Policy & Rare Disease
Policy doesn’t change unless we speak up—and it hits differently when the people most impacted are leading the conversation. This section is your launchpad into rare disease policy: created by a patient who’s been in the hospital bed, the Hill meeting, and the briefing room. Whether you’re just starting out or already raising your voice, here you’ll find tools, templates, and insights to help make the system work with us, not against us. This is advocacy, activated.
What Is Rare Disease Policy?
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.
Understanding the Policy Landscape
The policy world can feel like a maze—but once you understand the map, it becomes a powerful tool for change. The rare disease policy landscape stretches across federal and state levels, weaving through agencies, legislative bodies, and stakeholder communities.
This section breaks it down clearly: who’s responsible, what each level of government can do, and how patients, caregivers, researchers, providers, and other advocates fit into the process. By understanding the roles and relationships between federal, state, and community players, you’ll see how the pieces connect—and where your voice matters most.
Current Rare Disease Policy Priorities
Each year, the rare disease community comes together to champion policies that push for better care, faster innovation, and greater equity.
These policy priorities don’t just shape programs—they shape lives. Whether you’re a patient, provider, advocate, researcher, or policymaker, here are the key legislative issues to watch and support in 2025 and beyond.
Who Policy Impacts—And How
Policy isn’t just a process. It’s personal. Every rare disease policy—whether it’s about research funding, drug development, insurance coverage, or data collection—touches real lives. From patients navigating endless diagnoses to providers facing resource gaps, every stakeholder is shaped by the systems that policy creates or neglects.
Understanding who is impacted, how they’re affected, and how they can speak up is key to making rare disease policy truly inclusive and effective.
This section provides a breakdown of how different groups experience rare disease policy—and how their voices can help shape it.
Advocacy 101
Advocating for rare disease policy isn’t just for professionals or lobbyists. It’s for all of us. You don’t need to be an expert—just someone willing to share your story, speak up, and show up.
Policy Toolkit
Whether you’re ready to call your Senator or just want to understand the basics, here’s a toolkit to help you take the next step—wherever you are in your advocacy journey.
Rare Voices: Stories That Move Policy
Stories change hearts—and laws. In this section, we highlight real advocates shaping policy through lived experience. Want to share your voice? Reach out to be featured.