The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely on orphan drugs and rare diseases and features stakeholders from across the rare disease world, such as patients, regulatory authorities, pharmaceutical companies, patient advocacy organizations, families, clinicians, scientists and more all coming together to share ideas and further the shared goal of improving outcomes for rare disease patients. This year, Patient Worthy was in attendance to cover the event.
The event began with a pre-conference segment on the afternoon of the 23rd. We covered sessions such as “Fueling the Power of Patient Data,” which discussed how patient data can be vital to improving the quality of research and can then be used to contribute to the development of new therapies. There was also a panel discussion of patients living with a variety of autoimmune disorders, which are rare diseases that causes the immune system to attack another part of the body. Some of the patients on the panel, such as Rory Smith, dealt with multiple diagnoses, such as chronic inflammatory demyelinating polyneuropathy (CIDP), Sjögren’s syndrome, and narcolepsy.
The panel discussed a number of different challenges that they faced, such as the toll living with their conditions could take on their mental health. They also discussed their own tools and copings mechanisms that helped them pull through on especially difficult days. Rory’s mantra was particularly resonant:
“Do something for yourself, do something with someone else, do something for someone else.” – Rory Smith, living with multiple rare disorders
The event also included sessions focused on hot button technologies, such as AI. Presenters discussed concerns about AI and digital health, such as ableism in digital health tools (20% of people that need them can’t use them), the risk of AI algorithms reproducing systemic racial biases (example: an algorithm that could only detect Noonan syndrome in white people), and the challenges of reaching patients that don’t have access to the internet or other technologies.
Along with attending sessions and posting about them on our Twitter and Instagram profiles, we also conducted multiple meetings and interviews throughout the event, so stay tuned for further stories from the conference.
