Megan O’Boyle, director of the Phelan-McDermid Syndrome Foundation, participates in “How Can We Help You and How You Can Help Us?” session.
|Dr. Amelie Gubitz, program director in the neurodegeneration cluster of the Division of Extramural Research, NINDS, leads “How Do You Build Your Research Program?” session.
||Dr. Kurt Fischbeck (r), chief of the Neurogenetics Branch, chairs the clinical outcomes panel. Fellow panelists include Dr. Jane Paulsen (l), Roy J. Carver chair in neuroscience and director of the divisions of neuropsychology and psychology at the University of Iowa Carver College of Medicine; and NINDS deputy director Dr. Walter Koroshetz.
The importance of building and maintaining partnerships was the central message delivered at NINDS’s 8th nonprofit forum held recently at NIH. Representatives from more than 50 nonprofit organizations attended the 1½-day meeting, “Progress through Partnership,” which focused on multiple aspects of the NINDS research portfolio and the necessity of collaborations.
“When I first became the institute director, I realized that I did not know enough about the issues and concerns and difficulties that patients with neurological disorders have,” said NINDS director Dr. Story Landis in opening remarks. “And I knew that one of the most important things I could do was to learn more about the patient community.” The meeting served as one of Landis’ last official acts before she retired Oct. 3, after 19 years of government service. “This forum is an opportunity for people who run foundations and patient advocacy groups to learn what’s going on at NIH and NINDS, network with colleagues and engage in discussions with NINDS staff.”
Dr. Rajesh Ranganathan, director of the NINDS Office of Translational Research, led “When Is the Time for Handoff? How to Engage the Pharmaceutical Industry in Your Disease.” Panelists from the pharmaceutical industry and the nonprofit community talked about best strategies for partnering. They encouraged nonprofit groups to start the dialogue with industry early, understand the landscape well, be strategic and network effectively.
A session on nonprofit success stories highlighted experiences of four groups: the Hydrocephalus Association, Parent Project Muscular Dystrophy, Dysautonomia International and Friedreich’s Ataxia Research Alliance.
The meeting also featured panels on Clinical and Translational Science Awards (CTSAs), clinical outcomes and the Patient-Centered Outcomes Research Institute (PCORI). “How Can Patients Work with CTSAs?” explored the NCATS CTSA program with a particular interest in neuroscience.
A panel led by NINDS senior investigator Dr. Kurt Fischbeck asked “How do we know if a treatment works?” and discussed the importance of developing clinically meaningful outcome measures to assess the effectiveness of a treatment.
The “Power of Partnerships: Engaging Patients in PCORI Research” panel looked at one of the newest funders of biomedical researchers. PCORI is a nonprofit, nongovernmental organization that was created as part of the Patient Protection and Affordable Care Act of 2010 to help patients and their caregivers make better informed decisions about health.
“The pendulum is shifting,” said panelist Megan O’Boyle, director of the Phelan-McDermid Syndrome Foundation and a PCORI principal investigator. “The patient should be in the room with the FDA and with industry and with the clinicians who are designing the studies. It’s wonderful to be in the room at the table. In order for patient engagement to continue to work, thrive and really prove its point, we need to show that patients are collaborators and that we, too, can play well with others.”
During a plenary session on the NIH BRAIN Initiative, Landis shared information on the current state of the effort’s 12-year plan and first grants.
“Brain disorders are the leading source of disease burden and cost in the United States,” Landis said. “Recent breakthroughs are transforming how we study brain structure and function, but the best is yet to come. The BRAIN Initiative will build on this recent progress to create tools that will accelerate discovery and build the foundation we need to reduce the burden of brain disorders.”
The meeting then split into breakout sessions, after which nonprofit representatives met informally with NINDS program directors. The annual forum represents the institute’s continued commitment to sustain partnerships with the nonprofit community.