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Director’s Corner

July 15, 2016: Engaging Patients Is Crucial to Improving the Translational Process

Christopher Austin

Engaging patients at all stages of translation is crucial: Their input and perspectives as members of the research team help provide insights, focus, urgency and connectivity that can be instrumental in making the development, testing and deployment of new interventions more efficient and effective.

NCATS views the science of patient engagement as a key area for exploration and innovation. As a related communications endeavor to help make researchers and the public more aware of the challenges that rare disease patients and their families face, NCATS has produced 10 video stories that I hope you will take the time to view and share.

In the pre-clinical arena, James Inglese, Ph.D., director of NCATS’ Assay Development and Screening Technology Laboratory, forms collaborative relationships with numerous rare disease organizations to launch early-stage drug discovery efforts. These agreements involve foundation support for postdoctoral researchers with expertise in a particular disease to work with Inglese, a world expert in developing drug testing systems for novel therapeutic targets. Most recently, Inglese announced such an agreement with the Global Foundation for Peroxisomal Disorders and the Wynne Mateffy Research Foundation, which fund research on a group of rare genetic conditions that appear at birth or in early life, causing multi-organ dysfunction and life-threatening complications. The aim is to identify a promising potential treatment.

On the clinical side, NCATS requires all Rare Diseases Clinical Research Network (RDCRN) consortia to include patient groups as full partners on their research teams, an innovative approach that helps achieve greater success, including in design and enrollment of clinical studies. The RDCRN Coalition of Patient Advocacy Groups develops and shares best practices, and the RDCRN website includes a web-based contact registry for patients who may be interested in participating in RDCRN clinical studies. NCATS also engages patients through its Genetic and Rare Diseases Information Center (GARD), which provides comprehensive information about rare and genetic diseases to patients, their families, health care providers, researchers and the public. GARD provides accurate, up-to-date information about ongoing research, symptoms, treatment options and other details.

In addition, NCATS continues to collaborate with organizations that share our commitment to understanding and implementing the science of patient engagement. For example, NCATS Health Scientist Shelley Brown, Ph.D., recently spoke at a FasterCures workshop focused on better understanding the influences that are helping or hindering patient-centered activities, identifying and prioritizing tools and templates to reduce resistance and remove practical barriers to patient engagement, and shaping the future agenda of collaborative activities to enable greater patient centricity.

These are just some of the ways in which NCATS is committed to engaging patients, their families and the advocacy organizations that serve them, to form the teams that will get more treatments to more patients more quickly.

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