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Wednesday, June 27 • 2:15pm - 2:45pm
#377: First-In-Human Studies: An Examination of the Evolving Regulatory and Clinical Practices to Ensure Subject Safety

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Component Type: Session
Level: Intermediate
CE: IACET 0.50

Seating is limited
First-in-human studies are a key milestone in drug development. In such studies, a drug already tested in a preclinical setting (in vitro, animals) is tested in humans for the first time. Study participants include mostly healthy volunteers, sometimes in patient populations, face an element of risk as the ability to predict the effects in humans is limited. There have been a couple of recent notable cases where study subjects have experienced serious harm in such trials. Regulatory guidelines and concepts in study designs have evolved following such events to ensure the safety and well-being of study subjects, and most recently in 2017 the European Medicines Agency (EMA) has revised its 2007 guidance on first-in-human trials. This revised guideline outlines how non-clinical data in PD, PK and toxicology and their translation to humans are important basis for the planning and conduct of first-in-man/ early phase clinical trials. It includes additional strategies to mitigate and mange risks for study subjects, including guidance for the calculation of the first-in-man starting dose, rules for subsequent dose escalation and the criteria for establishing the maximum dose. The guidance also provides criteria to stop a study, review emerging data and handling of adverse events in relation to the study stopping rules. Over the years and especially more recently, first-in-human studies have become increasingly complex and include multiple parts such as single-dose ascension, multiple-dose ascension, food interactions, drug-drug interactions in patient populations, different age groups or gender, proof of concept, or relative bioavailability of different formulations. As such, data generated during the course of these trials should be carefully reviewed and used to inform the decision to initiate a subsequent study part or to inform the selection of the doses to be evaluated.

Learning Objectives

Describe the evolving requirements for conducting first-in-human studies; Discuss the key regulatory and clinical considerations in ensuring subject safety; Discuss the impact of the EMA guidelines on first-in-human studies conducted in North America.


Beatrice Setnik, PhD

avatar for Beatrice Setnik

Beatrice Setnik

Dept of University of Toronto; VP, Scientific and Clinical Strategy, Early Phase, Syneos Health
Dr. Setnik has been working in the area of CNS research and clinical drug development for over 16 years and is an expert in the area of abuse and dependence potential evaluation of drugs. Dr. Setnik is currently the Vice President of Clinical Pharmacology at INC Early Phase and oversees... Read More →

Wednesday June 27, 2018 2:15pm - 2:45pm EDT
Content Hub NE Lobby Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  16: Content Hubs, Session