New FDA Draft Guidance for Medical Foods Suggests that Some Common Products and Certain Label Statements Violate FDA Regulations

By Etan Yeshua –

Earlier this week, FDA provided new insight into its interpretation of the definition and regulation of medical foods — a legal category established by the Orphan Drug Act of 1988, incorporated into the Federal Food, Drug, and Cosmetic Act in 1990, and seemingly narrowed in scope by subsequent FDA regulations.  The category – which exempts certain “medically necessary” foods from regulations that govern health claims, nutrient content claims, and other labeling requirements – was the subject of FDA’s 2007 Guidance for Industry: Frequently Asked Questions About Medical Foods.  That document described the statutory and regulatory definitions of “medical food” and discussed general labeling requirements and exemptions.  On Tuesday, FDA updated the 2007 guidance document with specific examples of diseases or conditions for which a medical food may or may not (in FDA’s view) be marketed, as well as certain labeling statements that would render a medical food misbranded. 

Briefly, the statutory hallmarks of a medical food are that it must be formulated for oral or enteral use under physician supervision “for the special dietary management of a disease or condition for which distinctive nutritional requirements, based on recognized scientific principles, are established by medical evaluation.”  Orphan Drug Act, 21 U.S.C. § 360ee(b)(3).  By FDA’s regulation, a medical food must be “specially formulated” (rather than naturally occurring), and the patient’s distinctive nutritional requirements must be impossible to meet “by the modification of the normal diet alone.”  21 C.F.R. § 101.9(j)(8).

Tuesday’s draft guidance document distinguishes between conditions – such as certain inborn errors of metabolism, or IEMs – that FDA considers to be appropriate for medical foods, and others – such as diabetes, pregnancy, and classic nutrient deficiency diseases, like scurvy – that FDA does not.  Although the agency acknowledged that diabetes and pregnancy are associated with certain nutritional requirements (i.e., controlling carbohydrate consumption and increasing fiber intake for diabetics, and special nutrient recommendations for pregnant women), a product intended to address those requirements (and, therefore, those conditions) should not be marketed as a medical food.  According to FDA, diabetics can achieve proper carbohydrate and fiber levels, and pregnant women can meet their special nutrient requirements, through “modification of the normal diet alone.”  Conversely, FDA asserts, certain IEMs – such as those that “require significant restriction of particular amino acids and/or total protein… or significant modification of fatty acids/total fats” – can only be managed by “modifying the normal diet” in a way that deprives patients of “adequate levels of essential nutrients.”  To achieve adequate levels of these essential nutrients in patients with these types of IEMs, a “medical food is required in addition to a specific dietary modification.”

FDA also opined on the use of certain labeling statements that, in the agency’s opinion, would render a medical food misbranded.  First, FDA said that “labeling of medical foods should not include National Drug Code (NDC) numbers.”  Because the NDC number –  a “unique, three-segment number… which is a universal product identifier for human drugs” –  is intended “for uniquely identifying drugs,” the “presence of an NDC number on a product that is not a drug may be a false or misleading representation that misbrands the product.” 

Second, FDA said that the “labeling of medical foods may not bear the symbol ‘Rx only.’”  FDA previously said (in connection with dietary supplement labeling) that “the use of the word ‘prescription’ or its abbreviation ‘Rx’… should not automatically be interpreted as a disease claim,” 65 Fed. Reg. 1000, 1022 (Jan. 6, 2000).  However, Tuesday’s medical food Draft Guidance interpreted the statute’s prohibition on using  the “Rx only” term on non-prescription drugs to apply not only to drugs, but to non-drug products as well.  The 1997 statute that required “Rx only” to appear on all prescription drugs, FDA argues, sought to replace the previously-required caution statement – “Caution: Federal law prohibits dispensing without prescription” – with the “Rx only” symbol.  Therefore, the “Rx only” symbol is meant to “communicate the same message to consumers that the longer caution statement communicated,” and could thus mislead consumers by suggesting that the medical food bearing the symbol is prohibited by federal law from being dispensed without a prescription.  Nevertheless, because medical foods are statutorily defined as intended for use under physician supervision, FDA said that it would not object to statements that convey this message to consumers (e.g., “must be used under the supervision of a physician”).

The updated Draft Guidance also reiterates that medical foods are not considered to be, or regulated as, drugs; that they must comply with cGMPs for foods, allergen labeling requirements, certain registration requirements, and FDA’s compliance program guidance manual; that any ingredient included in a medical food must be Generally Recognized as Safe, a prior sanctioned substance, an approved food additive, or a color additive; and that a medical food must be a “specially formulated and processed product,” as opposed to a conventional food (e.g., fruits, vegetables, fats, sugars, etc.) that – in its natural state – happens to provide high (or low) amounts of a required nutrient.

Comments to the Draft Guidance must be submitted by October 11, 2013.