WASHINGTON, D.C., September 18, 2024 — Today, members of the House Energy & Commerce Committee unanimously voted to advance out of Committee the Give Kids a Chance Act (H.R. 3433), bipartisan legislation to spur more pediatric rare disease drug development, and the Telehealth Modernization Act (H.R. 7623), which would extend for another two years flexibilities that have enabled broader use of telehealth services.
The Give Kids a Chance Act includes provisions to reauthorize the Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program through 2029. The RPD PRV program offers a vital incentive encouraging drug developers to invest in rare pediatric diseases, which often face significant challenges in research and development. According to a recently released NORD report, as of April 30, 2024, the program has successfully supported the development of 53 life-enhancing treatments, benefiting 39 rare disease patient communities, nearly all of which had no FDA-approved treatment prior to the program’s establishment in 2012. This program has tremendous support within our patient community, with close to 200 patient organizations joining NORD in sending a letter of support to House and Senate leaders earlier this week, urging its swift reauthorization.
The Give Kids a Chance Act also incorporates additional provisions from bipartisan legislation to ensure rare diseases are studied in children, including:
- The Innovation for Pediatric Drugs Act (H.R. 6664), which updates how research is currently funded as part of the Best Pharmaceuticals for Children Act (BPCA), which helps close data gaps around pediatric uses for approved drugs, and strengthens FDA’s ability to enforce post-market commitments around pediatric studies; and
- The RARE Act (H.R. 7384), which would cement the FDA’s long-standing interpretation of how to properly award orphan drug exclusivity, a key drug development incentive established by the life-changing Orphan Drug Act.
“In 2024, half of the estimated 30 million Americans living with rare diseases are children and many see their lives cut short by these devastating and progressive conditions,” said Heidi Ross, Vice President of Policy & Regulatory Affairs at the National Organization for Rare Disorders (NORD). “We are pleased to see the House Energy and Commerce Committee take this important step toward addressing the urgent and often life-threatening needs in rare disease. NORD applauds lawmakers on both sides of the aisle for working together to help millions of children who need treatment options. However, if Congress does not pass legislation to reauthorize the RPD PRV program, it will lapse on September 30, 2024, which would be devastating to pediatric rare disease patient communities desperate for new and innovative therapies.”
Additionally, the Energy and Commerce Committee also took an important step forward to protect continued access to telehealth services for millions of Americans by voting to advance the Telehealth Modernization Act out of Committee. Telehealth has been critical for rare disease patients, where there are often only a few providers in the country with the specialized knowledge necessary to treat their condition. This results in significant geographic barriers to access, longer wait times, and worse outcomes. A 2019 NORD survey of rare disease patients and caregivers found that nearly 40% of respondents traveled more than 60 miles to obtain medical care.
The Telehealth Modernization Act would extend flexibilities given to Medicare providers during the COVID-19 pandemic, which include waiving unnecessary paperwork requirements, permitting reimbursement for audio-only telehealth, expanding the types of providers eligible to provide care via telehealth, and permitting rural health centers and federally qualified health centers to receive reimbursement for telehealth for an additional two years.
NORD thanks the Energy and Commerce Committee members for their bipartisan, unanimous support to advance these important bills as a critical step towards ensuring rare disease patients of all ages have access to necessary care and treatment. Next, these bills head to the full House of Representatives for a vote.
The time for action is now, since the Rare Pediatric Disease PRV program is set to lapse at the end of this month, and telehealth flexibilities will expire at the end of this year, without Congressional action. Your voice can make a difference! Contact your lawmakers NOW to share why the Rare Pediatric Disease Priority Review Voucher program must continue and how telehealth has benefited you!
Related resources:
NORD Research: New analysis by NORD, Reauthorize the Rare Pediatric Disease Priority Review Voucher Program: Support H.R. 7384, the Creating Hope Reauthorization Act, can be read here.
Advocate with NORD: Individuals looking to make a difference by getting involved in rare disease policy and advocacy are encouraged to join our action alerts here.
NORD Policy Statements: Learn about NORD’s public policy work and our efforts to advance new and better therapies, shorten the diagnostic odyssey, and improve access to affordable health care and treatment, here.
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