Conducting Human Research on Probiotics in the United States: Legal Perspectives

On June 12, 2013, scientists gathered in the 40th floor of 7 World Trade Center in New York City to discuss “Probiotics, prebiotics and the host microbiome:  the science of translation.” This conference, cosponsored by the Sackler Institute of Nutrition Sciences, the New York Academy of Sciences and the International Scientific Association for Probiotics and Prebiotcs, addressed a range of topics (see conference agenda). One ongoing challenge for the probiotic field is how to conduct research on probiotic foods and dietary supplements without triggering the requirement for an investigational new drug application. Attorney Fred Degnan, of King & Spalding, shared his perspectives on legal approaches to human research on probiotics.

Both academicians and industry scientists in the audience were very interested in this topic. There are multiple converging issues at play. The industry scientists want the research to be on foods/supplements, not on drugs. Academics want to conduct the research, to advance the science and their careers.  The research in question is on the healthy population and the probiotics have a long history of safe use. Therefore, the safety of the studies is not in question. The FTC is cracking down on what they consider to be unsubstantiated claims on probiotic products in the US market. Industry is seeking guidance on how to conduct the needed human research to substantiate benefits of their foods and supplements without having the FDA interpret their products as drugs.

In brief, Mr. Degnan characterized the problem and then offered some simple solutions:

The problem:

  • As a general rule regarding probiotics, the degree of FDA regulation of a clinical trial depends on the purpose(s) for which a given probiotic is being investigated
  • Biologic products are drugs consisting of or derived from live microbes. Since a probiotic is a live microbe, regulation of probiotic drug studies has come under the jurisdiction of the Center for Biologics Evaluation and Research (CBER).
  • Unlike the definition of drugs, the definition of “biological product” does not specifically refer to intended use:  biological products are “applicable to the prevention treatment or cure of disease …”. As a result, real care needs to be exercised in documenting/substantiating non-biological product use.
  • Since both foods and drugs can impact the structure and function of the human body or reduce the risk of disease in the general (healthy or at-risk) population, it must be very clear when this type of research is targeted for a food/supplement product.

The approach:

  • Understand and appreciate the regulatory distinctions between “foods/supplements” and ”drugs/biological products”
  • Understand and appreciate the criteria and endpoints for establishing S/F, “health claim”, and medical food endpoints for research
  • If the goal is to research food/supplement based products, conceive and design a research plan to incorporate desired food endpoints and avoid non-food disease related endpoints in study protocols
  • Consult with FDA’s CFSAN for guidance/answers with respect to assuring that clinical investigations re: food/supplements are consistent with FDA-related “food/supplement” safety and categorization criteria

Consumers, scientists, industry and regulatory bodies benefit when well-designed research on probiotics proceeds. Mr. Degnan’s advice can provide some clarity in the path to conduct this research.