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A Gray Area for Reimbursement: Medical Foods for Non–Inborn Errors of Metabolism
Leslie Wilson, PhD; Tracy Kuo Lin, MSc, PhD; Anna Oh, RN, MPH; and Vicky Cao, PharmD
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Kelly Fust, MS; Anju Parthan, PhD; Xiaoyan Li, PhD; Anjali Sharma, MD; Xinke Zhang, MS; Marco Campioni, PhD; Junji Lin, PhD, MS; Xuena Wang, PhD; Richard Zur, PhD; Karynsa Cetin, MPH; Melissa Eisen, MD; and David Chandler, PhD
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Jason R. Goldsmith, MD, PhD; Akbar K. Waljee, MD; Tom George, PharmD; Alexandra Brown, BS; Hae Mi Choe, PharmD; Mohamed Noureldin, MBBS; Steven J. Bernstein, MD, MPH; and Peter D.R. Higgins, MD, PhD
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Zhou Zhou, MS; Zheng-Yi Zhou, PhD; Sneha S. Kellar, MPH; Vanja Sikirica, PharmD, MPH; Jipan Xie, MD, PhD; and Regina Grebla, PhD
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A Gray Area for Reimbursement: Medical Foods for Non–Inborn Errors of Metabolism

Leslie Wilson, PhD; Tracy Kuo Lin, MSc, PhD; Anna Oh, RN, MPH; and Vicky Cao, PharmD
Most non–inborn errors of metabolism (non-IEM) medical foods (MFs) do not meet the regulatory MF definition and lack scientific evidence for safety and efficacy. Non-IEM MFs are not yet ready for reimbursement by public insurers.
ABSTRACT

Objectives: The use of medical foods (MFs) specifically for non–inborn errors of metabolism (non-IEM) is rising. Concomitantly, evidence for the safety and efficacy of these non-IEM MFs is lacking. We examined the current use and costs of non-IEM MFs and determined whether the scientific evidence supporting their effectiveness and medical utility is adequate to warrant public reimbursement.

Study Design: We employed a qualitative literature review analysis.

Methods: PubMed and MEDLINE databases were searched for all years using relevant keywords, including names of non-IEM MFs identified in the California Workers’ Compensation System (CAWCS) claims dataset from 2011 to 2013. The quality of extracted data was scored with the Delfini Evidence Tool Kit.

Results: Only 2 (3.2%) of 62 studies were conducted with scientific rigor. These 2 studies were for dietary management of Alzheimer disease, which does not have a distinctive nutritional requirement necessitating an MF. Seventy-one percent of the studies of MFs used by patients in the CAWCS were considered to have uncertain validity.

Conclusions: Most reviewed non-IEM MFs lack evidence to support their safety and efficacy. These non-IEM MFs do not abide by FDA draft guidance, as they do not address a distinct nutritional requirement for a disease and yet often have a National Drug Code or “Rx only” label. Consequently, these products do not meet the statutory definition of an MF. We recommend that CAWCS and other payers not provide insurance coverage for non-IEM MFs until more scientific evidence supports their safety, efficacy, and use for nutritional need of a disease.

Am J Manag Care. 2018;24(Spec Issue No. 8):SP273-SP278
Takeaway Points
  • The cost of medical foods (MFs) is growing; however, there is a lack of scientific evidence to support their safety and efficacy.
  • Payers should not provide insurance coverage for non–inborn errors of metabolism (non-IEM) MFs until there is more scientific evidence supporting their safety, efficacy, and use for the nutritional need of a disease.
  • Independent studies that investigate the efficacy, safety, risk, and benefits of non-IEM MFs are needed.
  • The FDA should implement an MF registry and a framework for evaluation of non-IEM MFs.
Global sales for medical foods (MFs) are estimated at more than $9 billion and expected to exceed $2 billion in the United States.1,2 This projected growth in estimated sales will likely increase the financial burden in the California Workers’ Compensation System (CAWCS), which currently reimburses for MFs.3 The statutory definition and regulations of MFs were originally defined in the Orphan Drug Act, 21 USC 360ee(b)(3) (1983), to promote the easy accessibility of MFs for those with inborn errors of metabolism (IEMs); IEMs are inherited biochemical disorders in which a specific enzyme defect interferes with metabolism of protein, fat, or carbohydrates. MFs are indispensable for these individuals, and most insurers cover their costs.4 However, the current rise in insurance claims for MFs is primarily for non-IEM MFs, and uncertainty surrounds whether the MFs that are the subjects of these claims actually meet the requirements of an allowed MF and whether they should be reimbursed. Insurers and the public need to understand what MFs are and whether the products currently labeled as MFs actually fit the MF definition, are used appropriately to meet a medical nutritional requirement of a disease, and have scientific evidence of safety and effectiveness.

An MF is defined under the Orphan Drug Act 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, based on recognized scientific principles, are established by medical evaluation.”5 This statutory definition is considered by the FDA to narrowly constrain the types of products that fit within this MF category,6 and the regulations of MFs fall somewhere between those of drugs and foods. MFs are not subject to FDA drug regulatory requirements and are exempt from the labeling requirements for nutrient content claims, but they are mandated to adhere to the requirements concerning the misbranding of foods and food allergen labeling. Although any facility manufacturing or processing MFs must register as a food facility with the FDA, the FDA does not keep a list of MF products. MFs require evidence of a “distinctive nutritional requirement for the dietary management of a disease or condition” and “should be consumed or administered enterally under the supervision of a physician” in order to distinguish them from an overall diet to manage symptoms or reduce disease risk. MFs also must be “specially formulated and processed” as opposed to being naturally occurring food used in a natural state. Additionally, MFs are for patients who require a product as a major component of a disease or condition that requires a specific dietary management not met by ordinary diet alone. Finally, although some evidence of effectiveness, safety, and medical need, which is based on “recognized scientific principles,” must be provided, clinical trials are not required as evidence. The quality of this evidence is unknown.

Our objective is to examine if the quality of scientific evidence supporting the effectiveness and medical need for non-IEM MFs is adequate to warrant public payment. Our specific aim is to identify non-IEM MF use by patients in the CAWCS (P/CAWCS) and to conduct an evidence review to assess the extent and quality of the scientific studies for MFs used by P/CAWCS and of all additional non-IEM MF studies that have claimed to support the need for and use of non-IEM MFs.

METHODS

To identify how much evidence exists to support the need for and use of non-IEM MFs, we conducted a 2-stage systematic literature search. First, to gain insight into which non-IEM MFs are frequently used, we identified the CAWCS utilization and costs of paid MF claims, using Workers Compensation Information System complete claims datasets from 2011 to 2013 for both physician- and pharmacy-dispensed non-IEM MF claims. Non-IEM MFs were first identified by the National Drug Code (NDC), product name, and RedBook description that indicated MF status (see Table 1 for highlighted MFs). We also identified MFs based on product websites and package insert descriptions. We then coded the MFs by indication.

We then searched the PubMed database across all years using the MF names prescribed to P/CAWCS. Next, we combined this search with a second, broader systematic literature search using both PubMed and MEDLINE databases with the keyword medical food. Other similar terms were avoided to exclude medical supplements or foods.

Study eligibility and inclusion and exclusion criteria were the same for both search stages. The inclusion criterion was the use of a non-IEM MF as an intervention in an experimental comparative study. Experimental comparative studies included both randomized controlled trials and pre- and postintervention studies. Exclusion criteria included (1) no mention of an MF or the term MF, (2) MFs for IEM, (3) animal studies, (4) nonexperimental comparative studies or those not addressing an MF directly (eg, laboratory, assay, or MF composition study), (5) descriptive or opinion article, (6) patient MF case study, or (7) not in English. Two reviewers independently reviewed the full texts of the eligible studies.

We used the Delfini Evidence Tool Kit (DETK), checklists, and evaluation scheme to grade research designs, evidence, findings, and study conclusions (Table 2).7 The DETK assessed the study validity and usefulness of results and identified the strength of the scientific evidence, applied for our purposes here to non-IEM MFs. Checklists determined (1) if study type influenced results, (2) if methods increased chance findings, and (3) whether clinical outcomes were meaningful. The strength of the evidence grade was based on the Agency of Healthcare Research and Quality and Effective Health Care Program group methodology, assessing risk for bias, consistency, directness, and precision between the interventions and examining whether safety, tolerability, and efficacy were discussed. A final Delfini Evidence grade was given for each study, using the Validity and Usability Grading Scale of A, B, B-U, or U, from each reviewer (Table 2). Grade A indicates that evidence is strong and sufficient to make healthcare decisions; grade B indicates that evidence is potentially strong and probably sufficient; grade B-U indicates possible to uncertain usefulness; and grade U indicates sufficient uncertainty to caution against use in clinical decisions. The 2 reviewers discussed any nonmatching scores and came to an agreement for a single score.


 
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