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Discover Pediatric Collections on COVID-19 and Racism and Its Effects on Pediatric Health

American Academy of Pediatrics
Special Article

Medical Foods for Inborn Errors of Metabolism: History, Current Status, and Critical Need

Susan A. Berry, Christine S. Brown, Carol Greene, Kathryn M. Camp, Stephen McDonough, Joseph A. Bocchini and on behalf of the Follow-up and Treatment (FUTR) Workgroup for the Advisory Committee on Heritable Disorders in Newborns and Children
Pediatrics March 2020, 145 (3) e20192261; DOI: https://doi.org/10.1542/peds.2019-2261
Susan A. Berry
aDepartment of Pediatrics, University of Minnesota, Minneapolis, Minnesota;
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Christine S. Brown
bNational Phenylketonuria Alliance Inc, Eau Claire, Wisconsin;
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Carol Greene
cDepartment of Pediatrics, University of Maryland, Baltimore, Maryland;
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Kathryn M. Camp
dOffice of Dietary Supplements, National Institutes of Health, Silver Springs, Maryland;
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Stephen McDonough
eFollow-up and Treatment Workgroup,
fIndependent Doctors, Bismarck, North Dakota; and
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Joseph A. Bocchini Jr
gAdvisory Committee on Heritable Disorders in Newborns and Children, Health Resources and Services Administration, Rockville, Maryland
hDepartment of Pediatrics, Louisiana State University, Shreveport, Louisiana
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Abstract

Successful intervention for inborn errors of metabolism (IEMs) is a triumph of modern medicine. For many of these conditions, medical foods are the cornerstone of therapy and the only effective interventions preventing disability or death. Medical foods are designed for patients with limited or impaired capacity to ingest, digest, absorb, or metabolize ordinary foods or nutrients, whereby dietary management cannot be achieved by modification of the normal diet alone. In the United States today, access to medical foods is not ensured for many individuals who are affected despite their proven efficacy in the treatment of IEMs, their universal use as the mainstay of IEM management, the endorsement of their use by professional medical organizations, and the obvious desire of families for effective care. Medical foods are not sufficiently covered by many health insurance plans in the United States and, without insurance coverage, many families cannot afford their high cost. In this review, we outline the history of medical foods, define their medical necessity, discuss the barriers to access and reimbursement resulting from the regulatory status of medical foods, and summarize previous efforts to improve access. The Advisory Committee on Heritable Disorders in Newborns and Children asserts that it is time to provide stable and affordable access to the effective management required for optimal outcomes through the life span of patients affected with IEMs. Medical foods as defined by the US Food and Drug Administration should be covered as required medical benefits for persons of all ages diagnosed with an IEM.

  • Accepted November 27, 2019.
  • Copyright © 2020 by the American Academy of Pediatrics

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Pediatrics
Vol. 145, Issue 3
1 Mar 2020
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Medical Foods for Inborn Errors of Metabolism: History, Current Status, and Critical Need
Susan A. Berry, Christine S. Brown, Carol Greene, Kathryn M. Camp, Stephen McDonough, Joseph A. Bocchini, on behalf of the Follow-up and Treatment (FUTR) Workgroup for the Advisory Committee on Heritable Disorders in Newborns and Children
Pediatrics Mar 2020, 145 (3) e20192261; DOI: 10.1542/peds.2019-2261

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Medical Foods for Inborn Errors of Metabolism: History, Current Status, and Critical Need
Susan A. Berry, Christine S. Brown, Carol Greene, Kathryn M. Camp, Stephen McDonough, Joseph A. Bocchini, on behalf of the Follow-up and Treatment (FUTR) Workgroup for the Advisory Committee on Heritable Disorders in Newborns and Children
Pediatrics Mar 2020, 145 (3) e20192261; DOI: 10.1542/peds.2019-2261
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  • Article
    • Abstract
    • Medical Foods Are Essential Management Modalities for IEMs, Including Many That Are Identified by Newborn Screening
    • What Are Today’s Medical Foods?
    • How Do the Statutes Currently Define Medical Foods, and How Are They Regulated?
    • How Do Patients Access Medical Foods, and Who Pays for Them?
    • What Is the Cost to Families?
    • History of Remediating Efforts
    • Conclusions
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