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Microbiome Regulation Emerges From the Shadows: FDA to Co-Host Workshop on Live Cell Therapeutics

| Agnes Juang, Ph.D.

Research supporting the role of the microbiome in human, animal, and plant health continues to grow at a staggering rate.  While the wave of new health technologies emerging from this research rises, companies working in the microbiome space have had to confront a number of unique, interlinked technical, intellectual property, and regulatory issues that threaten to derail this extraordinarily promising area of development.  Scientists and entrepreneurs have been hard at work overcoming the technical obstacles to microbiome-based technologies (and intellectual property attorneys have been hard at work finding ways to protect their inventions), but until now the regulatory landscape surrounding the microbiome has been frighteningly unsettled.  Previously, the U.S. Food and Drug Administration (FDA) released an update to its guidance on early clinical trials with live biotherapeutic products as a first step toward providing a regulatory path for microbiome-facing technologies.  Nevertheless, uncertainty regarding the regulation of live probiotic organisms has driven many developers to alter their therapeutic strategy in favor of options that avoid the use of live cells, such as antimicrobials, prebiotics, and bacterial extracts. 

The movement toward regulatory certainty will shortly take a large step forward. The FDA has announced that it will hold a one-day workshop, co-hosted with the National Institutes of Health, on September 17, 2018 to discuss microbiome-based products.  Topics will focus on the clinical, manufacturing, and regulatory considerations for live microbiome-based products, and are expected to include possible assessments of safety and efficacy for microbiome-based therapeutics, unique aspects of the overall regulatory framework for microbiome-based products (including issues such as strain selection and cell banking), product risks, and future research directions.  The Director’s announcement notes that the FDA has thus far not approved any probiotic for therapeutic uses under its definition of a live biotherapeutic product, despite the fact that probiotics have long been used in dietary supplements and FDA-regulated foods.  The proposed workshop appears to be an effort to address this discrepancy.

The FDA has recently initiated a number of efforts to resolve novel or unsettled regulatory issues through aggressively seeking public and stakeholder comment.  The proposed microbiome workshop, while clearly an early step in the process, is a welcome continuation of these efforts, which takes into account the expertise of researchers and developers in this fast moving and increasingly important field.  How to properly oversee the development of these technologies still raises several significant questions, including those surrounding the introduction of bacterial strains novel to humans, the use of potentially pathogenic strains, and the use of genetically modified probiotic strains, among myriad others.  Establishing early frameworks for the development of regulations in this space in an open and transparent manner will be an immeasurable help to those wrestling with product development decisions surrounding promising probiotic or microbiome-modulating technologies. 

Information on registration for the workshop can be found here.