Rare Disease Policy
If you are interested in participating in advocacy training, check out the EveryLife Foundation advocacy toolkit here or consider joining Rare Disease Legislative Advocates (RDLA) Young Adult Representatives (YARR). Learn more about YARR here.
Federal Level Advocacy
- Legislative Scorecard
- Check out your legislators score for insights on your legislator’s voting record on rare disease legislation here
- Find your Congressional Representatives here
- Find your US Senators here
State Level Advocacy
- The RareAction Network ® (RAN) here
- Rare Disease Advisory Council (RDAC) here
- Find your State Legislators here
Legislation to Watch
People with Barth syndrome and their families are the experts about the impact of living with this condition. Your voice matters! Your journey gives you the power to shape public policy to improve the lives of people living with Barth syndrome. Below you will find legislation that is important to our rare disease community.
119th Congress
Accelerating Kids’ Access to Care Act of 2025
To amend titles XIX and XXI of the Social Security Act to streamline the enrollment process for eligible out-of-state providers under Medicaid and CHIP aka Accelerating Kids’ Access to Care Act H.R. 1509 /S. 752 allows states to streamline the process for out-of-state pediatric care providers to enroll in another state’s Medicaid program, while also safeguarding important program integrity processes. The legislation enables smooth coordination across state lines by clarifying the process by which state Medicaid programs can cover this care regardless of where the child lives and where their care is received.
Full text of House bill here. Full text of Senate bill here
Full list of supporting organizations here
See Senator Michael Bennet press release here
See Congresswoman Lori Trahan press release here
Give Kids a Chance Act of 2025
The Creating Hope Reauthorization Act H.R. 1262 combines two critical initiatives — the original Give Kids a Chance Act and the Creating Hope Reauthorization Act — to spur drug development for pediatric rare diseases, improve outcomes for patients, and close gaps in pediatric drug research.
Background:
Children comprise as many as half of those living with rare diseases, yet treatment options for children remain extremely limited compared to those for adults. The Give Kids a Chance Act would reauthorize the Food and Drug Administration priority review voucher (PRV) program which allows pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases. Since the bill’s passage in 2011, 53 PRVs have been awarded for 35 different rare pediatric diseases.
Additionally, thousands of successful drug combination therapies are now being studied and developed for adults, but not for children. The Give Kids a Chance Act also authorizes the FDA to direct companies to study a combination of cancer drugs and therapies in pediatric trials as well.
See full bill here
See Rep. Dingell's press release here
See EveryLife Foundation one pager here
EXPERT Act of 2025
Scientific External Process for Educated Review of Therapeutics (EXPERT) Act of 2025 H.R. 1532/ S. 822
Over 90% of rare diseases have no FDA-approved treatment, and applications in the pipeline often face challenges with approval. Evaluating rare disease therapy effectiveness and safety requires different clinical approaches than those for the average medical product, because of the small populations. Complicating this, FDA reviewers often lack expertise in specific rare diseases, hampering their ability to make informed decisions about applications. Meanwhile, rare disease experts often have conflicts of interest preventing them from participating in FDA advisory committees, due to their involvement in research and trials for rare disease therapies. This legislation bridges those gaps by allowing scientists and FDA reviewers to share their expertise and knowledge in a product-agnostic setting without compromising the integrity of the review process.
This legislation seeks to bridge the gap between rare disease expertise and regulatory expertise through the Externally-Led Scientific Focused Drug Development (EL-SFDD). These quarterly meetings will provide an opportunity for enhanced collaboration between medical experts, drug sponsors, scientific organizations, and patient advocates to discuss the challenges impacting the development of rare disease treatments, identify scientific opportunities to facilitate development, discuss novel clinical trial designs, and align on endpoints to address unmet medical needs for rare disease patients. Each meeting will focus on a different rare disease topic, and the FDA will report annually on how these sessions are helping to shape and improve its internal review process for rare diseases.
Read full text here
See Rep. Matsui's press release here
HELP Copays Act
The bill to amend title XXVII of the Public Health Service Act to apply financial assistance towards the cost-sharing requirements of health insurance plans, and for other purposes (HELP Copays Act) S. 864 offers a two-part solution that will eliminate barriers to treatment for some of the most vulnerable Americans—those who live with serious, complex chronic illness—and ensure that they can afford the medically necessary, often life-saving medications prescribed by their doctors:
- The bipartisan HELP Copays Act requires health plans to count the value of drug copay assistance toward patient cost-sharing requirements. This would bring much-needed relief to financially vulnerable individuals by ensuring that all payments—whether they come directly out of a patient’s pocket or with the help of copay assistance—count towards their out-of-pocket costs.
- This legislation also closes a loophole that allows many employer health plans to deem certain covered drugs as “non-essential,” which means that the insurer will not count any cost-sharing toward the patient’s deductible and out-of-pocket maximum. Your bill will require all private plans to count all costsharing for covered medications to accrue to a patient’s deductible and out-of-pocket maximum.
For more information visit the All Copays Count Coalition website here.
ORPHAN Cures Act
The "Optimizing Research Progress Hope And New Cures Act" or "ORPHAN Cures Act" H.R. 946 seeks to amend title XI of the Social Security Act to refine the exclusion criteria for orphan drugs within the Drug Price Negotiation Program. The primary objective is to expand and clarify the conditions under which orphan drugs are excluded from price negotiations. Specifically, the amendment to Section 1192(e) introduces a provision that excludes the time during which a drug or biological product was classified as an orphan drug from the calculation of the elapsed time since its approval or licensure. This aims to ensure that the period a drug is designated for treating rare diseases does not count against its eligibility for price negotiation. Additionally, the amendment modifies the language in paragraph (3)(A) to broaden the scope from "only one rare disease or condition" to "one or more rare diseases or conditions," aligning the definition with that in section 526(a)(2) of the Federal Food, Drug, and Cosmetic Act. This change allows for a more inclusive interpretation of what constitutes an orphan drug, potentially increasing the number of drugs that qualify for this exclusion. The legislation does not specify any new funding allocations or financial figures, nor does it outline specific timelines or deadlines for implementation.
See Rep. Don Davis press release here
