Notice2024-04618
Request for Information: Nomination and Evidence-Based Review Process of the Advisory Committee on Heritable Disorders in Newborns and Children
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Published
March 5, 2024
Issuing agencies
Health and Human Services DepartmentHealth Resources and Services Administration
Abstract
At the request of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC or Committee), HRSA is requesting input from the public on the process used by the Committee for nomination and evidence-based review of conditions that are considered for inclusion in the Recommended Uniform Screening Panel (RUSP). As an entity that advises the Secretary of Health and Human Services (Secretary) based on evidence-based information, ACHDNC periodically considers and evaluates its processes. During the November 2023 meeting, ACHDNC hosted listening sessions to learn more from stakeholders regarding their views on the process used by ACHDNC for nomination and evidence-based review of conditions. In support of this work, HRSA is seeking public input on a series of questions that will help inform the nomination and review processes.
Full Text
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<title>Federal Register, Volume 89 Issue 44 (Tuesday, March 5, 2024)</title>
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[Federal Register Volume 89, Number 44 (Tuesday, March 5, 2024)]
[Notices]
[Pages 15876-15878]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-04618]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Health Resources and Services Administration
Request for Information: Nomination and Evidence-Based Review
Process of the Advisory Committee on Heritable Disorders in Newborns
and Children
AGENCY: Health Resources and Services Administration (HRSA), Department
of Health and Human Services.
ACTION: Notice of request for public comment.
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SUMMARY: At the request of the Advisory Committee on Heritable
Disorders in Newborns and Children (ACHDNC or
[[Page 15877]]
Committee), HRSA is requesting input from the public on the process
used by the Committee for nomination and evidence-based review of
conditions that are considered for inclusion in the Recommended Uniform
Screening Panel (RUSP). As an entity that advises the Secretary of
Health and Human Services (Secretary) based on evidence-based
information, ACHDNC periodically considers and evaluates its processes.
During the November 2023 meeting, ACHDNC hosted listening sessions to
learn more from stakeholders regarding their views on the process used
by ACHDNC for nomination and evidence-based review of conditions. In
support of this work, HRSA is seeking public input on a series of
questions that will help inform the nomination and review processes.
DATES: Comments on this FRN should be received no later than April 4,
2024.
ADDRESSES: Responses must be submitted electronically as email
attachments to CDR Leticia Manning, MPH, ACHDNC's Designated Federal
Officer, at: <a href="/cdn-cgi/l/email-protection#aeefede6eae0edeec6dcddcf80c9c1d8"><span class="__cf_email__" data-cfemail="78393b303c363b38100a0b19561f170e">[email protected]</span></a>.
FOR FURTHER INFORMATION CONTACT: CDR Leticia Manning, MPH, Designated
Federal Officer, Maternal and Child Health Bureau, HRSA, 5600 Fishers
Lane, Rockville, Maryland 20857; 301-443-8335; or <a href="/cdn-cgi/l/email-protection#b4f5f7fcf0faf7f4dcc6c7d59ad3dbc2"><span class="__cf_email__" data-cfemail="0b4a48434f45484b6379786a256c647d">[email protected]</span></a>. A
copy of the ACHDNC charter may be obtained by accessing the ACHDNC
website at: <a href="https://www.hrsa.gov/advisory-committees/heritable-disorders">https://www.hrsa.gov/advisory-committees/heritable-disorders</a>.
SUPPLEMENTARY INFORMATION: ACHDNC was established in 2003 and provides
advice and recommendations to the Secretary on the development of
newborn screening activities, technologies, policies, guidelines, and
programs for effectively reducing morbidity and mortality in newborns
and children having, or at risk for, heritable disorders. ACHDNC
reviews and reports regularly on newborn and childhood screening
practices, recommends improvements in the national newborn and
childhood screening programs, and fulfills requirements stated in the
authorizing legislation. In addition, ACHDNC's recommendations
regarding inclusion of additional conditions for screening on the RUSP,
following adoption by the Secretary, are evidence-informed preventive
health services provided for in comprehensive guidelines supported by
HRSA pursuant to section 2713 of the Public Health Service Act (42
U.S.C. 300gg-13), for which certain health insurance plans and issuers
are required to provide coverage without cost-sharing. The ACHDNC meets
four times each calendar year or at the discretion of the Designated
Federal Officer in consultation with the Chair.
Responses
HRSA is seeking responses on the following questions. Responses to
all questions are voluntary, and a response to each question is not
required.
Nomination Process: The current nomination process can be found
here: <a href="https://www.hrsa.gov/advisory-committees/heritable-disorders/condition-nomination">https://www.hrsa.gov/advisory-committees/heritable-disorders/condition-nomination</a>. The Committee has already received feedback from
newborn screening stakeholders on the current nomination process, and
based on this feedback, the Committee is requesting that HRSA publish
this notice to obtain additional public feedback on the proposed
revisions to the questions addressed within the nomination package.
Please provide feedback in response to the questions on the
proposed elements below (i.e., the condition, newborn screening, and
benefits and harms of newborn screening), including:
(1) Whether these questions add clarity to what is required for a
condition nomination package?
(2) Whether appropriate language is used to describe the required
information for each section?
(3) Whether this question-based format makes clearer the
requirements for a nomination? If not, please propose edits and/or
changes to what is provided.
Please cite any available information that you may have to support
your responses.
Section I: The Condition
(1) What is the specific condition to be screened for (``target
condition'') and how is it defined?
(2) How is the condition diagnosed as part of usual clinical care?
Why is the current clinical diagnostic approach inadequate?
(3) What is the reported birth prevalence of the condition in the
United States (or comparable newborn population)? Is the condition more
common in certain populations?
(4) Describe the severity of the condition when detected as part of
usual clinical care.
Section II: Newborn Screening
(1) What testing approach(es) are you suggesting for newborn
screening? Please be specific regarding the approach to screening
(e.g., dried-blood spot, point-of-care screening, what specimen or
test). Is there one or more tiers of testing that should occur before a
diagnostic referral to a clinical specialist?
(2) How is the condition diagnosed after an at-risk child is
identified through newborn screening? (i.e., How does a clinical
specialist confirm that an infant has the condition after referral from
the newborn screening program?)
(3) What other conditions could be identified through newborn
screening for the target condition as nominated? This includes
phenotypes of the target condition that are not being nominated for
newborn screening (e.g., late-onset, mild variants). Will screening for
the target condition identify carriers?
(4) What examples are there of screening and diagnosis for the
condition at a prospective population level (e.g., through state
newborn screening (NBS) program or pilot studies)? Has at least one
case of the condition been identified, diagnosed, and treated through a
prospective population-based approach?
(5) Based on at least one example of a prospective population level
study from question #4, please describe the epidemiologic elements a-e
below. (Include a peer-reviewed study, if available.):
(a) The birth prevalence of the target condition.
(b) The birth prevalence of the other conditions that could be
identified by screening.
(c) The percentage of newborns with the target condition who had a
positive screen (sensitivity of NBS test).
(d) The percentage of newborns with one of the other conditions who
were identified through newborn screening with the target condition.
(e) The percentage of newborns without the target condition who had
a negative screen (specificity of NBS test).
Section III: Benefits and Harms of Newborn Screening
(1) What is the expected benefit to infants and/or families for
detection of the condition through newborn screening compared to
clinical care identification?
(2) What is the expected harm to infants and/or families for
detection of the condition through newborn screening compared to
clinical care identification?
(3) Are there other benefits or harms that may result from
implementing a state newborn screening program? (e.g., false positive
or negative results, infants identified with other conditions, or
opportunity costs to a state public health system)
(4) What treatment and management protocols are available for
newborns identified with the condition through newborn screening?
[[Page 15878]]
(5) What plan for longitudinal follow-up of newborns identified
through newborn screening is available? For example, will there be a
patient registry available for use by clinical providers or by
individuals/families? For how many years would infants with the
condition be followed?
Evidence-based Review Process: The current criteria for ACHDNC to
recommend inclusion of a condition on the RUSP to the Secretary is
based primarily on peer-reviewed evidence regarding the certainty that
benefits of universal screening outweigh harms (``net benefit''). These
criteria have been largely applied to focus on the benefits and harms
to the individual child, with much less consideration of benefits and
harms to the family, states, or to the public health system. Financial
and opportunity costs have received less attention by ACHDNC, in part
because of the lack of published evidence regarding such topics.
Below is an example of what published evidence should be considered
by the Committee when conducting a condition evidence review. The
Committee requests feedback regarding the example below.
When weighing certainty and net benefit of screening for a
condition, the Committee should consider the full range of relevant,
published, peer-reviewed evidence. Although such evidence in relation
to benefits and harms to the individual child remain paramount, the
Committee should also consider benefits and harms to the family and to
society at large, including disproportionate impacts or disparities
related to specific conditions or screening. For example, the Committee
could consider evidence demonstrating benefits for the family regarding
future planning (e.g., finances, geographic proximity to services, home
design, etc.), earlier access to early intervention programs, or
opportunity costs to the public health system. Ideally, potential harms
and benefits should be supported by evidence directly relevant to the
condition under review. When such evidence is lacking, Committee
members could consider peer-reviewed evidence from other disorders to
the extent that such evidence is considered potentially relevant to the
condition under consideration.
Special Note to Commenters
The information obtained through this request for information (RFI)
may help inform ACHDNC processes. Per the ACHDNC Charter, the Committee
has the responsibility to decide the processes for nomination, evidence
review, and making recommendations regarding the RUSP. How Committee
members ultimately vote on recommending a condition for inclusion on
the RUSP will continue to reflect their judgment on the certainty of
net benefit to the entire population of infants born in the United
States.
This RFI is issued solely for information and planning purposes; it
does not constitute a Request for Proposal, applications, proposal
abstracts, or quotations. This RFI does not commit the U.S. government
to contract for any supplies or services or make a grant or cooperative
agreement award. Further, HRSA is not seeking proposals through this
RFI and will not accept unsolicited proposals. HRSA will not respond to
questions about the policy issues raised in this RFI. Responders are
advised that the U.S. government will not pay for any information or
administrative costs incurred in response to this RFI; all costs
associated with responding to this RFI will be solely at the interested
party's expense.
Authority: ACHDNC is authorized by section 1111(g) of the Public
Health Service Act, 42 U.S.C. 300b-10(g), and the Federal Advisory
Committee Act, 5 U.S.C. chapter 10.
Maria G. Button,
Director, Executive Secretariat.
[FR Doc. 2024-04618 Filed 3-4-24; 8:45 am]
BILLING CODE 4165-15-P
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