BETHESDA, MD—Parents of children with rare metabolic disorders and the physicians who treat them are calling for tighter regulation of the dietary supplement industry, so that these children will be vouchsafed the therapeutic benefits they might obtain with routine supplement use.
“The primary victims of an unregulated marketplace are the very sick kids with metabolic diseases whose lives depend on the quality of nutritional supplements they take,” said Abby Meyers, president of the National Organization for Rare Disorders, at an NIH conference on dietary supplements in children. A disease is officially considered a “rare” disorder if it affects fewer than 200,000 people world-wide.
A number of rare diseases, including Wilson’s disease, sickle cell anemia, cystic fibrosis, and short bowel syndrome can be ameliorated with dietary supplements, said Ms. Meyers. But she said the quality of supplements on the market varies widely, and without some sort of widely recognized guarantee of quality, individuals have no way of knowing if a supplement is safe or sufficient for their needs.
She and other members of her association believe that the current regulatory mechanisms are inadequate to ensure that children with such disorders will get what they need. She called for more stringent government regulation, including standardization of dietary supplement products.
Without standards of bioequivalency, clinical trials are almost impossible; even the same manufacturer’s product can differ from lot to lot. And as long as dietary supplements are sold over-the-counter (with minimal FDA regulation), manufacturers have little incentive to make supplements in dosages or formulations needed by those with rare disorders, she argued.
“Insurance companies are happy to tell patients to buy inferior, subquality products that may have no efficacy at all,” said Ms. Meyers, because insurers refuse to reimburse their customers for over-the-counter remedies, even if they are used for medical treatment of severe disease.
Ms. Meyers said the Orphan Drug Act of 1983 has worked well as an incentive for pharmaceutical companies to develop drugs for rare disorders. However, the act does not apply to supplement makers, who do not have exclusive marketing rights for their products. Supplement makers have little incentive to invest in research and development, especially for products that will only be used by a very small percentage of the population.
For example, Ms. Meyers cited Wilson’s disease, a genetic defect that impairs the ability to metabolize copper. While zinc acetate can be used to treat Wilson’s disease, Ms. Meyers said, “OTC zinc acetate could not be trusted. In these patients, it’s critical that the medicine be safe and that all tablets be bioequivalent.”
“We sought sponsorship of zinc acetate as an orphan drug, but corporations were not interested because there was too much OTC competition. Eventually, the generic drug industry took up the issue, and now each zinc acetate tablet is bioequivalent. But health insurers still don’t cover it as a treatment because it’s available OTC,” Ms. Meyers explained.
She also cited problems with biotin, which is used to treat children with congenital biotin deficiency. “It’s available in any store, but the dosage needed for these kids would require taking 50–75 tablets per day.” It’s impossible to determine which of the products on the market have the appropriate bioavailability and contain the needed levels.
“The public deserves purity, an explanation of side effects, contraindications, and expiration dates on every bottle of dietary supplements,” said Ms. Meyers. “That’s not likely, because the bottom line of the dietary supplement industry is profit.”
Virginia Stallings, MD, a pediatrician and director of the Nutrition Center at the Children’s Hospital of Philadelphia, echoed Ms. Meyers’ comments. She notes nutritional supplementation is “common practice” in pediatrics for a number of chronic diseases. She pointed to iron for anemia, magnesium for chronic diarrhea, vitamin B12 for short bowel syndrome, and fat-soluble vitamins for cystic fibrosis.
For sickle cell disease, folate supplementation is recommended, as is avoiding iron supplementation. But, she said, “it’s hard to find a children’s multivitamin that does not have iron.”
Dr. Stallings added, “The nutritional components of disease have never gone through scrutiny” to determine ideal dosages and potential interactions. She emphasized the need for more research on safety of supplements (including dosages and purity), as well as clinical trials and more public education.
The supplement industry claims it has already established voluntary standards for supplements. However, recent laboratory tests by ConsumerLab.com demonstrated nine of 27 multivitamins did not contain what their labels claimed. ConsumerLab does not publish the brands that do not pass its tests, but does indicate products that passed only conditionally. For example, Sundown’s Pokemon Complete multivitamin may contain excessive levels of copper, niacin, vitamin A, and zinc for kids under 9. ConsumerLab.com’s findings seem to underscore the concerns raised by Ms. Meyers and Dr. Stallings.
