FDA Wants Men in Breast Cancer Clinical Trials: Draft Guidance Issued

Nick Mulcahy

August 26, 2019

The US Food and Drug Administration (FDA) today issued a draft guidance encouraging the inclusion of male patients in clinical trials of breast cancer drugs.

One of the recommendations is that eligibility criteria for clinical trials "should allow for inclusion of both males and females" and that, if men are excluded, there be an explicit scientific rationale.

Currently, patient advocates say that men are excluded from as many as two thirds of breast cancer clinical trials, on the basis of sex alone, as reported by Medscape Medical News.

Breast cancer is rare in males, with less than 1% of all cases occurring in men.

Men are not typically included in clinical trials for breast cancer treatment, which has led to a related lack of data. Men's treatments are generally based upon studies in women, said the FDA in a press statement.

"When finalized, the recommendations in the draft guidance will provide clarity for industry regarding how additional data to support efficacy and safety for male patients with breast cancer can be generated through a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," said Richard Pazdur, MD, acting director of the FDA's Office of Hematology and Oncology Products.

Some approved treatments are gender-neutral in their indication, but many therapies are only approved for women and further data may be necessary to support labeling indications for men, the agency pointed out.

The goal of the guidance is to provide additional FDA-approved treatment options for men.

Recently, the FDA extended the indication of the breast cancer drug palbociclib (Ibrance, Pfizer) to include male patients, based on "real world" data. Postmarketing reports and electronic health records showed that the safety profile for men treated with palbociclib was consistent with the safety profile for women.

The decision was also based on the acknowledgement that the majority of breast tumors in male patients express hormone receptors; such tumors are typically treated with endocrine-based therapies, which, in advanced cancers, may include palbociclib.

Call for Funding

Patient advocates have repeatedly called for more research funding for breast cancer in men.

One advocate, Oliver Bogler, PhD, of the University of New Mexico in Albuquerque, pointed out that breast cancer research grants from private and public funders in the United States between 2009 and early 2013 totaled $4.8 billion.

In 2016, using that data, Bogler asked major agencies/nonprofits that fund breast cancer research (National Institutes of Health and Susan G. Komen) to dole out money for men's research in proportion to their 1% incidence. "One percent of funding would be $14M per year, and represent a 20x increase," he said, as reported by Medscape Medical News.

"Men are excluded from two thirds of clinical trials on breast cancer based on sex alone, when perhaps in many instances they would be eligible based on their disease," Bogler noted at that time, citing a problem now addressed by the FDA guidance.

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