NORD: Rare Disease Advocates in ‘Unique Position’ to Educate Congress
Organization emphasizes importance of engaging lawmakers on health policy
As the 118th U.S. Congress begins its work, the National Organization for Rare Disorders (NORD) is showcasing the importance of engaging in health policy and what rare disease patient advocates can do to make their voices heard.
The organization is the leading patient advocacy organization dedicated to improving the lives of rare disease patients and their families.
Between 25–30 million U.S. residents are thought to live with at least one of the roughly 7,000 rare disorders, among which is aromatic l-amino acid decarboxylase (AADC) deficiency, an extremely rare condition with only about 120 cases reported.
“Engaging in health policy can be confusing, feel overwhelming, and at times appear quite tedious,” said Karin Hoelzer, NORD’s director of policy and regulatory affairs, in an organization post. “But rare disease patients and caregivers are in a unique position to educate lawmakers and to raise awareness about what it is like to live with a rare disease.”
No advocacy experience is needed to lobby lawmakers about rare disease patients’ needs and priorities, Hoelzer said, adding engagement can take a variety of forms, depending on the situation and a person’s comfort levels. It’s important for legislators to hear the diverse perspectives from a broad range of patients and caregivers.
“Your perspective makes a difference on Capitol Hill as well as in state capitals across our nation,” Hoelzer said. “Patient and caregiver engagement can, for instance, help ensure critical research programs are sufficiently funded, new health policies account for key patient priorities, and lawmakers do not make decisions impacting your ability to access lifesaving therapies hastily and in a vacuum.”
NORD suggests people engage in rare disease advocacy in five ways about issues important to the community:
- By sharing their stories online with the rare disease community. This can help “humanize” patient and caregivers’ experiences with rare disorders, many of which are unfamiliar to lawmakers. Posting narratives can also help them understand the impact of their decisions.
- By contacting a member of Congress about a key policy issue such as access to medical nutrition and out-of-state healthcare, regulatory reform, and federal efforts to accelerate new treatments. Advocates can send an email to their lawmaker or arrange a virtual or in-person meeting with them or a staff member.
- By joining a group such as NORD’s Rare Action Network or Advocacy Taskforce to lend support to federal and state policy issues.
- By participating in public comment opportunities at workshops or advisory committee meetings involving the U.S. Food and Drug Administration (FDA). These events provide opportunities to make patient and caregivers’ experiences and perspectives heard. The FDA and Medicare & Medicaid Services have slated meetings for this year that affect the rare disease community. Other ways include participating in FDA-hosted patient-focused drug development meetings and patient listening sessions.
- By engaging with their state’s Rare Disease Advisory Council. By now, most states have established such a council to better represent the rare disease community and give patients a greater voice.
“We need to educate lawmakers – new and returning – about our cause and foster new champions for rare disease policy,” Hoelzer said.
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