There remain a number of scientific and technical challenges when developing and marketing microbiome-based medicinal products in the European Union. As the regulatory framework is still in its infancy for these innovative biological medicines, industry and academic stakeholders will have to adopt a flexible, risk-based analysis method of medicinal product development when engaging with health authorities.

Europe’s first regulatory text on Live Biotherapeutic Products (LBP)

Former PRI Executive Director Magali Cordaillat-Simmons (PhD) has been invited to take part in the expert group which drafted the European Pharmacopoeia Monograph 3053 on Live Biotherapeutic Products. She was invited by the French medicinal products competent authority (ANSM) based on the extensive collaborative knowledge on microbiome-based products developed by the PRI over the last decade.

Today, the ANSM maintains its expert position as any new marketing authorization for LBPs might require an update of the dedicated European Pharmacopoeia Monograph.

Human microbiomes being considered as new Substances of Human Origin (SoHO)

Based on a recent assessment of the Blood Tissues & Cells (BTC) legislation, the European Commission has identified 5 important gaps which need to be addressed in the revised legislation, 3 of which directly impact the microbiome industry:

  1. Patients are not fully protected against avoidable risks,
  2. Divergent approaches to oversight cause unequal levels of safety and quality and barriers to the exchange of BTC across the EU
  3. BTC legislation lags behind innovation

In this regards the European Commission is looking at including human microbiomes and maternal milk as Substances of Human Origin (SoHO) in order to regulate their collection when intended for human application in the European Union.

As SoHO can be used as starting material for medicinal products, there is an interplay between the BTC legislation and the EU general pharmaceutical legislation, and this revision could be the opportunity for the EU to clarify the status or even assessment modalities for microbiome-based medicinal products derived from fecal material, maternal milk or other human microbiome samples.

In this area, the PRI, as the EU expertise center in microbiome regulatory science, was invited as a stakeholder in order to provide its input on the technological and scientific challenges emerging from the various options envisaged in the revision. This is another way the PRI supports its Members in the emergence of pre-competitive regulatory science projects in order to coordinate the efforts of  academia and industry in seeking the best-adapted standards for ongoing development.

Microbiome Science being considered in the EU Pharmaceutical Legislation Revision

With the purpose of obtaining additional stakeholder feedback to improve the aforementioned points, the European Commission mandated a study supporting the evaluation of the EU Pharma Legislation.

During the stakeholder workshops, the PRI has been sharing its expertise and has made proposals to help improve EU regulatory attractiveness as well as access to innovative medicinal products for which the EU could be lacking streamlined regulatory pathways (such as microbiome-based medicinal products). Clarification of regulatory status and requirements, and the initiation of formalized and intense regulatory science activities within the competent authorities in collaboration with experts in the field are of high importance if the EU intends to stay ahead in microbiome innovation.

Thus, the PRI is actively involved in ensuring that microbiome science and microbiome-based medicinal products are considered throughout the revision of such an important piece of legislation.