Non-clinical development and evaluation of microbiome-based medicinal products - Scientific guideline
HumanScientific guidelines
This concept paper proposes the development of a reflection paper to outline the current thinking on the non-clinical evaluation of microbiome-based medicinal products and support a harmonized approach across the European Union for clinical trials and marketing authorisation applications, as per Directive 2001/83 on medicinal products. The development and evaluation of microbiome-based medicinal products pose unique regulatory and scientific challenges that are insufficiently addressed by existing guidelines.
Currently, there is no regulatory framework for the non-clinical evaluation of microbiome-based medicinal products. Conventional pharmacological and toxicological approaches are often not fully applicable, particularly regarding biodistribution, safety, and the use of standard in vivo pharmacodynamic models to support dose selection and efficacy evaluation. The absence of tailored information may lead to increased uncertainty for developers, inconsistencies in regulatory submissions and evaluation, and potential delays in product development.
Keywords: microbiome, live biotherapeutic products, biologicals, whole/highly complex ecosystem-based products, human microbiota transplantation