Medical Foods – Insights Into FDA’s Interpretation and Enforcement
By Elizabeth Campbell and Allen Sayler
“Medical food” is a commonly misunderstood term, as it is used by the industry for the U.S. Food and Drug Administration (FDA)’s regulated products. Many manufacturers find marketing products as medical foods to be an attractive option, as medical foods are exempt from the disease statement limitations under the Nutrition Labeling and Education Act of 1990 (NLEA) [see 21 U.S.C. 343(r)] for health and structure/function claims, as long as they identify the diseased population for whom they are designed. Medical foods are also exempt from the NLEA labeling requirements for nutrient content claims. As with any food, a medical food that bears a false or misleading claim would be considered misbranded under section 403(a)(1) of the Federal Food, Drug, and Cosmetic Act (FD&C Act).
Medical foods can be described as:
A medical food is intended for people who have a disease or condition that results in a distinctive nutritional need which cannot be met by a diet of regular food, but is met by the medical food. The disease or condition must be identified, the distinctive nutritional need must be substantiated by data, and it must be demonstrated that the product would meet the identified nutritional need.
What Is a Medical Food?
The statutory definition of medical food is:
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, based on recognized scientific principles, are established by medical evaluation.
Medical foods are not those simply recommended by a physician as part of an overall diet to manage the symptoms or reduce the risk of a disease or condition. Likewise, not all foods fed to patients with a disease, including diseases that require dietary management, are medical foods. Instead, medical foods are foods that are specially formulated and processed (as opposed to a naturally occurring foodstuff used in a natural state) for a patient who requires use of the product as a major component of a disease or condition’s specific dietary management.
While some diseases benefit from the consumption of certain types of foods, if those foods are available as part of a healthy diet and the nutrients within those foods are able to be absorbed by the patient, then the foods would not be considered medical foods. Two examples of diseases which typically do not require a medical food are scurvy and pellagra. While these diseases are the result of essential nutrient deficiencies (e.g., deficiencies of vitamin C and niacin, respectively), those deficiencies are primarily caused by inadequate intake (e.g., famine, significant calorie restriction, eating disorders, alcoholism, diet practices/fad diets). In these two cases, deficiencies, excluding any permanent physical damage, can typically be corrected once foods with these essential nutrients (or dietary supplements, if necessary) are consumed. Because such diseases can typically be managed through consumption of a healthy, well -balanced diet, FDA generally would not consider a product labeled and marketed for these diseases to meet the statutory and regulatory criteria for a medical food [see 21 CFR 101.9(j)(8)(ii)].
Think of medical foods as classified into the following categories:
• Nutritionally complete formulas
• Nutritionally incomplete formulas, including individual “modular” type products that may be mixed with other products before use (e.g., protein, carbohydrate or fat modulars)
• Formulas for metabolic (genetic) disorders in patients over 12 months of age
• Oral rehydration products
The following criteria that clarify the statutory definition of a medical food can be found in FDA’s regulations at 21 CFR 101.9(j)(8). A medical food is exempt from the nutrition labeling requirements of 21 CFR 101.9 only if:
• It is a specially formulated and processed product (as opposed to a naturally occurring foodstuff used in its natural state) for the partial or exclusive feeding of a patient by means of oral intake or enteral feeding by tube, meaning a tube or catheter that delivers nutrients beyond the oral cavity directly into the stomach or small intestine
• It is intended for the dietary management of a patient who, because of therapeutic or chronic medical needs, has limited or impaired capacity to ingest, digest, absorb or metabolize ordinary foodstuffs or certain nutrients, or who has other special medically determined nutrient requirements, the dietary management of which cannot be achieved by the modification of the normal diet alone
• It provides nutritional support specifically modified for the management of the unique nutrient needs that result from the specific disease or condition, as determined by medical evaluation
• It is intended to be used under medical supervision
• It is intended only for a patient receiving active and ongoing medical supervision wherein the patient requires medical care on a recurring basis for, among other things, instructions on the use of the medical food
Regulatory History of Medical Foods
Prior to 1972, medical foods were primarily formulas for managing patients with inherited metabolic diseases and were mainly “orphan” products for limited populations and considered to be drugs to ensure their use was under medical supervision. However, in 1972 the FDA reclassified these products from drugs to foods for special dietary use to enhance their development and availability. In the intervening years, a wide variety of products categorized as “medical foods” have been developed. Currently marketed medical foods with a wide variety of claims are used extensively as a life support modality in the management of the critically ill and elderly. They are required to conform only to those regulations dealing with general food safety and labeling to be distributed in the United States.
The 1988 Orphan Drug Act [21 U.S.C. 360ee(b)(3)] provides financial incentives (such as drug application fee exemptions and market exclusivity) to promote development of “orphan drugs” for treating rare diseases and conditions. Medical foods were included in the Orphan Drug Act to include dietary therapies, along with pharmaceuticals, as eligible for financial incentives for developing orphan drugs. Medical food was defined within the context of new drug development, not for regulating the use of such products. The Orphan Drug Act did not amend the FD&C Act to add medical foods as a regulatory category.
FDA published in 1996 an Advanced Notice of Proposed Rulemaking (ANPR) in which the agency discussed its interpretation of the Orphan Drug Act definition of medical food. While FDA later withdrew this ANPR due to lack of resources to devote to the subject of medical foods, the agency stated that it still retained the positions described in the document. A subsequent guidance document on medical foods provided advice consistent with positions in the ANPR. The Compliance Programs The Guidance for Industry: Frequently asked Questions about Medical Foods (second edition) and Medical Foods Program – Import and Domestic offer a basis for understanding what approach companies should take when considering if their product meets a medical food definition as well as the types of enforcement FDA will take on this class of food. In addition, manufacturers of medical foods must comply with all applicable FDA requirements for foods, including the following:
• Current good manufacturing practice (21 CFR part 110)
• Registration of food facilities (21 CFR part 1 subpart H)
• Thermally processed low-acid foods packaged in hermetically sealed containers (21 CFR part 113)
• Acidified foods (21 CFR part 114)
• Emergency permit control (21 CFR part 108)
Labeling of a Medical Food
Medical foods must comply with all applicable food labeling requirements except for those specific requirements from which medical foods are exempt. The requirements concerning the misbranding of food (21 CFR 101.18) apply to medical foods. Specifically, the labeling of medical foods must contain:
• A statement of identity (21 CFR 101.3)
• An accurate statement of the net quantity of contents (21 CFR 101.105)
• The name and place of business of the manufacturer, packer, or distributor (21 CFR 101.5)
• A complete list of ingredients, listed by their common or usual name and in descending order of predominance (21 CFR 101.4)
Medical food labels must also conform to the principal display panel requirements under 21 CFR 101.1 and the applicable information panel requirements under 21 CFR 101.2.
Medical Food Ingredient Requirements:
An ingredient that is added to a medical food must be demonstrated to be safe and in compliance with all applicable provisions of the FD&C Act and FDA regulations. Any ingredient added to a medical food should be:
1. A food additive used in accordance with FDA’s food additive regulations (see 21 CFR part 172)
2. A color additive used in accordance with the color additive regulations (see 21 CFR parts 73 and 74)
3. A substance that is generally recognized, by qualified experts, to be safe under the conditions of its intended use (generally recognized as safe) [see 21 CFR 170.30 and 21 U.S.C. 321(s)]
4. A substance that is authorized by a prior sanction [see 21 CFR 170.3(l), 21 U.S.C. 321(s)(4)]
In conclusion, FDA considers the statutory definition of medical foods to be narrow, constraining the types of products that fit within the category. FDA has considered most products marketed as medical foods not to meet the definition of a medical food. The types of products that have been the target of FDA medical food warning letters include products intended for the dietary management of allergies and asthma, lactose intolerance, psoriasis, constipation, Irritable Bowel Syndrome, Crohn’s disease, colitis, arthritis, failure-to-thrive, injury/trauma, pre/post-surgery and prenatal vitamins. As a matter of fact, the FDA guidance document specifically states that pregnancy and diabetes are not diseases or conditions for which there is a distinctive nutritional need that could only be met by a medical food.
Those patients for whom medical foods are necessary receive in some cases life-saving, or at least life-enhancing benefits from the absorption of the nutrients within. Unfortunately, through industry misunderstanding, some foods that are marketed as medical foods really are not. A thorough review of the requirements and guidance documents should help manufacturers to understand if their products meet FDA’s narrow definition. Companies requiring additional assistance or clarification can reach out to FDA directly or consult with an expert in this area.
Elizabeth Campbell is an independent advisor for labeling and claims and Allen Sayler is senior director for food & cosmetic consulting services, both with the EAS Consulting Group.