CPN | Clinical Trials: Phase 1 defined – focusing on safety, with less certainty about benefit. Phase 1 trials for rare disease are designed for some efficacy

Clinical Trials: Phase 1 defined – focusing on safety, with less certainty about benefit. Phase 1 trials for rare disease are designed for some efficacy

In this video a Principal Investigator discusses the differences between phases of a trial. He explains that early phase trials are focused on safety as the primary endpoint where later phases trials have a greater focus on efficacy. He uses the terms “first in human”, “dose finding” and “risk/benefit balance”. He explains that with many trials for rare diseases, the phases may be combined.