July 25, 2017: Engaging Patient Communities to Advance Translational Science

NCATS’ scientific goals include discovering new insights and approaches that will make the translational process more effective and efficient. One such potential breakthrough approach — involvement of patient communities in the translational science process from the outset — has shown striking success in individual cases, such as in the development of interventions for the rare disease cystic fibrosis. But as in so many areas of translation, there is no general understanding yet of what practices in patient community engagement lead to improved translational success. So, true to our mission, NCATS is taking a systematic approach to this question, aiming to develop a translational science of patient community engagement, which can be applied to any translational research project to make it more effective.

Our Center took a big step forward in this effort on June 30, 2017, at NCATS Day: Partnering with Patients for Smarter Science. Attended by dozens of patients, caregivers, support organization representatives and social scientists — and nearly as many NCATS scientists and staff — the day was structured as a two-way dialogue. Attendees engaged directly with NCATS staff to learn from each other: attendees about Center programs and initiatives with which they might engage, and NCATS staff about how attendees thought NCATS could most effectively involve patients at every stage of the translational science spectrum.

The day began with a striking case study: the Center’s collaboration with the Alpha-1 Foundation to find treatments for alpha-1 antitrypsin deficiency, a rare disease characterized by liver and lung damage. Though the purpose and progress of the project were presented, more important to the day was the joint discussion of success factors — that is, what explained the remarkably successful pace and productivity of the collaboration — and how they could be applied to other projects. Insights included details on team composition, project plans, milestones, focus and continuous communication. 

Next, NCATS scientists from each of the Center’s programs presented a brief description of how their program currently works to incorporate patient engagement. In the breakout groups that followed, attendees provided feedback on current efforts, suggestions for improvements and entirely new areas of opportunity and need. In a rich final session, conclusions were summarized and an action plan for next steps created, which NCATS is now using to improve its programs and to plan for continuation of this enormously valuable and important interchange.

The next big event in our patient community engagement efforts will be on Sept. 8, 2017, when we will host the patient community for the NCATS Toolkit for Patient-Focused Therapy Development: Demonstration and Dissemination Meeting. This event will unveil, explain and plan future development of the Toolkit, an online portal developed in collaboration with patients and rare disease advocates as a gateway to “how-to” resources and tools that span the therapy development landscape, including how to:

  • Establish a patient registry;
  • Drive patient-focused discovery and pre-clinical research and development;
  • Work with NIH and the Food and Drug Administration; and
  • Conduct post-market surveillance.

Register now to attend!

Through these efforts, NCATS is developing the translational science of patient community engagement — a key part of our mission to improve health through smarter science.

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