A number of articles have been published in medical journals on insurance coverage for medical foods within the last 5 years. In addition, National PKU News featured an interesting piece about health insurance written by a parent. You may want to review these for background information on this issue of significant relevance to our patient families. Links to these articles can be found here:
1. Khamsi R. Rethinking the formula. Nature Medicine. 2013; 19 (5): 525-529.
http://www.nature.com/nm/journal/v19/n5/full/nm0513-525.html?WT.ec_id=NM-201305)
Abstract:
Health insurance covers drugs approved by regulatory agencies, but it often doesn't pay for the products known as 'medical foods' needed to keep individuals alive and well. This lack of reimbursement means that many who cannot afford these life-saving diets suffer brain deterioration and disability—or worse. Roxanne Khamsi reports on the battle for medical foods and how it could affect the treatment of diseases as diverse as osteoporosis and Alzheimer's.
2. Berry SA, Brown C, Grant M, Greene CL, Jurecki E, Koch J, Moseley K, Suter R, van Calcar SC, Wiles J, Cederbaum S. Newborn Screening 50 years later: access issues faced by adults with PKU. Genetics in Medicine. Published online March 7, 2013.
http://www.nature.com/gim/journal/vaop/ncurrent/full/gim201310a.html
Abstract:
Fifty years after the implementation of universal newborn screening programs for phenylketonuria, the first disease identified through newborn screening and considered a success story of newborn screening, a cohort of adults with phenylketonuria treated from birth provides valuable information about effects of long-term treatment for inborn errors of metabolism in general, and phenylketonuria specifically. For phenylketonuria, newborn screening allows early implementation of the phenylalanine-restricted diet, eliminating the severe neurocognitive and neuromotor impairment associated with untreated phenylketonuria. However, executive function impairments and psychiatric problems are frequently reported even for those treated early and continuously with the phenylalanine-restricted diet alone. Moreover, a large percentage of adults with phenylketonuria are reported as lost to follow-up by metabolic clinics. While a group of experts identified by the National Institutes of Health convenes to update treatment guidelines for phenylketonuria, we explore individual patient, social, and economic factors preventing >70% of adult phenylketonuria patients in the United States from accessing treatment. As more conditions are identified through newborn screening, factors affecting access to treatment grow in importance, and we must continue to be vigilant in assessing and addressing factors that affect patient treatment outcomes and not just celebrate amelioration of the most severe manifestations of disease.
3. Berry SA, Kenney MK, Harris KB, Singh RH, Cameron CA, Kraszewski JN, Levy-Fisch J, Shugar, J, Greene CL, Lloyd-Puryear MA, Boyle CL. Insurance coverage of medical foods for treatment of inherited metabolic disorders. Genetics in Medicine. Published online 18 April 2013.
http://www.nature.com/gim/journal/vaop/ncurrent/full/gim201346a.html
Abstract:
Purpose:Treatment of inherited metabolic disorders is accomplished by use of specialized diets employing medical foods and medically necessary supplements. Families seeking insurance coverage for these products express concern that coverage is often limited; the extent of this challenge is not well defined.
Methods: To learn about limitations in insurance coverage, parents of 305 children with inherited metabolic disorders completed a paper survey providing information about their use of medical foods, modified low-protein foods, prescribed dietary supplements, and medical feeding equipment and supplies for treatment of their child’s disorder as well as details about payment sources for these products.
Results: Although nearly all children with inherited metabolic disorders had medical coverage of some type, families paid “out of pocket” for all types of products. Uncovered spending was reported for 11% of families purchasing medical foods, 26% purchasing supplements, 33% of those needing medical feeding supplies, and 59% of families requiring modified low-protein foods. Forty-two percent of families using modified low-protein foods and 21% of families using medical foods reported additional treatment-related expenses of $100 or more per month for these products.
Conclusion: Costs of medical foods used to treat inherited metabolic disorders are not completely covered by insurance or other resources.
4. Camp K, Lloyd-Puryear, MA, Huntington, KL. Nutritional treatment for inborn errors of metabolism: Indications, regulations and availability of medical foods and dietary supplements using phenylketonuria as an example. Molecular Genetics and Metabolism. 2012; 107(1-2):3-9.
http://www.sciencedirect.com/science/article/pii/S1096719212002612
Abstract:
Medical foods and dietary supplements are used to treat rare inborn errors of metabolism (IEM) identified through state-based universal newborn screening. These products are regulated under Food and Drug Administration (FDA) food and dietary supplement statutes. The lack of harmony in terminology used to refer to medical foods and dietary supplements and the misuse of words that imply that FDA regulates these products as drugs have led to confusion. These products are expensive and, although they are used for medical treatment of IEM, third-party payer coverage of these products is inconsistent across the United States. Clinicians and families report termination of coverage in late adolescence, failure to cover treatment during pregnancy, coverage for select conditions only, or no coverage. We describe the indications for specific nutritional treatment products for IEM and their regulation, availability, and categorization. We conclude with a discussion of the problems that have contributed to the paradox of identifying individuals with IEM through newborn screening but not guaranteeing that they receive optimal treatment. Throughout the paper, we use the nutritional treatment of phenylketonuria as an example of IEM treatment.
5. Belanger-Quintana A, Dokoupil K, Gokmen-Ozel H, Lammardo AM, MacDonald A, Motzfeldt K, Nowacka M, Robert M, van Rijn M, Ahring K. Diet in phenylketonuria: A snapshot of special dietary costs and reimbursement systems in 10 international centers. Molecular Genetics and Metabolism. 2012; 105: 390–394
http://www.sciencedirect.com/science/article/pii/S1096719211006226
Abstract:
Background and aims: To gather exploratory data on the costs and reimbursement of special dietary foods used in the management of phenylketonuria (PKU) from ten international specialist PKU centers.
Methods: Experts from each center provided data on retail costs of the three most frequently used phenylalanine-free protein substitutes and low-protein foods at their center; reimbursement of protein substitutes and low-protein foods; and state monetary benefits provided to PKU patients.
Results: The mean annual cost of protein substitutes across 4 age groups (2 y, 8 y, 15 y and adults) ranged from €4273 to €21,590 per patient. The cost of low-protein products also differed; the mean cost of low-protein bread varied from €0.04 to €1.60 per 100 kcal. All protein substitutes were either fully reimbursed or covered by health insurance. However, reimbursement for low-protein products varied and state benefits differed between centers.
Conclusions: The variation in the cost and reimbursement of diet therapy and the level of additional state benefits for PKU patients demonstrates the large difference in expenditure on and access to PKU dietary products. This highlights the inequality between healthcare systems and access to special dietary products for people with PKU, ultimately leading to patients in some countries receiving better care than others.
6. Weaver MA, Johnson A, Singh R, Wilcox WR, Lloyd-Puryear MA. Medical Foods: Inborn errors of metabolism and the reimbursement dilemma. Genetics in Medicine. 2010; 12(6):364-369.
http://www.nature.com/gim/journal/v12/n6/full/gim201058a.html
Abstract:
Purpose: Medical foods and pharmacological doses of vitamins are used to treat certain genetic diseases for the duration of a patient's lifetime, which necessitates life-long management of the condition and diet by the patient and a health care provider. However, payment for medical foods and health insurance coverage of medical foods is not uniform.
Methods: A survey of states' newborn screening (NBS) representatives and a review of state policies (as of 2008) were conducted to ascertain payment and insurance coverage of medical foods.
Results: According to the NBS representatives, 61% of the states provided or guaranteed medical foods for all or a subset of the population detected by NBS, whereas 82% of states provided or guaranteed medical formulas for the same population. Policies for private health insurance coverage existed in 33/50 states, and range from providing medical food for one specific metabolic condition to providing it for any NBS disorder. In addition, there is variability among states in the specificity of defining what conditions qualify for medical foods.
Conclusion: This article suggests four options, not mutually exclusive, options for addressing the patchwork of state policies regarding coverage of medical foods, ranging from amending Medicaid legislation to enacting federal legislation, or changing the Food and Drug Administration's stance on oversight of medical foods.
7. Huntington KL, Buist NRM. Medical Food for Treatment of Inborn Errors of Metabolism and State Legislative Mandates. Topics in Clinical Nutrition. 2009; 24 (4): 289-306.
http://journals.lww.com/topicsinclinicalnutrition/toc/2009
Abstract:
Newborn screening for inborn errors of metabolism (IEM) is a model of preventative medicine in public health. The primary treatment for about 30 IEM depends on medical foods as defined by federal legislation. Insurance reimbursement for medical foods in the United States is typically denied despite recognition of the therapeutic legitimacy of those foods. State legislation mandating medical food coverage represents a chaotic patchwork of laws limiting treatment of different disorders and medical food options. In addition, obstacles within the healthcare system also interfere with reimbursement. Families dealing with the same disorders in different states are faced with an inequitable financial burden. Federal standards for healthcare benefits should be established, recognizing the existence of rare diseases and the treatment requirements in relation to the developmental needs of children and adult metabolic homeostasis that will support uniform reimbursement
8. Corry S. A look at insurance coverage and PKU. NPKU News. 2012;24 (3): 7- 9, 16.
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