September 12, 2019

FDA recently released draft guidance that calls on researchers to include more male patients in clinical trials for breast cancer treatments in response to concerns that men are missing out on "lifesaving therapies," Roni Rabin reports for the New York Times.

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For men, breast cancer is a 'rare disease'

Among men, breast cancer is a rare disease. Fewer than 1% of breast cancer patients are male, according to FDA.

"Some men are not even aware they have breasts and not aware they can have breast cancer," according to Fatima Cardoso, director of the breast unit at Champalimaud Clinical Center in Lisbon. "Many, many oncologists have never seen a case of breast cancer in a male patient." 

As a result, men "get short shrift" when it comes to breast cancer treatment, Rabin writes.  

Male patients are often excluded from clinical trials of new drugs and treatments, meaning that when these treatments hit the market, there is little or no data to indicate whether the treatments are safe or effective for them. In some cases, new treatments are approved only for female patients, Rabin writes.

"It's so frustrating in clinic to see patients and say, 'Well, we don't really know—the drugs have been tested in women. We think it should work in men, but there's no real evidence to back that up,'" said Sharon Giordano, a professor of breast medical oncology at M.D. Anderson Cancer Center in Houston.

Compounding the problem is the fact that men with breast cancer tend to be diagnosed when the disease is in more advanced stages—in part because they don't suspect they have breast cancer, Rabin writes.

That means that male breast cancer patients are "often not getting optimal care and may be missing out on lifesaving therapies" at a critical time, Rabin writes.

For instance, a study of 1,500 male breast cancer patients published in Annals of Oncology in 2017 found that, of all male patients, most of the men whose cancer had not spread had tumors fueled by estrogen. However, only 77% of those patients had received anti-estrogen therapy, meaning one in five men did not receive the "potentially lifesaving therapy," Rabin writes.

FDA's recommendations

FDA's new draft guidance calls on researchers to include more male patients in clinical trials for breast cancer treatments, in what some breast cancer specialists are calling a "long overdue step," Rabin writes. The agency is accepting comments until Oct. 26.

In the guidance, FDA recommends that eligibility criteria for clinical trials for breast cancer drugs encourage inclusion of both male and female patients.

If sponsors propose to exclude male patients from a breast cancer trial, "scientific rationale should be included," according to FDA. However, the agency said it does not intend to consider "low expected accrual rates of male patients with breast cancer" as sufficient rationale for excluding male patients.

In the case that male patients are excluded from the trial or male enrollment is limited, FDA said, "It may be possible to extrapolate findings to include male patients in the FDA approved indication for the drug."

Discussion

While most experts support the proposed guidelines, some researchers are concerned that the trials for male breast cancer patients will still be too small.

"Some data is better than no data, but it's not the ultimate solution," said Larry Norton, chair of clinical oncology at Memorial Sloan Kettering Cancer Center.

Cardoso welcomed the new guidelines but added that even if researchers look to expand these trials, pharmaceutical companies may not be interested in funding them. "No one wants to invest in a disease that is only 1% of all breast cancers," she said (Rabin, New York Times, 9/9; FDA draft guidance, August 2019).

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