Rare Disease Policy Disparities Persist Despite Incremental State Progress

Event summary

  • NORD’s 2025 State Report Card graded states on rare disease policies, assigning a national average grade of “B”.
  • Four states (Colorado, Delaware, Maine, and New Jersey) received the highest grade of “A”.
  • Thirteen states received failing grades for medical nutrition coverage, a key area of concern.
  • Federal agencies’ reduced enforcement of short-term insurance plans has shifted patient protection responsibility to state laws.
  • Nineteen states currently lack Rare Disease Advisory Councils (RDACs), limiting patient input on policy.

The big picture

NORD's report highlights the ongoing challenge of equitable healthcare access in the US, particularly for individuals with rare diseases. The decentralization of healthcare policy, driven by federal regulatory shifts, places increased pressure on states to fill the void and protect vulnerable populations. The lack of uniform standards creates a patchwork system where access to care is heavily dependent on geographic location.

What we're watching

Policy Shifts
The continued reliance on state-level regulation for short-term insurance plans will likely lead to increased lobbying efforts and policy divergence across states, creating complexity for patients and providers.
RDAC Expansion
The pace at which remaining states establish RDACs will be a key indicator of patient advocacy effectiveness and the willingness of policymakers to incorporate patient perspectives into healthcare decisions.
Nutrition Coverage
How states address the widespread lack of medical nutrition coverage will reveal the political will to support vulnerable patients and potentially influence reimbursement models for specialized therapies.