Research, industry and advocacy join on NIH Rare Disease Day
Event returns to NIH Visitors Center
After two years of online-only participation, NIH Rare Disease Day 2023 (RDD) returned on Feb. 28 to its previous hybrid format for a vigorous day of renewed, in-person communication and networking in the rare disease community.
Organized by National Center for Advancing Translational Sciences (NCATS) and the Clinical Center, RDD brings together diverse research interests in government and academic settings. It also brings together patient advocacy organizations, including representative patients and families affected by rare diseases. Sharing highlights in rare disease research, recognizing those who are making exceptional contributions and proposing an agenda to keep research moving forward, Rare Disease Day is established as a cornerstone event in the field.
Several presentations discussed relevant activity at NCATS, including the Division of Rare Disease Research and Innovation, the Genetic and Rare Diseases Information Center resource, the Rare Disease Alert System project and the newer RARe-source program.
The Clinical Center's own involvement with clinical trials was addressed by its Office of Patient Recruitment. They focused on resources to help volunteers with a rare disease find useful clinical research studies at the NIH.
"Our mission is to support the research performed at the Clinical Center by coordinating the referral process as the primary point of contact between those seeking to volunteer in clinical studies and the researchers conducting them," stated Nikita Curry, supervisor of the Office of Patient Recruitment.
Presenters addressed the special attention needed for the AYA (adolescent and young adult rare disease patients) population as they move between pediatric and adult care settings. Speakers also highlighted the essential role of The Children's Inn at NIH in supporting pediatric and young adult patients and their families.
Other sessions included "Rare Stories," where patients, research, treatment professionals and other roles aligned with rare diseases spoke about their specific interests and activities. Participants also spoke about the complementary roles and collaborative efforts that bring industry and advocacy groups together.
Both ground and lower levels of the NIH Natcher Visitors Center, the host site for RDD '23, were lined with exhibitors – patient advocacy nonprofits and NIH programs alike. All expressed enthusiasm for the return of face-to-face interaction with attendees.
Dynamic interaction between all constituents is what makes each RDD a time of hope, promise and renewed commitment from all segments of the rare disease community.
Two years of virtual RDD didn't diminish the energy and level of interest in making history in rare disease by making rare diseases history.
This year’s RDD was attended in person by more than 570 attendees and more than 1,700 attendees viewed the presentations online. If you would like to watch the RDD presentations online, please visit NIH Videocast.
- Robert Burleson