The U.S. Food and Drug Administration (FDA) has drafted a series of recommendations urging the inclusion of men in breast cancer clinical trials, aiming to broaden treatment options for these patients.

Men are rarely included in clinical trials assessing the effectiveness of medications to treat breast cancer because the incidence of this particular type of cancer in males is extremely low. However, by the time it is diagnosed, breast cancer in men is usually more advanced, and there are few treatment options.

The “Draft Guidance for Industry: Male Breast Cancer: Developing Drugs for Treatment,” is available here.

Comments on the draft guidance may be submitted online using the link available here. To ensure the FDA considers your comment before it begins work on the final version of the guidance, submit either online or written comments before the Oct. 26 close date. Written comments may be mailed to: Division of Dockets Management (HFA- 305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD, 20852. All written comments should be identified with the docket number FDA-2019-D-2966.

“Less than one percent of all breast cancer cases occur in men, but men are more likely to be diagnosed at an older age and have a more advanced stage of disease,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products (OHOP) in the FDA’s Center for Drug Evaluation and Research (CDER), said in a press release.

“As breast cancer in men is rare, they have typically not been included in clinical trials for breast cancer treatment. This has led to a lack of data, so their treatment is generally based upon studies and data collected in women,” Pazdur added.

This poses a problem, as many medications that have only been approved in women may require further testing and validation before being used to treat men. As a result, FDA-approved therapies currently available to treat men with breast cancer are very limited.

In an effort to reverse this, the FDA has drafted a series of recommendations to encourage the inclusion of men in clinical trials testing breast cancer therapies. These recommendations include the following:

• Trials in breast cancer should be designed in a way that ensures men are eligible candidates. The lower incidence of breast cancer in males should not be seen as an acceptable argument to exclude males from participating in clinical trials of breast cancer.
• If men were not included, or their participation was limited in a trial for a specific medication, the clinical findings may be extrapolated. That will allow men to have access to medication once it has been approved, provided that no gender-specific differences in drug efficacy or safety are anticipated.
• If more data is necessary to support the extrapolation of clinical findings obtained in women to men — for instance, in the case of medications that interfere with hormones, such as endocrine therapy — the additional data may be generated by resorting to smaller single-arm studies, or by using real-world data sources.

“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,” Pazdur said.

“We hope that the recommendations in the draft guidance issued today will, when finalized, encourage drug development for the treatment of male breast cancer and ultimately, provide additional FDA-approved treatment options for patients,” he added.