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
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Rebecca N Jerome, Robert R. Lavieri, Gordon R. Bernard, Helen M. Naylor, Somsundaram N. Chettiar, Kenneth J. Holroyd, Nicole M. Zaleski, Leslie J. Crofford, Andrea J. Pruijssers, David A. Edwards, Jana K. Shirey-Rice, Jill M. Pulley, Laura L. Dugan, David M. Aronoff, and Colleen M. Niswender
- Subjects
0301 basic medicine ,Clinical Trials as Topic ,Computer science ,Decision Making ,Drug Evaluation, Preclinical ,Drug Repositioning ,Representation (systemics) ,MEDLINE ,Translational research ,Context (language use) ,Multiple-criteria decision analysis ,Data science ,03 medical and health sciences ,Drug repositioning ,030104 developmental biology ,Models, Animal ,Perspective ,Drug Discovery ,Predictive power ,Animals ,Humans ,Molecular Medicine ,Repurposing - 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.
- Published
- 2017
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