The decision to suspend a key advisory committee has stakeholders concerned that newborns’ health, especially those with rare conditions, may be at risk.
Newborn Screening Panel Terminated
In April, the Department of Health and Human Services opted to disband the Advisory Committee on Heritable Disorders in Newborns and Children. The Committee has long shaped newborn screening programs across the country.
Since 2003, the panel had advised federal and state authorities by creating the Recommended Uniform Screening Panel, which outlines disorders to include in tests given to every newborn at birth.
Currently, the Recommended Uniform Screening Panel includes 36 core disorders and 26 secondary disorders from hyperplasia to heart disease to hearing loss. The Committee’s input has been critical in ensuring early detection of conditions that, if left untreated, can lead to irreversible harm and preventable deaths.
While states make the ultimate decision on which disorders are screened, most health plans are required to cover screenings included in the HRSA-supported comprehensive guidelines without charging a co-payment, co-insurance or deductible.
Diagnosis and Treatment in Limbo
More than 15 million children in the United States live with a rare disease, most of them genetic.
Diagnostic tests have improved significantly following the Committee’s past guidance. And through recommended universal testing, many conditions can be treated or managed before symptoms appear. Eliminating this advisory body creates a dangerous gap not only in expert knowledge, but in the quality of care the youngest Americans receive.
Two serious conditions – metachromatic leukodystrophy and Duchenne muscular dystrophy – were being considered for inclusion in the screening panel, until the committee’s February meeting was abruptly canceled. Both conditions now face indefinite delays in being added, meaning babies may not be diagnosed in a timely manner and new FDA-approved treatments may not reach patients early, when they could be most effective.
Advisory Committees Under Scrutiny
A broad federal initiative to shrink the number of advisory bodies resulted in the termination of the Advisory Committee on Heritable Disorders in Newborns and Children, which was not mandated by statute. Experts warn that its dissolution is irresponsible in a time of growing medical need.
At the same time, new conflict of interest policies at the FDA have stirred debate over who will remain eligible to serve on the remaining committees.
For families navigating the unknowns of rare disease, the loss of expert guidance on newborn screening is more than a bureaucratic shift. It’s a setback with the potential for lifelong consequences. Congress and agency leaders will need to consider how to restore the trust and continuity that are vital to protecting some of the nation’s most vulnerable patients.
