Speeches & Testimony

Access to Dietary Supplements for Valid Medical
Uses in Children

Thank you for inviting me to speak about access to dietary supplements for valid medical uses in children. I am Abbey Meyers, President of the National Organization for Rare Disorders (NORD). NORD represents the orphan disease community. Under federal law, rare diseases each affect fewer than 200,000 Americans. There are more than 6,000 of these little known disorders, cumulatively affecting an estimated 25 million people in the United States. In general, Americans with unusual diagnoses are medically disenfranchised. It can take years to simply get a diagnosis, treatments do not often exist, and research is rarely pursued.

In 1983 Congress passed the Orphan Drug Act, which created incentives to entice pharmaceutical companies into developing “orphan drugs” to treat rare diseases. This includes tax credits to reduce the cost of clinical research, and seven years exclusive marketing rights, which prevents other companies from marketing the same drug for the same disease.

The Orphan Drug Act has worked very well. At last count there were about 210 FDA approved orphan drugs on the U.S. market, and about 800 more in the research pipeline. Only a handful of these compounds could be classified as nutritional supplements. Thus the success of the law cannot be extrapolated to the supplement industry. This is because the few manufacturers that spent many years and millions of dollars getting compounds that could ordinarily be classified as nutritional supplements, approved as pharmaceutical products, had such hair-raising experiences that other companies refuse to follow.

Primarily, there can be no exclusivity for a nutritional supplement because anyone can put a similar product on the over-the-counter market and make any claim without having to conform to FDA’s quality control requirements, nor do they have to pay for research to prove safety or efficacy. Therefore, despite orphan drug exclusivity for those who are brave enough to create a prescription drug out of a dietary supplement, there will be competition from lower cost unregulated supplements.

Firstly, no one has clearly defined what is a “drug” and what is a “nutritional supplement”. FDA’s federal court battle to remove the cholesterol lowering agent, Cholestin, from the market, proves that a legal differentiation is critically needed. But if we ever get passed that point, there are compounds that will clearly be defined as supplements which may have therapeutic benefit for certain people, and particularly children with a group of about 600 rare diseases known as “inborn errors of metabolism”. These are generally hereditary diseases that are characterized by an inability to metabolize certain elements that naturally occur in nature, such as food.

The most familiar of the metabolic diseases would be phenylketonuria, better known as PKU. Every newborn baby in the United States is tested for PKU through a drop of blood taken from their foot. If they test positive, their diet must be drastically altered because they cannot eat normal food. If they adhere to a very strict diet primarily eating medical formulas, they will grow up normally; but if they eat ordinary food containing certain nutrients, they will become mentally retarded and suffer other very serious complications.

In future years, newborn screening programs in every state will certainly expand the number of metabolic diseases that are screened for in these tests. Today some states screen for three or five diseases, while others screen for up to 40. It is safe to predict that at least some of these diseases may be eligible for treatment with nutritional supplements.

However, the FDA does not regulate the supplement industry. Therefore, there is no way to determine whether each pill or tablet contains the labeled ingredient, and if it does, whether each tablet or brand is bioequivalent, dissolves properly, etc.

Take the case of Wilson’s disease, for example. This is a hereditary inborn error of metabolism caused by a defect in liver enzymes. It is characterized by an abnormal accumulation of copper in body tissues. When the copper accumulates in the brain, it causes severe behavioral abnormalities, neurological deficits and death. It is therefore critically important that any medication these people use is safe and effective, and that every tablet is absolutely bioequivalent.

Years ago a scientist at the university of Michigan recognized that zinc acetate chelates copper from the body, so he wondered if zinc acetate could be used to maintain low copper levels in Wilson’s disease patients. But every brand of zinc was different, and sometimes every pill in a bottle was different. Over-the-counter zinc acetate could not be relied upon to have a consistent therapeutic effect. Thus a controlled clinical trial of commercial zinc acetate was not possible. Additionally, a scientist cannot get a drug approved by FDA because he doesn’t have a manufacturing facility, and he is not a company, so a corporate sponsor was necessary.

Because NORD is the consumer organization that advocated for the Orphan Drug Act, we sought a pharmaceutical company that might be willing to sponsor zinc acetate as an orphan drug. But only 2,000 people in the United States have Wilson’s disease so no company was interested. They also told us that even if a prescription version of zinc acetate could be approved by FDA for marketing, they would have too much low cost competition from over-the-counter supplement manufacturers, and therefore it wasn’t worth the investment. After several years of denials from large companies, we sought help from our friends in the generic drug industry. Ultimately a generic company adopted the drug as a public service, and the university scientist was able to pursue clinical trials.

When the prescription version of zinc acetate finally achieved FDA approval, every Wilson’s disease patient in the United States could be assured that every tablet in the prescription drug contained exactly the same ingredients, that they dissolved in the blood stream at the same rate, and that each tablet released the exact amount necessary to keep copper levels low in these patients. However, their health insurers refused to reimburse for the prescription product because they said that zinc acetate was available over-the-counter at a lower cost.

A similar scenario occurred with L-carnitine for babies with carnitine deficiency. Because they have little muscle strength, these babies die. It is absolutely essential that they receive a dependable product to replace carnitine in their muscles, but health insurers insist that nutritional supplements should suffice. We tested some of these over-the-counter carnitine brands several years ago and found that some of them had barely discernable carnitine content, and one had none at all. It’s like playing Russian Roulette with babies.

In the case of carnitine, when one state Medicaid system told parents that they would not pay, the manufacturer of the prescription drug had enough, and he went to court. He proved that because the FDA does not regulate nutritional supplements, they cannot be substituted for prescription drugs. Nevertheless, that manufacturer continues to battle health insurers on a daily basis, as well as Medicare which in some states refuses to pay for the intravenous version of carnitine.

Besides the quality problems, and the reimbursement problems that patients with valid medical needs experience with nutritional supplements, there is also a dosage problem. With some inborn errors of metabolism that do not have prescription drug versions, they must use whatever they can find over-the-counter. For biotin deficiency, for example, one can find biotin tablets on the shelves of any health food store, but not in the milligram strength that it is needed for children with biotin deficiency. It is possible, for example, for a child with this disorder to need 50 or 75 pills per day in order to obtain the proper dosage. It is impossible for a parent to know which brand of biotin might have close to the amount of biotin listed on the label, not to mention the cost of several bottles of Biotin needed each week, which is not reimbursed by health insurers.

The experience of the metabolic disease community is an unfortunate lesson. The public is learning that the unfounded claims of supplement makers has reverted the health care marketplace back to the beginning of the 20th century when snake oil salesmen claimed that their tonics and potions cured everything. In those days, there was no requirement to accurately list the ingredients in each medicine, and the same is true today. It is a buyer’s beware marketplace and the public is just learning to beware.

But for those who have a valid medical need for substances that appear in nature, politics further complicates the scenario. The supplement industry is very rich and powerful, and it does not want to be regulated. On the other hand, the FDA does not want another unfunded mandate, so it is not eager to regulate the industry. One answer to this riddle is to find a way to get the pharmaceutical industry interested in manufacturing these products as drugs, but they refuse. Most of the large multi-national companies have gone into the supplement manufacturing business, and they like selling unregulated products. Here is a niche where they can market their products and make unproven health claims that they don’t dare make for their prescription products, and they rely on the public associating their pristine corporate names with quality products.

It might be possible to identify a few small entrepreneurial companies willing to develop prescription versions of supplement product, but, if they do, will insurance pay? Insurers are perfectly happy to tell patients to buy the inferior unregulated supplements that may contain no active ingredients at all! They simply do not want to pay the bills.

Consumers want access to dietary supplements for medical uses that are valid or invalid. But they should expect assurance that every pill contains the appropriate ingredients. The least we deserve is a quality-controlled system like the generic drug program that requires every tablet to be absolutely equivalent to all other brands of the same drug. However, since the main concern of the supplement industry is healthy individuals, not sick people, their products do not have to show any therapeutic effect. Healthy consumers buy these products on faith that they may prevent disease, not cure it.

We believe, however, that the public deserves purity, an explanation of side effects, contraindications, and expiration dates on every bottle. Will we get this? Hardly likely, because the bottom line of the supplement industry is profits and politics. It is Congress that will decide whether the industry ought to be regulated, and Congress is vulnerable to carefully orchestrated propaganda campaigns that threaten the profit of the industry. Until there is government regulation, however, those with valid medical need for nutritional supplements must find their own way in the buyer beware marketplace. Unfortunately, the primary victims of this unregulated marketplace will not be the healthy people who fear heart disease, cancer, sexual impotence or aging, but instead the very sick children with metabolic diseases whose lives depend on the quality of nutritional supplements.

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