Abstract
Brain metastases cause significant morbidity and mortality in patients with advanced cancer. In the era of precision oncology and immunotherapy, there are rapidly evolving systemic treatment options. These novel therapies may have variable intracranial efficacy, and patients with brain metastases remain a population of special interest. Typically, only patients with stable, asymptomatic and/or treated brain metastases are enrolled in clinical trials, or may be excluded altogether, particularly in the setting of leptomeningeal carcinomatosis. Consequently, this leads to significant concerns on the external validity of clinical trial evidence to real-world clinical practice. Here we describe the current trends in cancer clinical trial eligibility for patients with brain metastases in both early and late phase trials, with a focus on targeted and immunotherapies. We evaluate recent newly FDA approved therapies and the clinical trial evidence base leading to approval. This includes analysis of inclusion and exclusion criteria, requirements for baseline screening for brain metastases, surveillance cerebral imaging and incorporation of trial endpoints for patients with brain metastases. Finally, the use of alternative sources of data such as real-world evidence with registries and collaborative studies will be discussed.
Highlights
Brain or central nervous system (CNS) metastases remain a significant cause of morbidity and mortality in patients with advanced cancers [1]
We describe the current trends in cancer clinical trial eligibility for patients with brain metastases in both early and late phase trials, with a focus on recently approved targeted and immunotherapies
Key recommendations previously published by the American Society of Clinical Oncology (ASCO)–Friends of Cancer Research (FCR) Brain Metastases Working Group for the inclusion of patients with brain metastases in clinical trials to improve generalizability of trial evidence are considered [5]
Summary
Brain or central nervous system (CNS) metastases remain a significant cause of morbidity and mortality in patients with advanced cancers [1]. In the era of precision oncology and immunotherapy, there are rapidly evolving systemic treatment options for many cancers These novel therapies may have variable intracranial efficacy, and patients with brain metastases remain a population of special interest [3]. Only patients with stable, asymptomatic, and/or treated brain metastases are enrolled in clinical trials, or may be excluded altogether, in the setting of leptomeningeal carcinomatosis Key recommendations previously published by the American Society of Clinical Oncology (ASCO)–Friends of Cancer Research (FCR) Brain Metastases Working Group for the inclusion of patients with brain metastases in clinical trials to improve generalizability of trial evidence are considered [5] This includes an analysis of inclusion and exclusion criteria, requirements for baseline screening for brain metastases, surveillance cerebral imaging and incorporation of trial endpoints for patients with brain metastases. The use of alternative sources of evidence such as real-world evidence with registries and collaborative studies are discussed
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