Today, the EveryLife Foundation for Rare Diseases applauded the signing of HB 736 in North Carolina that will help save the lives of nearly 700 babies born in the state each year. The law, referred to as RUSP alignment legislation, implements a three-year timeline in which the screening must begin for new conditions added to the federal Recommended Uniform Screening Panel (or RUSP), and ensures that the North Carolina Department of Health and Human Services shall provide a report on the status of the addition at 18 months and every six months the addition is delayed past three years.
North Carolina currently screens for 32 out of the 35 conditions on the RUSP. Newborn screening detects conditions that, if left untreated, can cause disabilities, developmental delays, serious illness or even death. If diagnosed early, many of these disorders can be managed successfully and at a lower long-term cost – not only saving lives, but also saving state funds.
This new law brings the total number of states that have adopted RUSP alignment legislation in 2021 to four, following similar legislation that was passed in Ohio, Arizona, and Georgia. Nationally, the total is six states, including California and Florida which passed in 2016 and 2017 respectively. In 2022, the Foundation will lead efforts to pass RUSP alignment legislation in Iowa, Maryland, and Mississippi. Advocates residing in these states are invited to take an online survey to join the effort.
“Each year, new screening tests are developed to diagnose diseases early enough to benefit from life-saving treatment. RUSP alignment legislation will enable North Carolina families to ensure that their babies have the best chance at a healthy life possible,” said EveryLife Foundation Chief of Policy, Advocacy, and Patient Engagement Annie Kennedy. “We have seen a growing interest in states passing this critical legislation and families are counting on it to enhance and expand newborn screening. We are grateful to the North Carolina state legislature for passing, and to the Governor for signing, this impactful legislation. These new laws will ensure that babies born in North Carolina have the same opportunity for diagnosis and treatments as babies born across state lines.”
The EveryLife Foundation partnered with more than 50 patient advocacy organizations to support the legislation, including the National MPS Society, Rare Disease Innovations Institute, and Taylor’s Tale. The bill was championed by Representatives William Brisson (Bladen, Sampson), Michael Wray (Halifax, Northampton) and Becky Carney (Mecklenburg).
“This law addresses the critical element of time and will provide babies screened for treatable rare diseases, a chance at life, before it is too late. These terminal diseases are progressive, and time cannot be reversed. The sooner we can provide therapy to an infant, the better their outcome,” said Terri Klein, National MPS Society President and CEO. “The FDA approved Aldurazyme ™, a life-saving therapy for MPS I, 18 years ago. Five years ago, MPS I was added to the RUSP, and it has been three years since North Carolina passed the legislation to add MPS I to its Newborn Screening Program. Thanks to this law, babies born in North Carolina will finally have access to screening for MPS I.”
The EveryLife Foundation’s newborn screening advocacy program is made possible by the support of Sanofi Genzyme, and other industry partners including Alexion Pharmaceuticals, Pfizer, bluebird bio, BridgeBio, Novartis Gene Therapies, Orchard Therapeutics, and Sarepta Therapeutics.
To learn more about the legislation and how to support newborn screening, visit RareScreening.org.
