[Federal Register Volume 90, Number 243 (Monday, December 22, 2025)]
[Notices]
[Pages 59847-59848]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2025-23574]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Health Resources and Services Administration
Addition of Metachromatic Leukodystrophy to the Recommended
Uniform Screening Panel
AGENCY: Health Resources and Services Administration (HRSA), Department
of Health and Human Services.
ACTION: Notice.
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SUMMARY: The Health Resources and Services Administration (HRSA)
published a Federal Register notice on August 14, 2025 (90 FR 39196),
requesting comments from the public on the potential recommendation of
adding Metachromatic Leukodystrophy (MLD) to the Recommended Uniform
Screening Panel (RUSP). After consideration of public comments and
evidence-based reports, HRSA recommended to the Secretary of Health and
Human Services (HHS) that MLD be added to the RUSP. The Secretary of
HHS has accepted the recommendation as detailed in this notice.
Conditions listed on the RUSP are part of the evidence-informed
preventive health guidelines supported by HRSA for infants, children,
and adolescents. Non-grandfathered group health plans and group health
insurance issuers are required to cover screenings included in these
HRSA-supported comprehensive guidelines without cost-sharing (e.g.,
copayment, co-insurance, etc.). Please see the RUSP (https://newbornscreening.hrsa.gov/about-newborn-screening/recommended-uniform-screening-panel) for additional information.
FOR FURTHER INFORMATION CONTACT: CDR Leticia Manning, Newborn Screening
Team Lead, Division of Services for Children with Special Health Needs,
Maternal and Child Health Bureau, HRSA, 5600 Fishers Lane, Rockville,
Maryland 20857 or [email protected].
SUPPLEMENTARY INFORMATION: The RUSP is a list of conditions that the
Secretary of HHS recommends for states to screen as part of their state
universal newborn screening (NBS) programs. Conditions on the RUSP are
chosen based on evidence that supports the potential net benefit of
screening, the ability of states to screen for the disorder, and the
availability of effective treatments. Although states ultimately
determine what conditions their NBS program will screen for, it is
recommended that every newborn be screened for all conditions on the
RUSP. Conditions listed on the RUSP are part of the comprehensive
preventive health guidelines supported by HRSA for infants, children,
and adolescents under section 2713 of the Public Health Service Act.
Non-grandfathered group health plans and health insurance issuers are
required to cover screenings included in these HRSA-supported
comprehensive guidelines without charging a co-payment, co-insurance,
or deductible for plan years beginning on or after the date that is one
year from the Secretary's adoption of the condition for screening.
The Advisory Committee on Heritable Disorders in Newborns and
Children (ACHDNC), now inactive, was tasked with reviewing available
scientific evidence and then making recommendations to the Secretary of
HHS regarding what conditions should be on the RUSP. When a condition
is nominated, ACHDNC determines whether there is sufficient evidence
available for early screening and refers it to ACHDNC's Evidence Review
Group (ERG). The ERG is responsible for identifying and assessing all
available evidence and summarizing for ACHDNC the strength and
effectiveness of the evidence found on the net benefit of screening,
the ability of states to screen for the condition, and the availability
of effective treatments. The ERG completed an evidence review for MLD.
Following the completion of the evidence review for MLD, but prior to
issuing a recommendation to the Secretary on the inclusion of MLD to
the RUSP, the ACHDNC was terminated.
The condition for addition, MLD, is a rare genetic condition that
leads to progressive motor and brain damage. Children with early-onset
forms of the condition who do not receive treatment before symptom
onset experience motor function loss/paralysis and neurological
impairment followed by death at 5-6 years of age. Gene therapy is
extremely effective if started early in life, with those who were
placed on the earliest treatment pilots continuing to live into their
mid-teen years today.
Summary of Public Comments
A Federal Register notice sought public comment on the potential
recommendation of including or not including MLD on the RUSP. HRSA
requested that the respondents consider the ERG's report summary on MLD
and the suitability of state NBS programs screening for MLD within the
newborn period in their response. HRSA considered all public comments
as part of its deliberative process along with review of the completed
MLD evidence review report prior to making a recommendation to the
Secretary of HHS. A total of 98 respondents commented on the inclusion
of MLD on the RUSP. Of these, 96 responses (98 percent) expressed
support to add MLD to the RUSP, 1 response (1 percent) opposed its
addition, and 1 response (1 percent) provided feedback on awaiting
results from pilot studies and early adopting states to inform
implementation. The responses in support of or against adding MLD to
the RUSP are summarized below.
Comments on Adding MLD to the RUSP
The majority of respondents (96 responses or 98 percent) described
benefits of adding MLD to the RUSP including highly accurate screening
for MLD with minimal false positives, if any; an FDA-approved gene
therapy proven to prevent symptoms if administered before onset of
disease; and early diagnosis can save lives of children with MLD.
The respondent that opposed adding MLD to the RUSP cited resource
challenges for implementing MLD screening in public health laboratories
and the high cost of treatment as reasons why MLD should not be added
to the RUSP. However, HRSA notes that adding a condition to the RUSP
does not require states to implement screening immediately. States
determine their
[[Page 59848]]
resource allocations for NBS screening based on their specific state
budget and public health priorities.
After consideration of the evidence review report and public
comments, no changes were made to the recommendation and HRSA
recommended to the Secretary of HHS that MLD be included for addition
to the RUSP.
Acceptance of Recommendation
On December 16, 2025, the HHS Secretary accepted HRSA's
recommendation. The RUSP is updated and can be accessed at the
following link: https://mchb.hrsa.gov/programs/newborn-screening.
Thomas J. Engels,
Administrator.
[FR Doc. 2025-23574 Filed 12-19-25; 8:45 am]
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