Policy Statement: Coverage for Medical Foods

18 April, 2016


In support of patients and families in our clinics and of the public health system that serves our communities, the members of the SIMD strongly urge that all private and public systems for health care payment be mandated to cover specialized diets, including medical foods, for treatment of inborn errors of metabolism found by newborn screening or clinically diagnosed. This statement reiterates our concerns presented in 2007. Little or no progress has occurred in that interval.

Inborn errors of metabolism result from genetic alterations in biochemical pathways that, if left untreated, can lead to brain damage, physical disability and death. Some of these conditions, for example, phenylalanine hydroxylase (PAH) deficiency (also known as phenylketonuria or PKU), are treated with specialized diets using medical foods, and in some cases specific amino acids or cofactors, to normalize biochemistry and improve health and developmental outcomes. In fact, the success of newborn screening to prevent cognitive impairment from PAH deficiency was entirely founded on the use of medically prescribed diets using medical foods.

The 1988 Orphan Drug Act defined "Medical Food" as "a food which is formulated to be consumed or administered enterally under the supervision of a physician and which is intended for the specific dietary management of a disease or condition for which distinctive nutritional requirements on the basis of recognized scientific principles are established by medical evaluation."

Unlike ordinary nutritional products marketed to the general population, such as those designed to supplement a normal diet, and unlike a variety of foods for special dietary use, medical foods are an essential, medically necessary treatment for many inherited metabolic disorders, including PAH deficiency. However, many health care payers deny coverage for medical foods, citing provisions in their local or national policies that exclude coverage for "nutritional supplements" or foods. Many state legislatures have required that private insurance companies cover medical foods; however, these mandates are not consistent across states, they vary as to the disorders and ages covered, and they do not apply to individuals covered by Federal insurance or the Employee Retirement Income Security Act (ERISA). Thus, the complex pattern of health care coverage in the United States means that many individuals with inborn errors of metabolism are at significant risk of disability or death because of lack of access to the medical foods that are a critical part of their medical care.

While the Affordable Care Act mandates coverage of newborn screening without co-pay to families, it is silent on coverage of the medical food treatments for individuals identified with inborn errors of metabolism. The lack of uniform and consistent coverage of medical foods throughout the United States threatens individuals and families. Because medical foods are essential treatments for many of the conditions detected by expanded newborn screening, failure to provide lifelong access to these treatment modalities also threatens the success of public health policy.