Novel Clinical Trial Designs in Neuro-Oncology

Scientific and technologic advances have led to a boon of candidate therapeutics for patients with malignancies of the central nervous system. The path from drug development to clinical use has generally followed a regimented order of sequential clinical trial phases. The recent increase in novel therapies, however, has strained the regulatory process and unearthed limitations of the current system, including significant cost, prolonged development time, and difficulties in testing therapies for rarer tumors. Novel clinical trial designs have emerged to increase efficiencies in clinical trial conduct to better evaluate and bring impactful drugs to patients in a timely manner. In order to better capture meaningful benefits for brain tumor patients, new endpoints to complement or replace traditional endpoints are also an increasingly important consideration. This review will explore the current challenges in the current clinical trial landscape and discuss novel clinical trial concepts, including consideration of limitations and risks of novel trial designs, within the context of neuro-oncology.