title: Lack of evidence for an unmet need to treat Clostridium difficile infection in infants aged <2 years: expert recommendations on how to address this issue.
abstract: The role of Clostridium difficile in causing disease in infants is unclear, and the existence of C. difficile infection (CDI) in this population is controversial. As part of the drug licensing process for new CDI therapies, a pediatric investigation plan is required to define studies in infants aged <2 years. This assumes an unmet medical need, even though clinical trials in this age group may not be feasible. Three pharmaceutical companies developing CDI treatments came together to seek advice from a panel of experts. Our unanimous opinion is that the existence of CDI is questionable in infants, and if it exists, is rare. There is therefore no unmet need for CDI treatment in this population. Interventional studies are not feasible with the current level of knowledge, and studies should be limited to noninterventional studies or open-label pharmacokinetic and safety studies to better define CDI in infants.
authors.affiliation: National Institute for Health Research Wellcome Trust Clinical Research Facility, University Hospital Southampton NHS Foundation Trust and Academic Unit of Clinical and Experimental Sciences, University of Southampton.
authors.forename: Saul N
authors.initials: SN
authors.lastname: Faust
authors.affiliation: Leeds Teaching Hospitals and Leeds Institute of Biomedical and Clinical Sciences, Faculty of Medicine and Health, University of Leeds, United Kingdom.
authors.forename: Mark H
authors.initials: MH
authors.lastname: Wilcox
authors.affiliation: Department of Paediatric Gastroenterology and Nutrition, Medical University of Warsaw, Poland.
authors.forename: Aleksandra
authors.initials: A
authors.lastname: Banaszkiewicz
authors.affiliation: Microbiology and Infectious Diseases Division, Department of Medicine, Universidad Complutense Madrid, Gregorio Maranon Hospital, Spain.
authors.forename: Emilio
authors.initials: E
authors.lastname: Bouza
authors.affiliation: Université René Descartes-Bacteriology, Paris, France.
authors.forename: Josette
authors.initials: J
authors.lastname: Raymond
authors.affiliation: Stritch School of Medicine, Loyola University Chicago, and Hines Veterans Affairs Hospital, Chicago, Illinois.
authors.forename: Dale N
authors.initials: DN
authors.lastname: Gerding
mesh_terms: D000900:Anti-Bacterial Agents / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D016360:Clostridioides difficile; D003015:Clostridium Infections / Q000188:drug therapy*; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male
chemical_list: D000900:Anti-Bacterial Agents
keywords: C. difficile infection; clinical trial; diarrhea; infants; pediatrics