06/13/2025 | News release | Distributed by Public on 06/13/2025 09:15
June 13, 2025
Early diagnosis, especially for children, has long been a critical issue. But the government's disbandment of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) in April raises significant concerns about the future of newborn screening in the U.S., despite the administration's public claims that they are working to improve Americans' health.
Benjamin Franklin said, "An ounce of prevention is worth a pound of cure." Although this well-known proverb dates back to the formation of the United States, it is an especially important one to keep in mind just now. With the elimination of the ACHDNC, it's time for pharmaceutical and biotechnology industry leaders to heed history and take action by supporting newborn screening as a key preventive program, one which provides the "ounce of prevention" that will save lives.
At the World Orphan Drug Congress USA this past April, four moms led an impassioned panel discussion on newborn screening: Katheryn Alexander, Lesa Brackbill, Christina M. Harman, and Jennifer McNary. The inspirational panelists outlined the void left by the disbandment of the committee, which effectively eliminated support for early identification and intervention for many rare diseases, such as Type 1 spinal muscular atrophy (SMA). The loss of the ACHDNC also leaves no identified pathway to add more conditions, such as metachromatic leukodystrophy (MLD), to the health secretary's Recommended Uniform Screening Panel. The panel demonstrated that affected families will lose their quality of life, and at worst, will suffer preventable deaths.
Pharmaceutical and biotech companies have a long history of supporting diagnostic and screening tests for obvious reasons. Diagnosis is a prerequisite for treatment and enrolling in clinical trials to identify new treatments. Without the ACHDNC, the path forward for newborn testing is left without a north star. Industry leaders have an unmatched opportunity, not only to call for reinstatement of the committee, but also to step in and champion screening efforts that ensure that our most vulnerable populations are healthy and thriving through long-term, sustained investments.
Specific challenges remain unresolved, which pharmaceutical and biotech experts will need to address. These include:
Fast approaching, BIO 2025 provides an opportunity for the biotech and pharmaceutical industry to discuss this challenge with patient advocacy groups and explore how they might stand together to support reinstating the ACHDNC and also expand and standardize newborn screening. Infants and their families in this country deserve to be healthy. It's not just good medicine; it's good business.
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POSTED BY: Deborah Hernandez