The FDA announced draft guidance, which provides recommendations for the design of clinical trials and biological products for product labeling for those with brain metastases regulated by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research.
The FDA announced draft guidance called “Evaluating Cancer Drugs in Patients with Central Nervous System Metastases” which provides recommendations for the design of clinical trials and biological products for product labeling for those with brain metastases regulated by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research.
Within the recommendations, the FDA covers assessment for central nervous system (CNS) disease, study end points, available therapy, prior therapies, and leptomeningeal disease—a rare complication of cancer where the disease spreads from the original tumor site to the meninges surrounding the brain and spinal cord.
“There are few effective treatments for patients with 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,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement. “In addition, there are also few clinical trials testing therapies for CNS metastases. More attention is warranted to address this unmet medical need.”
If the draft guidance is finalized, it would not only offer sponsors the recommendations, but also review how treatment effects would be described in drug labeling and explain
that these brain metastases should be evaluated in the context of the entire disease burden.
Pazdur, who is also acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said the FDA joined 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to “facilitate drug development for patients with CNS metastases including hosting a public workshop and releasing draft guidance on clinical trial eligibility for brain metastases.”
The workshop, which was held in March 2019, was intended to provide a place for open discussion between the member of the FDA, physicians, researchers, industry in the setting of neuro-oncology product development for clinical trials of these patients, and patient advocates. It was also for educating researchers and product developers on regulatory science and policy issues related to this topic, accelerating development of products for treating brain metastases, allowing an open discussion between major stakeholders in the field, and identifying end points for clinical trials.
“…Clinical trial design for CNS metastases at large remains a challenge. Sponsors struggle with uncertainty regarding optimal study end points, 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,” he said.
There is significant morbidity and mortality associated with CNS metastases in patients with cancer, and development of treatment is needed to change the landscape for them.
“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,” Pazdur concluded.
FDA in brief: FDA works to evaluate cancer therapies in patients with brain metastases. News release. FDA. August 26, 2020. Accessed August 28, 2020. https://bit.ly/3jt1yQ3