FDA In Brief: FDA Works to Evaluate Cancer Therapies in Patients with Brain Metastases | FDA

FDA In Brief: FDA Works to Evaluate Cancer Therapies in Patients with Brain Metastases | FDA



FDA In Brief: FDA Works to Evaluate Cancer Therapies in Patients with Brain Metastases

The U.S. Food and Drug Administration issued a draft guidance document, “Evaluating Cancer Drugs in Patients with Central Nervous System Metastases,” which, if finalized, would provide recommendations to sponsors designing clinical trials of drugs and biological products that are intended to support product labeling describing anti-tumor (preventing or inhibiting the formation or growth of tumors) activity in patients with central nervous system (CNS) metastases from solid tumors originating outside the CNS.

The draft guidance, if finalized, would include recommendations for clinical trials of systemic anti-cancer drugs where patients with CNS metastases are included in the study population and for clinical trials conducted exclusively in patients with CNS metastases.

Also, if finalized, the draft guidance would provide sponsors with recommendations regarding the patient population, available therapy, prior therapies, assessment of CNS disease, study endpoints, and leptomeningeal disease (a rare complication of cancer in which the disease spreads from the original tumor site to the meninges surrounding the brain and spinal cord). Additionally, the draft guidance, if finalized, would explain that CNS metastases should be evaluated in the context of the entire disease burden and discuss how treatment effects may be described in drug labeling .