USA – FDA finalizes guidance on studying CNS metastases in cancer trials

A final guidance document from the US Food and Drug Association (FDA) provides recommendations for drug developers on how best to evaluate the safety and efficacy of cancer drugs in patients with central nervous system (CNS) metastases. The document finalizes a draft guidance issued in August 2020.

« 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risk of death,” said Richard Pazdur, director of FDA’s Oncology Center of Excellence, in a statement issued when the draft guidance was released. At the time, Pazdur also noted that “few clinical trials” test cancer drugs in patients with CNS metastases, despite the fact that many cancers metastasize to the brain or other CNS sites.

“Metastatic cancer is a systemic disease; therefore, evaluation of the effectiveness of cancer drugs includes consideration of whether the cancer is controlled at all disease sites,” notes FDA in the guidance…