FDA Commissioner Scott Gottlieb, MD, has issued the following statement on advancing the science and regulation of live microbiome-based products used to prevent, treat or cure diseases in humans: "Over the last several years, there’s been growing scientific and public interest in the role of microorganisms in the maintenance of overall health and prevention and treatment of disease. Today, more so than ever before, consumers are familiar with the phrase “good” or “helpful” bacteria and the expectation that it will aid in treating or preventing various illnesses. As the field advances, the U.S. Food and Drug Administration is playing a key role in sorting through the science and the science fiction of this evolving field.
"We know that certain medical issues, such as bacterial infections and their treatment with antibiotics, may disrupt the normal complement of microorganisms that naturally live within the body. Microbiota, the collection of organisms in and on our body, may also affect how patients respond to certain drugs, as some research has shown the possibility of manipulating the microbiota to improve cancer treatment. There is also some preliminary evidence suggesting that certain microorganisms or “good bacteria” given proactively may help prevent some diseases.
"For example, researchers are studying whether the administration of certain microorganisms in the form of probiotics can help prevent a serious disease in premature infants called necrotizing enterocolitis, which has been associated with systemic infection and death. With the health and safety of this most vulnerable population in mind, more research is needed as to whether this is a viable treatment.
"The data collected so far in premature infants are difficult to interpret because different probiotic preparations have been studied, often in conjunction with other interventions. Understanding whether the use of probiotics for premature infants can have a clinically significant beneficial effect, and which types of probiotics may be most effective, is important, as serious side effects have been reported with their use in this population. We are very concerned by these potential risks and are actively monitoring these uses. These risks underscore the need for more definitive evidence showing benefit in preventing serious diseases. We need to make sure that the products are delivering benefits that justify any potential risks. This is especially important as these products are being used in vulnerable patients. We will consider taking action when we see unsafe or violative products.
"In 2016, the FDA issued a guidance document that explains how researchers studying probiotics as drugs can meet the manufacturing requirements necessary for early clinical trials. More work and continued partnership between the FDA and various stakeholders is needed to advance the clinical science necessary to appropriately understand the safety and effectiveness of these products. We are committed to these goals.
"The FDA has also not approved any probiotic as a live biotherapeutic product (LBP), a biological product other than a vaccine that contains live organisms used to prevent or treat a disease or condition in humans. However, there are FDA-regulated foods, including dietary supplements, containing probiotics that are legally available, though these products cannot lawfully be marketed to cure, mitigate, treat, or prevent any diseases. The 2016 guidance also clarifies how some LBPs that are lawfully marketed as a food or dietary supplement can also be studied for investigational use.
"The FDA is also considering ways to provide additional clarity on information provided on the supplement facts label for declaring colony forming units of probiotics and weight. We understand that there continues to be growing interest in the potential benefits of probiotics in foods, including dietary supplements, and we are committed to working with industry on efforts to provide information that can help consumers make more informed choices about these products.
"Given the strong interest and rapidly evolving science in this field, the FDA is convening a workshop co-hosted with the National Institutes of Health on September 17 that will discuss microbiome-based products and how manipulation of the microbiome may potentially be used to prevent or treat a variety of different diseases. Presentations will be given on a variety of topics including: the regulatory framework for live microbiome-based products; safety and effectiveness of live microbiome-based products used to prevent, treat, or cure diseases in humans; and strain selection for live microbiome-based products to prevent, treat, or cure diseases in humans. The workshop will also include panel discussions. We look forward to the robust scientific conversations that are certain to ensue.
"This workshop is part of our broader effort to advance regulatory science in this field, and make sure we establish modern, efficient policies for how we evaluate products that fall under our different regulatory authorities. We recognize the potential benefit offered by these products, but we also have identified certain risks. We need to make sure that these are properly evaluated when these products are marketed and used as drugs, and that consumers have good information on which to base decisions when probiotics are used in conventional foods and dietary supplements. By doing our part in facilitating the conduct of well-designed clinical trials, the FDA and NIH hope that the full potential of the new science of the microbiome can be realized to benefit both individual and public health."