In 2007, the National Institutes of Health (NIH) launched the Human Microbiome Project (HMP), a $150 million initiative to characterize the microbial communities found at several different sites on the human body and to analyze the role of these microbes in human health and disease.² Many lines of research have demonstrated the significant role of the microbiota in human physiology. The microbiota is involved, for example, in the healthy development of the immune system, prevention of infection from pathogenic or opportunistic microbes, and maintenance of intestinal barrier function. Goals of the HMP have been described as “identifying new ways to ‘determine health and predisposition to diseases [as well as defining] the parameters needed to design, implement and monitor strategies for intentionally manipulating the human microbiota, to optimize its performance in the context of an individual’s physiology.’”³

As part of the HMP, NIH funded the “Healthy Cohort Study,” an effort to create a reference catalogue of microbial DNA in healthy adults⁴ as well as fifteen demonstration projects focusing on bacterial, fungal, and viral changes in microbiomes in individuals with various diseases. Early findings of the HMP were published in June 2012.⁵ While the findings are helping us understand the role and variation of microorganisms within and across individuals, they are also promoting interest in the development of probiotic products.⁶

Probiotics are substances containing live microorganisms that are thought to have a beneficial effect on the human body by manipulating microbiome and host properties.⁷ Research has shown that it is possible to categorize the microbiota components on the basis of whether they exert potentially pathogenic or health-promoting aspects. For example, lactic acid-producing genera such as bifidobacteria or lactobacilli have a long-standing association with health. These bacteria can be increased in the human body (at least for a period of time) either by feeding individuals appropriate strains as a probiotic or through the provision of prebiotic growth substrates. While several probiotics are now undergoing preclinical and clinical trials,⁸ none have as yet been approved as drugs in the United States. Probiotics have, however, been available as foods and dietary supplements for many years. Initially marketed in yogurts and dairy products, the use of probiotics in commercial products has skyrocketed in recent years.⁹ Other probiotic products include juices, nutrition bars, infant formulas, relishes and condiments, sweeteners, waters, pizza crust, gum, lozenges, dietary supplements, toothpaste, and cosmetics.¹⁰

In addition to funding the Healthy Cohort Study and the demonstration projects, NIH set aside a portion of HMP funds to study the ethical, legal, and social implications (ELSI) of the HMP’s scientific goals. Among the funded ELSI studies was an effort to look at the current regulatory framework for probiotics¹¹ and to determine if it is a good fit for the range of probiotics that are on the market, under development, or that may be developed in the future as a result of the HMP. New claims are being made about the role and value of probiotics in promoting human health and well-being, and there is both uncertainty and debate about how these products should be regulated.¹²

As probiotics begin to proliferate in the market, policy makers and regulators need to critically consider the regulatory structure that is most appropriate for them. This consideration should incorporate the wide range of probiotic products that are and may become commercially available as foods, food additives, drugs, dietary supplements, and cosmetics, and should anticipate the future types of probiotic products that may be developed. Scientists have theorized that, in the future, there may be interest in combining probiotics to leverage their different properties, perhaps with personalized probiotics for a healthy microbiome. Experts in the field also expect that there will be more interest in genetic engineering of probiotics for specific medical purposes as more is known about probiotic mechanisms of action. In order to protect and guide consumers and health care providers who may use or recommend the use of probiotics, a regulatory structure that adequately accounts for the risks of probiotics as well as the accuracy of claims of effectiveness is necessary. In addition, the regulatory structure needs to be flexible enough to allow for (or at least not discourage) research on new probiotic products that may have therapeutic benefits.¹³

This article reports on the findings of a Working Group (WG) consisting of NIH-funded HMP scientists, physicians, legal academics, government regulators, industry and consumer representatives, bioethicists, food and drug lawyers, and health policymakers who were assembled to address the adequacy of the current regulatory framework for probiotics under the HMP ELSI funded project.¹⁴ Specifically, after discussion of the features of probiotics that are relevant to their regulation and an overview of FDA’s current regulation of probiotics, the article addresses the following questions: 1) Do current regulations adequately address the safety of new probiotic products? 2) Should probiotic foods and dietary supplements be classified as drugs and required to go through the drug approval process? 3) What types of product characterization requirements are appropriate for probiotics? 4) Are current claim regulations appropriate for probiotics and, if not, how might they be improved?

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