The medical foods market has grown to a multi-billion-dollar business and continues to expand in the U.S. and around the world. Globally, the market was valued at $12.3 billion in 2015 and is expected to grow at compound annual rate (CAGR) of 6.9% to 24.4 billion by 2025.

Conditions associated with aging, Alzheimer’s disease, and diabetes, to name a few, are fueling growth. Advances in diagnosis and treatment favor the use of medical foods in conditions such as attention deficit disorder and kidney disease. The shift in patient management from inpatient to outpatient settings also supports the increased use of medical foods.

Category Introduction
Medical foods are not new to the U.S. market. The first product to use the term “medical food” was Lofenalac, a meal replacement product for the dietary management of people with PKU (phenylketonuria) who cannot metabolize the amino acid phenylalanine. In the U.S., Lofenalac and other medical foods were classified and regulated as drugs until passage of the Orphan Drug Act in 1988. The Orphan Drug Act created a category of nutritional therapeutics, separating them from drugs.

The medical food category experienced significant growth in the late 1980s through the 1990s with the launch of products for the dietary management of, for instance, pulmonary and renal diseases. The FDA describes medical foods as specially formulated or processed foods designed for patients’ partial or sole source nutrition as part of an oral diet or by enteral feeding tube. While medical foods do not require FDA approval for commercial use, they must be used under the supervision of a physician 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.

Existing medical foods may look like normal food but have an altered nutrient content, or they may be beverages, bars, pills, or tablets. They often can be categorized into one of the following categories:

• Sole source nutrition products that provide all nutrients except the offending one. Lofenalac, for example, provides all nutrients except for the amino acid phenylalanine.
• Other products may have an altered content of macronutrients (protein, carbohydrate, fat) to compensate for altered organ function.
• Products that can be added to the usual diet to fulfill special nutritional needs.

Lingering Questions & Controversies
Even though regulations exist for certain aspects of medical foods, such as some labeling criteria, several elements remain unclear or controversial. These include:

• Marketplace uncertainty regarding the difference between nutritional supplements and medical foods;
• Confusion about the distinction between structure-function, disease management, and drug claims;
• Absence of clear standards for clinical substantiation requirements of medical foods;
• Difficulty in defining the distinctive nutritional requirements imposed by disease or health conditions necessitating use of medical foods;
• A lack of clarity about what constitutes physician supervision and how medical foods are to be marketed to patients;
• Limited third-party reimbursement for medical foods;
• Lack of an FDA-approval process for medical foods; and
• Most healthcare professionals have little knowledge about the availability of medical foods, their use, and regulations.

These and other issues have made it difficult for companies hoping to develop and commercialize medical foods.

Regulatory Complexity
FDA’s final guidance document and FAQs on medical foods can be downloaded at http://bit.ly/2hYfDHR. Regarding this guidance, FDA clearly states that the documents “do not establish legally enforceable responsibilities. Instead, guidances describe our current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited.”

However, FDA has issued warning letters to several companies based on these guidances. The primary reason letters were issued was because companies have not demonstrated a “distinctive nutritional requirement” for the product.

Clearly, compliance with FDA guidance about medical foods requires expert interpretation. A problem we often observe is that producers assume that if their product contributes to improved clinical outcomes, then it qualifies as a medical food. Common sense says this should be true; unfortunately, it is not.

Without meeting the precise definition of a medical food as dictated by FDA, the agency would likely view such a product as an unapproved drug. Drug claims may not be made for medical foods. FDA requires that companies seeking to market a medical food must develop a persuasive argument to convince the agency and the medical community that:

1. The product is especially formu lated/processed to meet distinctive nutritional requirements imposed by the disease or condition;
2. The product is necessary to provide particular nutrients required by patients to achieve the expected outcome benefit; and
3. The product is distinct from a dietary supplement intended for health maintenance.

The Solution: The Medical Foods Qualification Assessment
Companies often find they do not have the internal expertise to accomplish these stipulations. Windrose Partners has developed a process for screening potential products. Our Medical Food Qualification Assessment is not a comprehensive plan for new products. Rather, it is an initial evaluation of a product or concept that defines the potential for medical food status. If the product does not qualify according to the assessment, a gap analysis identifies items that, upon completion, increase the likelihood that the product can be marketed as a medical food.

After establishing confidentiality, the Windrose Partners team of medical foods experts uses information provided about the product such as the following:

• Product formulation, ingredient specifications, targeted indication, proposed form/application, proposed daily use, directions for use;
• Relevant scientific and clinical research studies demonstrating safety and nutritional action.

The team, made up of clinical, regulatory, business development, toxicology, and manufacturing experts, analyzes the following factors to determine a product’s potential to qualify as a medical food:

• Product indication;
• Product composition and intended use;
• Presence of an established nutritional need which can be surmised for the indication and intended use;
• Mechanism of action of the active ingredient(s);
• Clinical study data supporting product composition and application to the particular condition(s);
• Evidence in scientific and clinical literature supporting product use;
• Ingredients are GRAS and meet FDA safety requirements for medical food use.

Regulatory guidance about medical foods is not definitive, nor does it carry the force of law. While FDA approval is not required prior to product commercialization, it is possible for manufacturers to get warning letters saying that products do not meet FDA guidances. Use of a structured, organized process to qualify potential medical food products can serve as a valuable screening tool for companies that are considering this route to market. 

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Gregory Stephens
Windrose Partners

Greg Stephens, RD, is president of Windrose Partners, a company serving clients in the the dietary supplement, functional food and natural product industries. Formerly vice president of strategic consulting with The Natural Marketing Institute (NMI) and Vice President of Sales and Marketing for Nurture, Inc (OatVantage), he has 25 years of specialized expertise in the nutritional and pharmaceutical industries. His prior experience includes a progressive series of senior management positions with Abbott Nutrition (Ross Products Division of Abbott Laboratories), including development of global nutrition strategies for disease-specific growth platforms and business development for Abbott’s medical foods portfolio. He can be reached at 267-432-2696; E-mail: gregstephens@windrosepartners.com.