The North America Rare Disease Summit focused on the need for centralized data to improve the lives of 30 million people living with rare conditions
Stakeholders debate at the North America Rare Disease Summit on the challenges regarding treatment approvals, the importance of a deep proactive registry, and breaking data silos
NEW YORK, Nov. 02, 2023 (GLOBE NEWSWIRE) -- The North America Rare Disease Summit, organized by Bamberg Health, gathered over 40 specialists on Rare Diseases. The debate focused on the role of public awareness and patients, government initiatives, access and financing to research and treatments, and the use of new tools in accelerating processes.
In the session regarding government initiatives, panelists agreed that every stakeholder needs to be involved to achieve sustainable policies, considering the perspective and experience of everyone who will participate in its implementation. “Patients and their families are the real experts. Knowing how to read the parameters isn’t actually understanding what it means to live with this disorder”, said Benjamin Forred, Director of Translational Research at the CoRDS Rare Disease Registry.
When asked what patient associations can do to help the FDA move forward on rare diseases, Peter Marks, Director of the CBER in the FDA, responded that the agency has a lot of participation stages where it is crucial to have the patient experience, and organizations need to participate as much as they can. “We’re changing things inside the FDA, to accelerate the response, but it’s huge and it will take longer than we’d like”.
In the panel “Innovation, Funding and Access to Orphan Drugs”, Megan Nolan, CEO and co-founder of The Children's Rare Disorders Fund, called for the urgency of funding research that can improve patients' lives. “We’re talking about millions of dollars and, now, most are funded by patient advocates, because for industry is not profitable. Families can’t take on this battle too, it’s too much. Families of caregivers are breaking, they can’t also be in charge of financing research”.
Another key topic addressed was the challenges brought by a healthcare system that is not centralized when it comes to sharing interoperable data and unified approaches to improve diagnosis and therapies.
This event is the first edition in the United States from a series held by Bamberg Health with the goal of setting a strong network that connects stakeholders to achieve real change and positive impact in healthcare.
Camila Demarco
camila@bambergsummits.com
