#332 Building Stronger Rare Disease Organizations: NORD CEO’s Insight

This week we attended the 2025 Connecticut Rare Disease Forum organized by BioCT and held at the beautiful Jackson Laboratory. This afternoon was filled with industry-led discussions on innovation in rare disease diagnostics and treatments, along with the promising impact of these advancements on patient outcomes.

After the events wrapped, I caught up with Pamela Gavin, who serves as the President and CEO of the National Organization for Rare Disorders (NORD). She was on the panel, “Bringing Science to the Patient: Moving Upstream Toward Durable Response and Cures.”

This in-person conversation explores the vital role of patient advocacy organizations, particularly NORD, in supporting individuals with rare diseases. It discusses the evolution of patient advocacy, the importance of genetic testing, and the need for a robust workforce in genetic counseling. We emphasize the significance of building effective organizations and learning from successful models to enhance the impact on rare disease communities.

Takeaways

• NORD serves as a safe, trusted place for people to connect regarding rare diseases.
• Patient advocacy organizations play a crucial role in drug development and research.
• The landscape of patient advocacy has evolved significantly in recent years.
• Building effective rare disease organizations requires understanding best practices and community needs.
• Learning from successful organizations can help new groups avoid common pitfalls.
• Genetic testing is becoming increasingly important in diagnosing rare diseases.
• The future of genetic counseling faces challenges due to increasing diagnoses and limited workforce.
• Engaging the next generation in genetics is essential for the future of healthcare.
• Collaboration and communication within the rare disease community can lead to better outcomes.
• Advocacy organizations must adapt to the rapid advancements in genetic technology and testing.

Our Guest

Pamela Gavin was appointed chief executive officer of the National Organization for Rare Disorders (NORD®) in May 2024, becoming the third CEO in the organization’s 42-year history. Gavin’s deep connection to the rare disease community and extensive professional experience make her uniquely suited to lead NORD into its next chapter of innovation, advocacy and impact. Before joining NORD, she held several influential roles in healthcare innovation and safety, including as senior director, Strategic Business Initiatives at the University of Pittsburgh Medical Center, where she launched emerging technologies to improve healthcare delivery; government consultant responsible for developing the Federal Safety Reporting Portal for the NIH, FDA and other organizations; founder and executive of SafeCare Systems, a pioneer in safety management information systems; and division director at the Harvard Risk Management Foundation. Gavin holds a Master of Business Administration in Healthcare Management from Northeastern University and a bachelor’s degree from Smith College.

DNA Today Episodes Referenced

#147 CF Series: Bijal Trivedi on "Breath From Salt"

#149 CF Series: Gunnar Esiason on Patient Advocacy

#293 Smith-Magenis Syndrome with Parent Scotti Taylor

There are countless episodes about rare diseases, many of which are captured in this YouTube playlist.

Get involved with a rare disease organization here on NORD’s website.

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