We are nearly two-thirds of the way through 2025, and it’s no secret that the first seven months have been marked by ground-shifting changes in the rare disease policy, biomedical, public health, and regulatory ecosystems. As Congress enters its August recess, and more than 600 rare disease advocates fan out across the country to meet with members of Congress in their home offices as part of Rare Across America, let’s look at what opportunities still exist for Congress to support rare disease policy priorities.
The Schedule – A Tight Window of Opportunity
August recess is when lawmakers spend time in their home districts or states, meeting with constituents, attending community events, and visiting local institutions. It is typically the longest break of the year. The House went into recess last week, and the Senate will follow on August 4. Both chambers are set to return to DC on September 2, with a looming September 30 deadline to fund the government for the next fiscal year. That leaves limited time to move critical legislative priorities, either as part of a funding package or a year-end legislative package.
Rare Disease Priorities in Congress
In addition to the critical implications of the appropriations process, which we have covered in previous posts, Congress can and must take action to advance rare disease policies affecting the diagnosis, treatment opportunities, and access to care for the more than 30 million Americans living with rare diseases.
Rare Pediatric Disease Priority Review Voucher (PRV) Program
One of the most urgent opportunities is the renewal of the now expired Rare Pediatric Disease Priority Review Voucher (PRV) Program as proposed in the Give Kids a Chance Act. The PRV Program incentivizes companies to develop treatments for rare pediatric conditions which are particularly challenging due to small populations, difficulties associated with conducting trials for children, diagnostic delays, and more.
You can learn more about the PRV Program here.
In 2024, the Give Kids a Chance Act was unanimously passed in the House and was set to be included in an end-of-year package of healthcare policies. Unfortunately, most of that package was ultimately dropped from the final bill, and as a result, the Rare Pediatric Disease PRV Program expired on December 20, 2024. But there is hope!
There are promising signs of progress. Give The Give Kids a Chance Act already has over 200 cosponsors in the House, and it has been reintroduced in the Senate, reviving the possibility of the PRV Program being renewed. Given its bipartisan support and the implications for pediatric therapy development, Congress must act before the end of the year to restore this effective program that carries no cost to the government or taxpayers.
We know the PRV Program works, and without Congressional action, less research into pediatric treatments will occur. Our recent study in collaboration with Tufts Medical Center, predicted that an average of 45 new treatments for non-oncology, rare pediatric diseases would be approved over the next 10 years, exciting progress that could be at risk.
Accelerating Kids’ Access to Care Act
Another commonsense policy that could have big impacts for patients is the Accelerating Kids Access to Care (AKAC) Act. The bill would make it easier for kids who have Medicaid to access care in another state by streamlining the provider enrollment process and reducing administrative burdens.
Learn more about AKAC Act here.
Millions of rare disease patients rely on Medicaid to access the care and services they need, oftentimes requiring them to travel to another state if no specialist familiar with their condition exists in their home state. One hurdle in the approval process to obtain care in another state is the doctor must be enrolled in the patient’s home state’s Medicaid program. This can add months and heavy administrative burdens for the doctor, delaying essential care.
The AKAC Act has strong bipartisan support and was also included in – but then dropped from – the end-of-year healthcare policy package in 2024. The bill has been reintroduced in the House and the Senate and was included in the House-passed version of the recent reconciliation legislation, H.R.1. The bill was removed in the Senate’s version for cost reasons, even though the predicted cost is very low.
Newborn Screening
The Newborn Screening Saves Lives Act (NBSSLA) in 2008 established national newborn screening guidelines and helped facilitate comprehensive improvements in the federal government’s support of newborn screening programs. Prior to NBSSLA, the number and quality of newborn screening tests varied greatly by state. Today, all 50 states and the District of Columbia require screening for at least 32 treatable conditions.
As is often the case with federal programs, NBSSLA authorized newborn screening programs for a defined period, initially for 5 years. A second NBSSLA was passed in 2014, extending the programs through 2019. Unfortunately, since 2019, efforts to pass a new bill reauthorizing the core newborn screening programs have been unsuccessful. Due to the essential nature of the federal government’s role in supporting newborn screening programs in the states, Congress has continued to appropriate money to support the programs and grants run by the CDC, NIH, and HRSA, despite the authorization lapsing.
Earlier this year, the federal government’s role in newborn screening was drastically altered when the Advisory Committee on Heritable Disorders in Newborn and Children (ACHDNC) was terminated and the Health Resources & Services Administration’s newborn screening programs were slated for elimination in the President’s budget proposal. In April, the National Academies of Science and Medicine also released a Congressionally requested study outlining the current status and opportunities to advance newborn screening in the US. These developments make it even more essential for Congress to act.
Thankfully, there are signs of bipartisan support for newborn screening action in the fall, starting with its inclusion in an Energy and Commerce Committee hearing on July 16. Together with other leading organizations, the EveryLife Foundation is working with Congressional champions to craft changes to the latest version of the reauthorization bill and support its movement in Congress.
The Time is Now
In a year marked by changes to our rare disease ecosystems, Congress has the opportunity to advance policies that will shape the rare disease landscape for years to come, advancing treatment options for pediatric diseases, improving access to care for children, and preserving and enhancing our newborn screening infrastructure.
Continue to follow the EveryLife Foundation for updates as these bills advance in Congress and opportunities to take action arise.
