FDA Urges Inclusion of Males in Breast Cancer Studies

September 16, 2019

Because the disease in males is rare, they have historically been excluded from clinical trials of breast cancer drugs.

Breast cancer is rare in males, with less than 1% of all breast cancer cases occurring in male patients. Males are more likely to be diagnosed at an older age, with a more advanced stage of disease, and are more likely to have lymph node involvement compared to females with breast cancer, according to the FDA.

Because the disease in males is rare, they have historically been excluded from clinical trials of breast cancer drugs. This exclusion has resulted in limited FDA-approved treatment options for them. Treatment strategies for males with breast cancer are generally based on clinical experience and data from studies conducted in females with the disease.

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In August, the FDA issued a draft guidance document encouraging the inclusion of male patients in breast cancer clinical trials. Eligibility criteria for clinical trials of breast cancer drugs should allow for inclusion of both males and females, the document states. Scientific rationale should be included in the protocol when proposing to exclude males from breast cancer trials. FDA does not intend to consider low expected accrual rates of male patients with breast cancer to be a sufficient scientific rationale for excluding them from a clinical trial.

"When finalized, the recommendations in the draft guidance will provide clarity for industry regarding how additional data to support efficacy and safety for male patients with breast cancer can be generated through a variety of trial designs using different data sources, including studies using real-world data," according to a statement from Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.