August 26, 2020
The following quote is attributed to Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research:
“There are few effective treatments for patients with central nervous system (CNS) metastases – a type of cancer that has spread from the original tumor to the CNS and is associated with a higher risk of death. In addition, there are also few clinical trials testing therapies for CNS metastases. More attention is warranted to address this unmet medical need.
“The FDA has participated in efforts to facilitate drug development for patients with CNS metastases including hosting a public workshop and releasing draft guidance on clinical trial eligibility for brain metastases. However, clinical trial design for CNS metastases at large remains a challenge. Sponsors struggle with uncertainty regarding optimal study endpoints, lack of standardized response assessments, understanding how CNS metastases are evaluated in the context of the entire burden of metastatic disease to characterize a drug’s potential benefit, and interpreting radiographic response in the setting of recent radiation therapy or surgery.
“Today we are releasing draft guidance which, if finalized, would provide recommendations on evaluating cancer drugs in patients with CNS metastases. When finalized we hope this helps sponsors design clinical trials that improve the ability to understand the therapy’s benefit-risk profile across the patient population likely to use the drug. The development of therapeutic products for patients with CNS metastases is greatly needed.”
- Today, the U.S. Food and Drug Administration issued a draft guidance document, “Evaluating Cancer Drugs in Patients with Central Nervous System Metastases,” which, if finalized, would provide recommendations to sponsors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- The draft guidance, if finalized, would include recommendations for clinical trials of systemic anti-cancer drugs where patients with CNS metastases are included in the study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, today’s 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 .
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.