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Assay Drug Dev Technol. 2017 Dec;15(8):354-361. doi: 10.1089/adt.2017.821. Epub 2017 Dec 1.

When Enough Is Enough: Decision Criteria for Moving a Known Drug into Clinical Testing for a New Indication in the Absence of Preclinical Efficacy Data.

Author information

1
1 Vanderbilt Institute for Clinical and Translational Research, Vanderbilt University Medical Center , Nashville, Tennessee.
2
2 Center for Knowledge Management, Vanderbilt University Medical Center , Nashville, Tennessee.
3
3 Division of Infectious Diseases, Department of Medicine, Vanderbilt University School of Medicine , Nashville, Tennessee.
4
4 Division of Pain Medicine, Department of Anesthesiology, Vanderbilt University School of Medicine , Nashville, Tennessee.
5
5 Department of Pharmacology, Vanderbilt Center for Neuroscience Drug Discovery, Vanderbilt University Medical Center , Nashville, Tennessee.
6
6 Vanderbilt Kennedy Center for Research on Human Development , Nashville Tennessee.
7
7 Division of Geriatric Medicine, Department of Medicine, Vanderbilt University School of Medicine , Nashville, Tennessee.
8
8 Division of Rheumatology and Immunology, Department of Medicine, Vanderbilt University School of Medicine , Nashville, Tennessee.
9
9 Center for Technology Transfer and Commercialization, Vanderbilt University , Nashville, Tennessee.

Abstract

Many animal models of disease are suboptimal in their representation of human diseases and lack of predictive power in the success of pivotal human trials. In the context of repurposing drugs with known human safety, it is sometimes appropriate to conduct the "last experiment first," that is, progressing directly to human investigations. However, there are not accepted criteria for when to proceed straight to humans to test a new indication. We propose a specific set of criteria to guide the decision-making around when to initiate human proof of principle without preclinical efficacy studies in animal models. This approach could accelerate the transition of novel therapeutic approaches to human applications.

KEYWORDS:

animal models; drug repurposing; translational research

PMID:
29193979
PMCID:
PMC5731541
DOI:
10.1089/adt.2017.821
[Indexed for MEDLINE]
Free PMC Article

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