NINDS recently hosted its fifth nonprofit forum: “Partnering to Advance Therapeutics for Neurological Disorders.” The meeting—attended by representatives from 60 nonprofit organizations across the country—gave patient advocacy groups an opportunity to learn from each other and about NIH and NINDS, provided them with an environment to share their interests, and allowed them to interact directly with program staff. The forum was held on June 1 at the Neuroscience Center Building in Bethesda, MD.
NINDS Director Dr. Story Landis opened with welcome remarks and an overview, “NIH 101: What It Is and Isn’t and How Council Works,” which covered NIH’s mission and makeup, and NINDS funding basics.
“Basic scientists think NIH is about enabling them to discover exciting new facts about how the brain works and how the heart works, and people interested in diseases think NIH is about learning about how to treat diseases,” said Landis. “But it’s the marriage of that basic science and the application of that basic science that makes NIH unique.”
Participants discussed the National Center for Advancing Translational Sciences (NCATS)—the proposed new NIH institute scheduled to open later this year—and then split into parallel breakout sessions, led by panels of NINDS program staff as well as nonprofit representatives. Topics dealt with peer review and priority setting, translational and preclinical science, resources for patient registries and recruitment, and clinical research.
The interactive breakout sessions were new to the forum this year, as was the new off-campus location. These changes were suggestions by a planning group of advisors invited to help NINDS maximize the benefits of the meeting for the groups. Members of the planning group were NINDS Director Dr. Story Landis; NINDS Deputy Director Dr. Walter Koroshetz; Ronald Bartek, president of the Friedreich’s Ataxia Research Alliance (FARA); Mary Cobb, senior vice president of Membership and Organizational Strategy of the National Organization for Rare Disorders (NORD); Marian Emr, director of the NINDS Office of Communications and Public Liaison; Petra Kaufmann, associate director for Clinical Research, NINDS; Natacha Pires, director of medical and public affairs for the Neuropathy Association; Amy Rick, chief executive officer of the Parkinson’s Action Network; NINDS Public Liaison Officer Gregory Roa; Dr. Cynthia Rothblum-Oviatt, science coordinator of the Ataxia Telangiectasia (A-T) Children’s Project; and Dr. Vicky Whittemore, former vice president and chief scientific officer of the Tuberous Sclerosis Alliance.
During lunch, smaller networking groups were divided among six broad topics: Rare Disease Network, Multisystem Diseases, the Benefits of Building Nonprofit Alliances, Nuts and Bolts of Establishing a Nonprofit Agency/Selecting a Scientific Advisory Board, Public-Private Partnerships, and Working with FDA. Representatives could talk directly with the NINDS program staff who oversee the institute’s research portfolios as well as with their fellow patient advocacy colleagues and NIH and FDA staff.
“We all came together to learn and discuss how we could work collaboratively to create a better future for the patients we represent,” said Natacha Pires, director of Medical and Public Affairs of the Neuropathy Association. “For some, this was an opportunity to touch base and communicate updates; for others it was the exciting first of many more conversations to come,” she said.
The afternoon was filled with case histories, highlighting the accomplishments and experiences of three groups, and showing how collaboration and coordination with NIH, other HHS agencies, industry, and Congress could lead to successful outcomes.
At the end of the day the nonprofit representatives gathered independent of NINDS staff to talk with each other and brainstorm about working together. This session was also a first this year.
“The NINDS Nonprofit Forum is a recognition and acceptance of the evolving role of patient advocacy groups as research partners in the quest for better diagnostics and interventions for rare diseases,” said Dr. Stephen Groft, director of the NIH Office of Rare Disease Research. “It was an outstanding conference. The interchange that occurs between staff and patient group leaders really is remarkable.”