Adaptive Trial Designs To Advance Oncology Research

By Dirk Reitsma, M.D. and Scott Berry, Ph.D.

Advances in cancer therapy are seriously threatened by the escalating cost, time and numbers of patients required to conduct conventional oncology clinical trials. Oncology drug development consumes an average of seven years in clinical evaluation and yields a discouraging success rate—only 7 percent of oncology agents entering Phase I gain marketing approval. Oncology agents make up 25 percent of today’s research pipeline. To adequately evaluate these potential therapies, drug developers urgently need new research approaches. One of the most promising is the emerging practice of adaptive trial design.

Adaptive designs offer the means to make clinical trials more informative and efficient, advances that are urgently needed in oncology research. The benefits of adaptive designs attracted growing interest during the 2000s. However, these designs raise scientific and regulatory questions and their adoption by the biopharmaceutical industry has been slow. The FDA’s 2010 draft guidance, Adaptive Design Clinical Trials for Drugs and Biologics, encourages drug developers to expand their use of adaptive designs, and an ongoing collaboration among FDA, academia and industry is applying adaptive design in the I-SPY 2 breast cancer screening trial to streamline the identification of active drugs and predictive biomarkers. This groundbreaking trial is modeling a new adaptive approach to advance the clinical development process.