helen naylor
Last active: 4/17/2019

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.

Pulley JM, Jerome RN, Zaleski NM, Shirey-Rice JK, Pruijssers AJ, Lavieri RR, Chettiar SN, Naylor HM, Aronoff DM, Edwards DA, Niswender CM, Dugan LL, Crofford LJ, Bernard GR, Holroyd KJ
Assay Drug Dev Technol. 2017 15 (8): 354-361

PMID: 29193979 · PMCID: PMC5731541 · DOI:10.1089/adt.2017.821

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.

MeSH Terms (7)

Animals Clinical Trials as Topic Decision Making Drug Evaluation, Preclinical Drug Repositioning Humans Models, Animal

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