WASHINGTON, D.C., May 16, 2024, Today, the House of Representatives’ Energy and Commerce Health Subcommittee voted to advance several bipartisan bills important to the rare disease community, two of which encourage rare disease drug development and two that would make it easier for many rare disease patients to access necessary care.
“Today’s markup was a step in the right direction as we work to improve health outcomes for the millions of Americans living with rare diseases,” said Heidi Ross, Vice President, Policy and Regulatory Affairs at the National Organization for Rare Disorders. “There are more than 10,000 known rare diseases, many of which are severe and life-limiting, and too few treatments or knowledgeable health care specialists available to meet patients’ daily needs. NORD urges leaders of the Energy and Commerce Committee to resolve some of the remaining technical issues discussed during the markup today to maintain the strong, bipartisan support for these bills that will foster innovation and address some of the challenges felt by our patients and their families.”
The markup featured the following bills with implications for the estimated 30 million people living with rare diseases, which NORD and its Rare Action Network advocates have been urging Congress to pass:
- Creating Hope Reauthorization Act (H.R. 7384): Approximately half of those living with a rare disease are children and too often their lives are cut short or severely impacted by their rare condition. Rare pediatric disease priority review vouchers (PRVs) reward companies for developing much-needed therapies for rare diseases that primarily affect children, but this incentive will go away unless Congress reauthorizes it before the September 30 deadline.
- RARE Act (H.R. 7383): Life-saving drug development must continue until every adult and child living with a rare disease has access to safe and effective treatments. This bill will ensure incentives to spur rare disease research and development are properly awarded, protecting FDA’s long-standing interpretation of the life-changing Orphan Drug Act.
- Accelerating Kids’ Access to Care Act (H.R. 4758): A common challenge for many rare disease patients is finding a knowledgeable medical specialist, often resulting in the need to travel long distances, including across state lines, for necessary care and treatment. Bureaucratic barriers to out-of-state medical care can be even more daunting for children with health care coverage through Medicaid or the Children’s Health Insurance Program (CHIP). This bill will reduce red tape and make it easier for these members of our community on Medicaid and CHIP to receive the specialized care they need.
- Telehealth Modernization Act (H.R. 7623): Many rare disease patients must travel long distances to meet with their health care providers and expanded telehealth access can greatly reduce barriers in access to care. In 2019, a survey by NORD found that almost 40% of rare disease patients reported needing to travel more than 60 miles to see their health care providers. Robust telehealth access can help reduce barriers to necessary care and treatment for those living with a rare disease. This bill would extend Medicare telehealth flexibilities granted during the pandemic, which are set to expire at the end of this year, for an additional two years.
As part of the markup, the text of the Creating Hope Reauthorization Act and the RARE Act were incorporated into H.R. 3433, the Give Kids a Chance Act. Individually, each of these bills have strong bipartisan support, even if some differences must still be resolved, particularly related to how long to reauthorize the rare pediatric disease priority review voucher program. NORD urges the Committee to reauthorize it for no less than four years given the significant time it can take to develop a safe and effective rare disease therapy.
Ross added, “Negotiations around the Creating Hope Reauthorization Act must be based on current data. As Energy and Commerce Committee Chair Cathy McMorris Rodgers noted in her opening remarks, NORD’s recent analysis found more than half of all vouchers redeemed, awarded or designated through the rare disease pediatric priority review voucher program have happened since 2019. The vast majority of rare diseases helped by this program had no FDA-approved treatment prior to the rare pediatric disease priority review voucher program’s establishment. Rare disease patients are just starting to fully realize the benefits of the program and we urge Congressional leaders not to let it lapse.”
Related resources:
NORD Research: New analysis released on 5/9/2024, Reauthorize the Rare Pediatric Disease Priority Review Voucher Program: Support H.R. 7384, the Creating Hope Reauthorization Act, 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 efforts to advance new and better therapies, shorten the diagnostic odyssey, and improve access to affordable health care and treatment, here.
Media interviews available upon request by contacting [email protected].
