This document is based on information presented and discussed at a recent conference sponsored by The Foundation for Innovation in Medicine in New York. Participants included:
Stephen L. DeFelice, M.D.
The Foundation for Innovation in Medicine
William J. Curran, J.D., LL.M., S.M.HYG.
Professor of Legal Medicine
Harvard Medical School
Louis Lasagna, M.D.
Dean, Sackler School of Graduate Biomedical Science
Paul A. Lachance, Ph.D.
Professor and Acting Chairman, Food Science
Donald B. Louria, M.D.
Professor and Chairman, Preventive Medicine and Community Health
University of Medicine and Dentistry of New Jersey
Stephen H. McNamara, J.D.
Hyman, Phelps & McNamara, P.C.
Review of Existing Regulations
Nutritional products with disease related benefits- from diets, dietary supplements and isolated natural substances to genetically engineered designer foods- are rapidly becoming part of mainstream medicine as well as a focus of consumer interest. The result is the “nutraceutical” revolution, in which such products are increasingly developed and recognized for their medical value.
In order for this new field to reach its full potential, however, it must be science-based and proper information should be delivered to both physicians and the public. Just as a responsible, regulated industry has been established for pharmaceuticals, a research-based industry is also needed for nutritional products with medical benefits. Unfortunately, the existing U.S. regulatory structure fails to provide appropriate procedures for the development and approval of nutraceuticals, and actually discourages research investment and the dissemination of health information relating to products in this area.
At the heart of the problem is the fact that current regulations are designed to cover either foods or drugs – but nothing in between. For nutraceuticals, which fall into a gray area between these two categories, there is currently no separate system for regulatory compliance. Recently proposed food regulations, for example, focus almost exclusively on general food labeling. As such, they provide no mechanism for exclusive marketing rights to provide the necessary incentive for investment in the research and development of new products with specific health benefits. Furthermore, they fail to provide for the development of health claims tailored for specific product advances. At the other end of the spectrum, existing drug regulations require rigorous and costly safety and efficacy testing which in most cases is inappropriate for nutritional products.
The obvious solution is to update the regulatory process to adequately and appropriately address nutraceuticals. The goal should be to encourage rather than discourage a research-oriented approach to the development and marketing of nutritional products with health benefits. This can be accomplished by creating a regulatory system for nutraceuticals which diminishes the administrative barriers and financial risks for the research and development of important product innovations, and facilitates the development of exclusive and responsible health claims by individual corporations.
A conference on The Nutraceutical Revolution: Where Do We Go From Here? was held in New York City to review the pros and cons of existing regulations, and it was generally agreed that current guidelines fail to appropriately accommodate nutraceuticals. A proposed solution was presented by The Foundation for Innovation in Medicine, and various options were discussed by the participants.
Current food and drug regulations present a Catch-22 situation for nutritional products with health benefits.
Claims about nutrients usually trigger a requirement for nutrition labeling. U.S. Food and Drug Administration (FDA) regulations, however, state that any food with nutrition labeling will be deemed misbranded if it represents or implies that it is effective in the prevention, cure, mitigation or treatment of any disease or symptom.
Moreover, if a product is properly labeled as a food but independently promoted to show it will help cure, treat, prevent or mitigate disease, it then falls under the realm of drug regulations and is illegal unless in compliance with all federal requirements for drugs. The current research and development cost for a New Drug Application (NDA), which must be reviewed and approved by FDA prior to marketing, typically exceeds $200 million and requires seven years or longer from the start of preclinical testing to final approval. Further requirements include drug registration, drug listing and compliance with pharmaceutical Good Manufacturing Practices.
Another possible classification is as a food additive, which is defined as a food ingredient that is not generally recognized as safe for its intended use. Testing requirements for this classification can be almost as rigorous as for a new drug.
An additional product category which has been accepted by FDA is that of “medical foods.” A medical food is a product intended for use under medical supervision to provide essential nutrition in the context of some disease or symptom. Examples include post-surgical feeding solutions, diabetic foods and hypoallergenic foods. Such products are only promoted to medical professionals for essential nutrition, and need only comply with food requirements.
Within the context of these existing product classifications, however, paradoxes and inconsistencies abound.
In 1984, for example, the FDA implemented a so-called sodium initiative, in which it developed definitions for low-sodium foods and encouraged food processors to develop and market them because of the recognized effect of sodium on high blood pressure. At the same time, however, the FDA would not permit food processors to tell consumers why they should consume low-sodium foods – since the foods were not classified, tested and approved as drugs. This was a distinct disadvantage for the sodium initiative, since the low-sodium foods often did not taste as good as traditional products and the health benefit of low sodium could not be communicated. Food companies were left with the commercially infeasible task of trying to market foods that did not taste good without being able to promote their redeeming health benefits.
On the other hand, despite legal and regulatory positions to the contrary, the promotion of health claims for foods has now become widespread. According to data published earlier this year in the New York Times, 40 percent of all new food products introduced during the first half of 1989 included general or specific health claims, and a third of the $3.6 billion spent annually for food advertising now presents some form of health message.
A turning point may have occurred in the late 1960’s and early 1970’s, when the FDA sought to encourage breakfast cereal manufacturers to emphasize nutrition, rather than sugar and prizes for children, in their promotional activities. Since that era, the cereal industry has leapfrogged the FDA and now places its primary promotional emphasis on nutrition. At the same time, this promotional emphasis on the health benefits of cereals has broadened to encompass a multitude of food products in many categories- with virtually no direct challenge from the FDA.
In order to regain control of the plethora of health claims for food products, the FDA recently proposed regulations for general food labeling which would disallow health claims except in a few situations where they are well supported by publicly available scientific evidence. Claims considered allowable include the role of dietary fiber and low fats in the prevention of colon cancer and heart disease, sodium as a contributor to high blood pressure, and calcium to help prevent osteoporosis.
These proposed regulations essentially legalize current promotional practices in selected instances for general food categories, while ignoring the importance of nutritional supplements and the potential for significant medical and scientific advances. Unlike regulations for pharmaceuticals, no mechanism was proposed to permit exclusive health claims for nutritional products based on proprietary research. This, in effect, removes any financial incentive for private companies to invest in the research and development of innovative nutritional products and product uses.
Moreover, in the absence of research-based health claims developed for specific products, nutritional information reaching the public lacks balance and accuracy. Because specific claims are not developed, nutritional information is dominated by press inter pretations of non-proprietary scientific studies and a profusion of health-related messages for general food categories. In the face of this confusion, it seems evident that both physicians and large segments of the public would appreciate more detailed and balanced information on the role of nutrition in the prevention and treatment of disease.
At present there is no avenue for submitting a new nutritional product with medical value to the FDA short of classifying it as a drug and filing an NDA, at a cost of many millions of dollars and years of testing and evaluation. Even if approved as a drug, however, a company’s exclusive marketing rights for a nutraceutical are uncertain at best.
The nutraceutical revolution has arrived worldwide and cannot be stopped. For the first time in medicine, doctors have joined consumers in believing that nutrients have medical value. This movement will only gain momentum as knowledge of disease processes continues to advance, and new means of product innovation and mass production continue to unfold through biotechnology and other scientific resources.
In the past there was the health food industry, a consumer business largely based on traditional home remedies. Then calcium, fiber and fish oil came along – with clinical data to back them up. In effect, the product managers in this business were the National Institutes of Health and academic medicine, who had conducted clinical studies and presented suggestive scientific information. The marketing and sales force were the mass media, who publicized and dramatized it. And the customers were the physicians and consumers, who increasingly bought it.
This phenomenon has been possible because the FDA does not regulate scientists, or the media, or physicians and consumers. It only regulates industry, which has neither conducted the research nor in most cases made the claims. In other words, there is no regulatory system for the nutraceutical movement- despite the fact that it has entered the mainstream of science, medicine and the consumer marketplace.
What is a science-based company to do if it wishes to participate in the nutraceutical revolution? As of now, no practical and legal approach is available- which bodes poorly for progress in this area.
The obvious solution is to establish an updated regulatory process which serves the public interest and matches today’s scientific and medical reality. Instead of discouraging private research investment for the development of innovative products and responsible claims, a new system must be established which encourages a vital research-oriented industry for the development and marketing of nutraceuticals.
Because of the FDA’s difficulties in regulating nutrition through traditional food and drug guidelines and regulations, Dr. Stephen DeFelice proposed that a Nutraceutical Commission be established specifically for the regulation of this new product category.
The new commission should preside over a well conceived system designed to bring nutraceuticals into the mainstream of the food and health care industries. The new system should diminish the present barriers and risks for private research and development, encourage research by conferring a degree of exclusivity on nutraceutical products, and facilitate and regulate the development of responsible promotional claims.
In order to promote an appropriate level of research for nutritional products and create the exclusivity necessary to attract commercial investment, the Orphan Drug Act might be used as a model. Under the Orphan Drug system, designed to promote pharmaceutical innovations for rare diseases, a company may be permitted to conduct fewer clinical studies and may be granted exclusive marketing rights for seven years after approval.
Since nutritional products do not pose the same potential risks as new drug compounds, the cost of research might be further reduced by omitting preclinical testing requirements, such as the IND (Investigative New Drug application) process mandated for pharmaceuticals. In the case of nutraceuticals, clinical investigators themselves might serve as the initial clinical subjects.
As an additional streamlining measure, in reviewing products for approval the new commission might operate on a case-by-case basis, similar to a court system, rather than through general formal guidelines, which experience has found are difficult to formulate in the area of nutrition.
A regulatory process for nutraceuticals would fill a void not only for the research-based pharmaceutical industry, but for growing segments of the food industry as well. The National Cancer Institute, for example, is now funding research for the development of designer foods with cancer prevention potential through the use of biotechnology. Food science departments of major universities are increasingly becoming authorities on the health-related components of everyday foods. This activity could be further enhanced through block grants from the National Institutes of Health for the clinical testing of nutraceuticals. What is missing, however, is a mechanism for their development into commercial products with specific health claims.
As an alternative to creating a new commission for nutraceuticals, options also exist for developing a regulatory process for nutraceuticals within the existing framework of the FDA.
Streamlined testing and approval requirements could be developed within FDA specifically for this new classification. In order to allow companies to maintain a proprietary advantage from their research investment, the FDA might permit private studies to be held confidential, rather than limiting scientific evidence to publicly available data. Product labeling for specific health claims might in turn be negotiated on an individual basis, in the same manner pharmaceutical labeling is now negotiated with the FDA Drug Center.
Regardless of the form they might take, new regulatory procedures are needed to accommodate the growing array of new products and developments that fall outside the traditional concept of either a food or a drug. In order to be effective and to serve the best interest of the public, this new process should be designed to foster rather than discourage scientific research, encourage the commercial development and availability of important advances, and facilitate the communication of accurate and complete information on nutraceutical products with specific health benefits.