Medical foods for inborn errors of metabolism: History, current status, and critical need

on behalf of the Follow-up and Treatment (FUTR) Workgroup for the Advisory Committee on Heritable Disorders in Newborns and Children

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18 Scopus citations


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.

Original languageEnglish (US)
Article numbere20192261
Issue number3
StatePublished - Mar 1 2020

Bibliographical note

Funding Information:
Members of the Follow-up and Treatment (FUTR) Workgroup for the ACHDNC (in alphabetical order) are as follows: Jeffrey P. Brosco (University of Miami; Chair, FUTR Workgroup), Amy Brower (American College of Medical Genetics and Genomics), Debra Freedenberg (Texas Department of State Health Services), Kathryn Hassell (University of Colorado Denver), Christopher A. Kus (Association of State and Territorial Health Officials; Co-Chair, FUTR Workgroup), Nancy Doan Leslie (Cincinnati Children?s Hospital Medical Center), Sylvia Mann (Hawaii State Department of Health), Kamila B. Mistry (Agency for Healthcare Research and Quality Office of Extramural Research Education and Priority Populations), Jana Monaco (Organic Acidemia Association), Robert J. Ostrander (State University of New York Upstate Medical University), Margie A. Ream (Nationwide Children?s Hospital), Annamarie Saarinen (Newborn Foundation), Joseph H. Schneider (University of Texas Southwestern), Janet Thomas (Children?s Hospital Colorado), and Alan E. Zuckerman (Georgetown University). This article was written for the committee report entitled ?Medical Foods for Inborn Errors of Metabolism: The Critical Need to Improve Patient Access.?

Publisher Copyright:
Copyright © 2020 by the American Academy of Pediatrics


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