Phase 0 clinical trial- an overview

Drug discovery begins in the laboratory with target identification and validation followed by pre-clinical and clinical development. The entire process takes around 10 to 15 years. It is associated with high costs and a high rate of failure. The probability of a drug going beyond Phase I testing is quite low. The quest for discovering anti-cancer agents has shifted from non-specific chemotherapeutic agents to a specific molecular target-based approach. Drug development processes for various drugs need to be re-evaluated. Phase 0 clinical trials act as a novel tool to hasten and improve the drug development from laboratory to clinic. Phase 0 studies enable go versus no-go decisions for a new drug early in its development process. The administration of a single sub-therapeutic dose provides preliminary data on the pharmacokinetics of the drug. It helps in confirming whether the drug behaves in humans as was predicted by pre-clinical studies. Notwithstanding, Phase 0 clinical studies are associated with some disadvantages. This article describes about Phase 0- its rationale, conduct, potential benefits and limitations. Key Words: Drug development, phase 0, clinical trial