Although breast cancer most commonly occurs in women, approximately 2500 men are diagnosed with breast cancer each year in the United States alone. Unfortunately, men have historically been excluded from clinical trials for breast cancer treatments, resulting in limited data on treatment safety and efficacy in men. Despite limited data, breast cancer in men is managed similarly to how it is managed in women. On August 26, 2019, the US Food and Drug Administration (FDA) recognized this shortfall and released draft guidance encouraging inclusion of men in breast cancer clinical trials. While this guidance is currently in draft form, the ultimate goal is to provide a framework to support generating additional data on breast cancer treatment in men by leveraging nontraditional data sources, such as real-world data, to support labeling of breast cancer treatments for men.
High Altitude: Historically, the exclusion of men from clinical trials has meant that many breast cancer treatments are restricted to use in women and are either not available for men or must be used off-label. The FDA statement opens the door to potential label expansions on the basis of data outside the randomized controlled clinical trial setting. The guidance, once finalized, will be of great interest not only to any clinicians involved in clinical trial design and execution, but also to those who have the capability to generate observational and chart-review data for men with breast cancer. Most importantly, men suffering from breast cancer stand to receive the greatest benefit.
Ground Level: While less common than breast cancer in women, male breast cancer is a challenge to treat, with limited data on treatment efficacy and safety in men. The FDA is encouraging additional research on men with breast cancer, to provide additional data regarding the treatment of these patients. This may present an opportunity for community physicians to publish observational or retrospective cohort and case studies to support this unmet need.