Rare disease policy refers to the laws, regulations, and funding decisions that shape how people with rare conditions receive care. From newborn screening to insurance coverage, research investments to FDA approvals, these policies directly affect what treatments exist, who can access them, and how quickly. 

With more than 30 million Americans living with a rare disease, and 95% of those conditions lacking an FDA-approved treatment, policy is often the difference between hope and hardship. Advocacy ensures our voices are heard in the rooms where these decisions are made.