FDA Calls for Men to be Enrolled in Breast Cancer Trials

Regulatory NewsRegulatory News
| 26 August 2019 | By Michael Mezher 

The US Food and Drug Administration (FDA) on Monday issued new draft guidance encouraging drugmakers to enroll male patients in breast cancer clinical trials.
While men account for less than one percent of breast cancer cases, FDA says they are more likely to be diagnosed at a later stage of disease and have historically been excluded from breast cancer clinical trials. The American Cancer Society estimates that some 268,600 women will be diagnosed with breast cancer in 2019 compared to just 2,670 men.
“This has led to a lack of data, so their treatment is generally based on studies and data collected in women,” said Richard Pazdur, director of FDA’s Oncology Center of Excellence. Pazdur noted that while some breast cancer treatments are approved for gender-neutral indications, “many therapies are only approved for women and further data may be necessary to support labeling indications for men.”
Specifically, the two-page draft guidance recommends that sponsors enroll both males and females in breast cancer clinical trials and suggests that extrapolation or additional data generation could be applied to support the use of breast cancer drugs and biologics in male patients when clinical trial data is lacking.
FDA says it encourages sponsors to discuss their clinical development plans for breast cancer therapies with the agency early on and that clinical trial eligibility criteria “should allow for inclusion of both males and females.”
FDA also says that it will not consider “low expected accrual rates of male patients” to be a justified scientific rationale for excluding male patients from breast cancer clinical trials.
In cases where male patients have not been included in clinical trials, or when data in male patients is limited, FDA says sponsors may be able to extrapolate findings from female patients “where no difference in efficacy or safety is anticipated between males and females based on mechanism of action of a drug.”
When there is a concern that safety or efficacy may be different between male and female patients, FDA says sponsors may be able to use data from small, single-arm trials and studies using real-world data to support extrapolation.
FDA, Draft Guidance, Federal Register Notice


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