The College of Pharmacy's Center for Orphan Drug Research recently hosted its annual Rare Disease Day, on Thursday, March 6, 2025, at the McNamara Alumni Center. Themed “Navigating Rare Disorders: Insights from Lived Experience,”, this free, public and community-engaged event brought together patients, caregivers, healthcare professionals, and researchers to share perspectives on the challenges of rare disease diagnosis and management.
The 2025 Rare Disease Day event featured keynote speakers Stephanie Tomlinson, Haley Brunelle, and Dr. Kathleen Bogart. A diverse panel of speakers including a patient advocate, family caregiver, neuropsychologist, and physician provided multifaceted insights into the rare disease experience. The event, which offered both in-person and virtual attendance options, received support from several pharmaceutical companies and healthcare organizations committed to advancing rare disease research and patient care.
The Center for Orphan Drug Research has a threefold mission: research on new therapies for rare disorders, education, and public outreach/advocacy. Rare Disease Day at the College of Pharmacy began as a way to fulfill its mission of public outreach and advocacy. The first Rare Disease Day event (2013) was held in collaboration with Gillette Children's Hospital at the Mall of America, providing both an educational program and recreational opportunities for children with rare diseases and their families. Following the success of this inaugural event, the program moved to campus in 2014 and has evolved, with a different theme each year, it addresses various aspects of rare diseases.
Over time, the purpose of Rare Disease Day has expanded to serve the broader rare disease community by providing the latest information on medical advances, drug development, access challenges, and importantly, highlighting the lived experiences of patients and families. Dr. James Cloyd III, who leads the dynamic team at the Center, emphasized that a key element of every program has been to ensure participants leave with a sense of hope, despite the often devastating nature of rare diseases.
The event has grown substantially, moving to larger venues to accommodate increasing attendance and include poster presentations. It draws a diverse audience including students, faculty, healthcare providers, patient advocacy organizations, pharmaceutical representatives, legislators, and most importantly, patients and their families.
Dr. Cloyd, in reflecting over this year’s event, emphasized the impact of centering the voices of those most impacted. "The two moms that presented gave eloquent, powerful stories about what their life was like, what they confronted in the way of fear and despair and anxiety, their relationship with their partners. And they described it in such a vivid way that it became deeply embedded in my thinking. And I'm betting that was true for many other attendees," he said.
Through powerful personal stories and practical resources, Rare Disease Day has become an essential platform for education, advocacy, and community building in the rare disease space. The 2025 edition accomplished that . . . and much more. Kudos to the Center for Orphaned Drug Research Team.