Improving Access to Rare Disease Care

“We have established the country's first rare disease national network called NORD Rare Disease Centers of Excellence. It is made up of 31 institutions across 22 states that are rare disease organizations that specialize in providing diagnostic treatment and research services and participating in the research and the development of treatments for people living with rare conditions."

The National Organization for Rare Disorders (NORD) Breakthrough Summit, held October 17-18, 2022, in Washington, DC, brought together industry, advocacy groups, and institutions to share new research in rare diseases as well as to discuss what can be done to improve access to advanced therapies and inclusion in clinical trials.

CGTLive spoke with Pam Gavin, executive vice president, NORD, to learn more about what the organization is doing to improve access to care for rare disease populations. Gavin discussed a 2-pronged approach that NORD is taking to improve access: educating the community about new therapies and improving logistical access to therapies for many people that are far from specialists.

Gavin discussed the NORD Rare Disease Centers of Excellence network, which includes institutions and healthcare systems that meet strict requirements, including fostering community connection, patient advocacy, and medical expertise. She also touched on strategies to help alleviate issues with logistical access, including creating hub-and-spoke models of treatment centers and greater utilization of telemedicine.