Protect the Orphan Cares Act from Repeal
This is urgent.
You know the frustration of finding out that a treatment potentially won’t be covered by insurance, or the medication costs thousands of dollars a month, or that it's only approved for adults, even though your teenager desperately needs it.
The Orphan Cares Act was designed to fix many of these problems. And we won a major victory when it was included in H.R. 1, the "One Big Beautiful Bill" that passed earlier this year. This legislation builds on the Orphan Drug Act that made porphyria treatments possible in the first place but it adds new protections to make sure you can actually access those treatments.
What this law does:
Creates stronger incentives for companies to develop treatments for rare diseases
Improves insurance coverage and reduces out-of-pocket costs for orphan drug
Gives patients like you a real voice in how new treatments are developed
Makes it easier for doctors to prescribe medications off-label when no approved treatment exists
But now, there are efforts underway to repeal it.
Some members of Congress want to strip the Orphan Cares Act out of the law by passing a repeal. If they succeed, we go back to square one - back to the same access barriers, the same insurance denials, the same impossible costs that kept you from getting the care you needed.
We can't let that happen.
Take Action Now:
Congress needs to hear from you immediately. Your Senators especially need to understand what repealing this law would mean for real people with porphyria.
Call or email your two senators and your representative in Congress. You can find their contact information at www.congress.gov.
What to say or write:
"My name is [YOUR NAME] and I live in [YOUR CITY]. I'm calling to urge you to oppose any efforts to repeal the Orphan Cares Act, which was included in H.R. 1.
I have porphyria [TYPE], a rare disease. Even though treatments exist, [describe your access challenges, including insurance denials, high costs, availability issues]. The Orphan Cares Act would help people like me actually get the medications we need.
Repealing this law means taking away hope from hundreds of thousands of Americans with rare diseases. It means going backward on patient access, affordability, and innovation.
Please stand with rare disease patients and oppose any efforts to repeal the Orphan Cares Act. Don't take away the progress we've fought so hard to achieve.
Thank you."
Feel free to impart a sense of urgency. Let them know this isn't theoretical, and that repealing this law has real consequences for your health, your family, and your future.