Early-phase oncology clinical trial design in the era of molecularly targeted therapy: pitfalls and progress

Author(s): Penelope Bradbury, John Hilton , Lesley Seymour

Oncology drug development has seen a shift from the development of cytotoxic chemotherapy to that of molecularly targeted agents. Different mechanisms of action and toxicity profiles may mean that traditional oncology trial designs are no longer optimal for the development of these agents. Furthermore, the wealth of agents that are being developed, coupled with a constrained research environment has increasingly highlighted the need for efficient clinical trial design, both to filter agents as well as to advance promising agents rapidly into clinical development. We review the adaptations to traditional Phase I and II clinical trial design that are hoped to address some of the current challenges of drug development for oncology therapeutics.