, in which success is viewed differently from one stage to another. Well-meaning scientists often produce quality work that is highly regarded at their respective translational stage but is not acceptable for or even relevant to the next. This leads to wasted effort, mutual exasperation and — most importantly — failure to reach patients in need. 

This core characteristic of translation — which literally means “to carry across” — explains why NCATS is so focused on what we broadly call “community engagement,” with “community” defined differently depending on the stage of the translational process. Translational scientists must assume that what constitutes success to them is not what represents success to those to whom they are “carrying across.” Once this critical assumption is made, it is obvious that the only way to find out what will be successful across all stages is through community engagement. 

NCATS’ engagement with the regulatory community, specifically the Food and Drug Administration (FDA), is particularly important. Not every intervention that can improve human health is regulated by the FDA, but many — including drugs, devices and diagnostics — are. Traditionally, academic scientists have not been taught about the FDA or regulatory requirements, since publishing papers on scientific advances has been necessary and sufficient for their career success. But as more and more academic scientists become interested in translating discoveries to human health improvements, they often assume that the information needed for a publication is the same as that needed by the FDA to approve a new drug or device. That incorrect assumption is a very common cause of good science failing to progress in translation. 

To address this common translational roadblock, NCATS engages with the FDA on programs encompassing training, tool development and joint research initiatives. For example, NCATS and the FDA work together in our Tissue Chip for Drug ScreeningToxicology in the 21st Century, and Collaborative Use Repurposing Engine (CURE) programs. FDA input has been crucial to these programs’ success and impact. 

NCATS also partnered with the FDA as well as other international regulators and research organizations to create the Global Ingredient Archival System (ginas), which enables substances to be defined by a standardized, common identifier. The ginas software makes it more efficient for the FDA to review new drugs and identify safety concerns. The FDA, the European Medicines Agency and other agencies have adopted the system, which will facilitate communication with the worldwide health community. 

In addition, NCATS played a lead role in the NIH-FDA Joint Leadership Council’s development of the recently launched NIH-FDA clinical trial protocol template tool. This tool’s standardized format will make the writing and review of protocols easier and faster and will ensure that clinical protocols have all the information the FDA needs to evaluate studies of investigational new drugs and devices. NCATS also helped lead efforts for the Council’s Biomarkers, EndpointS, and other Tools (BEST) Resource, which provides a common language for NIH, the FDA and other researchers to use when communicating about biomarkers. 

Beginning with the FDA end in mind, NCATS is improving health through smarter science.

Christopher P. Austin, M.D.
National Center for Advancing Translational Sciences