Medical Nutritional Therapy: Achieving Reimbursement Across the Globe for Unique Non-drug Therapy Options
What Is Medical Nutritional Therapy?
Medical nutritional therapy (MNT), also known as medical foods or foods for specific medical purposes (FSMP), is a distinct product category regulated in the United States (US) by the Food and Drug Administration (FDA) and by the European Commission (EC) in the European Union (EU). MNT is distinguished from the broader category of foods for dietary use, which are intended for a modified normal diet to manage the symptoms or reduce the risk of a disease or condition (Figure 1). Common examples of foods for dietary use include meal replacement for weight control, such as SlimFast® products, or gluten-free foods. In contrast, MNT is intended to meet specific nutritional requirements of a disease or condition that cannot be met by normal foods alone and should be used only under medical supervision. MNT can serve as the sole source of nourishment for prolonged periods, with balanced nutritional composition of macro- and micro-nutrients that reflect dietary recommendations. MNT is distinctively different from dietary supplements and nutraceuticals, which are not intended for use as a conventional food or as the sole item of a meal or diet but rather are intended to enhance wellness among healthy adults, combat diseases, and provide other health benefits.
MNT is intended for patients who have limited or impaired capacity to ingest, digest, absorb, or metabolize ordinary foodstuffs or certain nutrients, or have dietary nutrient requirements that cannot be achieved by modification of normal diet alone. Patients who require MNT range from those with short-term post-surgery requirements, such as post-cholecystectomy, to those living with chronic illnesses, such as cancer, cystic fibrosis, end-stage renal disease, or chronic pulmonary disease, as well as some psychiatric and neurological conditions.
MNT products are also used in infants and children who have special nutritional requirements resulting from inherited biochemical disorders, where specific enzyme defects interfere with normal metabolism of protein, fat, or carbohydrates. These inborn errors of metabolism (IEM) may be associated with increased nutritional requirements, increased metabolic demands, decreased nutrient intakes, limitations of digestion and absorption, or increased nutrient losses. An optimal state of nutrition is critically important in these children since poor nutrition is associated with increased risk of infections, inadequate growth, and other severe health consequences, such as severe mental retardation and death. MNT provides necessary, often lifelong dietary modifications to prevent complications resulting from inadequate nutrition in children with IEM. For example, phenylketonuria (PKU) is an IEM that can result in growth failure, poor skin pigmentation, microcephaly, seizures, and global developmental delay, as well as severe intellectual impairment, if newborns are left on a regular diet. However, patients receiving ongoing MNT are able to maintain adequate levels of essential nutrients and can expect to live relatively normal lives without severe health complications.
Regulatory Approval Pathways for MNT in the US and the EU
Since MNT is not intended to prevent or treat disease states, it is not subject to the same regulatory requirements as prescription or over-the-counter (OTC) medications in the US or EU. As a result, MNT does not require the same type or level of clinical evidence necessary for the approval of prescription or OTC medications and is instead subject to food-like safety standards. Conducting phase I studies is not standard for MNT approval, since these trials focus on safety results that are often not necessary for approval of food products. Given the relatively small size of the targeted patient populations, existing randomized controlled clinical trials of MNTs tend to be phase IIa studies to evaluate product efficacy. Well-controlled phase IIb or large, phase III trials are not typically conducted for MNT to generate strong evidence for product efficacy. Manufacturers also have the option of completing open-label, case-control studies, case series, or comparator trials with drug therapies to demonstrate efficacy. These types of clinical evidence are generally far less expensive than conducting rigorous clinical trials that pharmaceutical products require for approval. The absence of stringent regulatory requirements for MNT approval has led to lack of robust scientific evidence supporting the need for MNT, resulting in lack of reimbursement coverage from payer stakeholders that then translates to large out-of-pocket costs for patients as well as increased societal costs.
Reimbursement Hurdles for MNT in the US and the EU
Despite the apparent need for MNT, there remains a lack of coverage and reimbursement from payers in the US and health authorities in the EU (Table 1). In the US, no national or uniform policy addresses coverage of MNT. Currently, a patchwork of coverage exists for patients requiring MNT, ranging from selective coverage of MNT only for specific disorders or specific MNT foods, termination of coverage after a certain age, no treatment coverage during pregnancy, and caps on amount covered per year. This results in gaps in reimbursement that lead to inadequate financial support for patients who require MNT. For example, despite a federal mandate to screen all newborns for early detection of metabolic disorders, only 16 states require the necessary MNT coverage by third-party payer reimbursement for management of inherited metabolic disorders. In addition, since MNT is not regulated as prescription medications, inconsistencies in reimbursement exist in both public and commercial healthcare plans. On the public payer side, only 38 state Medicaid programs provide MNT coverage, and many stop coverage at age 18. Medicare Part D also does not allow reimbursement for non-FDA-approved therapies, including MNT. This leaves patients who require lifelong MNT, or the addition of MNT later in life as adults, without coverage. Within commercial plans, coverage, when available, is subject to the limitations of each plan, including copayments, deductibles, and cost-sharing policies that apply to OTC products. As a result, patients are often burdened with significant out-of-pocket payments. For example, average out-of-pocket costs of MNT with protein can range from $9,551 to $11,201 annually.
Within Europe, both the European Food Safety Authority (EFSA) and separate government authorities within individual countries oversee MNT regulation. In European countries, MNT is more commonly called foods for special purposes (FSMP). This leads to substantial inconsistencies in coverage and reimbursement of MNT among EU members and the United Kingdom (UK). For example, in France, innovative nutritional therapies undergo formal evaluation through the medical device reimbursement process for coverage decisions. In the UK, FSMP is only reimbursed by the National Health Service (NHS) after product applications, which include a proposed product price, are approved by the Advisory Committee on Borderline Substances (ACBS). In contrast, Germany has specific reimbursement categories for FSMP products that are determined by the biochemical composition of a product and aimed at access for patients who are unable to have nutritional needs sufficiently met through normal food intake. In addition to this heterogeneity in reimbursement pathways within EU countries, there is also variance in market approval regulations within each country. FSMP manufacturers can place a product on the market after national authorities for a country are notified, given that the FSMP product complies with all EC regulations. This notification system allows efficient monitoring of FSMP in each country but results in discrepancies between classifications of a product. For example, the same FSMP may be classified as a food for special medical purposes in one country while qualifying as a food supplement in another. To address this inconsistency, the EC recently issued a new legal provision that requires the EFSA to evaluate information regarding the composition and proposed use of a product to ensure appropriate classification of an FSMP. This legal provision also requires the EC to adopt specific compositional and labeling rules for FSMP. This new provision will take effect February 2019 and will change labeling requirements for FSMP to ensure consistency with the EU on the provision of food information to consumers, prohibit nutritional and health claims for FSMP to avoid inappropriate promotion of products, and extend rules for infant formula labeling, presentation, advertising, marketing, and pesticide use to FSMP intended for infant use.