Clinical trial design in brain metastasis: approaches for a unique patient population.

Clinical trials in brain metastases present challenges and opportunities unique to this patient population. With the increase in awareness and screening for brain metastases, smaller and often asymptomatic lesions are detected, creating the opportunity for trials of pre-irradiation chemotherapy. The goal of earlier intervention is advanced by studies to prevent brain metastases in high-risk populations. Sequencing of systemic chemotherapy with experimental chemotherapy in the context of a clinical trial requires collaboration between the investigators and the treating medical oncologists beginning ideally during design of the study. Adaptive randomization improves the efficiency of randomized trials in the brain metastasis population. Finally, collaborative efforts between patients and physicians with the support from patient advocacy groups will help advance the quality and the clinical trial options for patients with brain metastases.