New Publication Recommends Phase 0 Approaches To Improve Human Drug Development

Study in a Sentence: Researchers recommend greater use of Phase 0 approaches that allow humans to be safely exposed to very low doses of drugs to examine drug effects as a way to improve the efficiency of drug development while reducing animal testing.

Healthy for Humans: Drug development is laborious and expensive, and the probability of success continues to decline. Phase 0 approaches, such as microdosing, are highly controlled human clinical trials that allow very low exposures to investigational medicines to help understand how a drug behaves in humans. This data better informs early decision-making about whether to proceed with developing the drug, and drugs that are likely to be unsafe for humans will not enter clinical trials at larger doses.

Redefining Research: Phase 0 solutions described in this publication have the potential to increase the efficiency of the translational process by providing human data earlier in the development pipeline, before many animal tests are required to be conducted. The article describes the expanding applications of Phase 0 approaches and opportunities to save animals from nonclinical studies.