title: Regulatory Forum Opinion Piece*: Use and Utility of Animal Models of Disease for Nonclinical Safety Assessment: A Pharmaceutical Industry Survey.
abstract: An Innovation and Quality (IQ) Consortium focus group conducted a cross-company survey to evaluate current practices and perceptions around the use of animal models of disease (AMDs) in nonclinical safety assessment of molecules in clinical development. The IQ Consortium group is an organization of pharmaceutical and biotechnology companies with the mission of advancing science and technology. The survey queried the utilization of AMDs during drug discovery in which drug candidates are evaluated in efficacy models and limited short-duration non-Good Laboratory Practices (GLP) toxicology testing and during drug development in which drug candidates are evaluated in GLP toxicology studies. The survey determined that the majority of companies used AMDs during drug discovery primarily as a means for proactively assessing potential nonclinical safety issues prior to the conduct of toxicology studies, followed closely by the use of AMDs to better understand toxicities associated with exaggerated pharmacology in traditional toxicology models or to derisk issues when the target is only expressed in the disease state. In contrast, the survey results indicated that the use of AMDs in development is infrequent, being used primarily to investigate nonclinical safety issues associated with targets expressed only in disease states and/or in response to requests from global regulatory authorities.
authors.affiliation: 1 AbbVie, Inc., Preclinical Safety, North Chicago, Illinois, USA.
authors.forename: Sherry J
authors.initials: SJ
authors.lastname: Morgan
authors.affiliation: 2 Genentech, Inc., Department of Safety Assessment, South San Francisco, California, USA.
authors.forename: Jessica
authors.initials: J
authors.lastname: Couch
authors.affiliation: 3 Janssen Research and Development, Preclinical Development and Safety, Spring House, Pennsylvania, USA.
authors.forename: Peggy
authors.initials: P
authors.lastname: Guzzie-Peck
authors.affiliation: 4 Sanofi, Preclinical Safety, Bridgewater, New Jersey, USA.
authors.forename: Douglas A
authors.initials: DA
authors.lastname: Keller
authors.affiliation: 5 Vertex Pharmaceuticals, Inc., Preclinical Safety Assessment, Boston, Massachusetts, USA.
authors.forename: Ray
authors.initials: R
authors.lastname: Kemper
authors.affiliation: 3 Janssen Research and Development, Preclinical Development and Safety, Spring House, Pennsylvania, USA.
authors.forename: Monicah A
authors.initials: MA
authors.lastname: Otieno
authors.affiliation: 6 Bristol-Myers Squibb, Drug Safety Evaluation, New Brunswick, New Jersey, USA.
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Schulingkamp
authors.affiliation: 7 Eli Lilly and Company, Toxicology and Pathology, Indianapolis, Indiana, USA.
authors.forename: Thomas W
authors.initials: TW
authors.lastname: Jones
mesh_terms: D000818:Animals; D003659:Decision Making, Organizational; D004195:Disease Models, Animal; D004353:Drug Evaluation, Preclinical / Q000379:methods* / Q000706:statistics & numerical data; D004345:Drug Industry / Q000331:legislation & jurisprudence / Q000458:organization & administration / Q000592:standards; D033161:Government Regulation; D011795:Surveys and Questionnaires
keywords: animal models; hazard identification; human disease; regulatory toxicology