Include men in clinical trials for breast cancer, stresses FDA in a draft guidance

USA: The U.S. Food and Drug Administration has stressed the inclusion of male patients in the clinical trials of breast cancer, in a recently released draft guidance, Male Breast Cancer: Developing Drugs for Treatment.

“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 the disease. 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. While some FDA-approved treatments are gender-neutral in their indication, many therapies are only approved for women and further data may be necessary to support labelling indications for men," Richard Pazdur, director of the FDA’s Oncology Center of Excellence, said in a press release.

"When finalized, the recommendations in the draft guidance will provide clarity for industry regarding how additional data to support efficacy and safety for male patients with breast cancer can be generated through a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," he continued. "We hope that the recommendations in the draft guidance issued today will, when finalized, encourage drug development for the treatment of male breast cancer and ultimately, provide additional FDA-approved treatment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 concluded.

Recommendations include:

• Eligibility criteria for clinical trials of breast cancer drugs should allow for the inclusion of both males and females
  
  
  
  • 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 males have not been included or when the inclusion of males is very limited in clinical trials for a specific breast cancer drug.
  
  
  
  • It may be possible to extrapolate findings to include male patients in the FDA approved indication for the drug where no difference in efficacy or safety is anticipated between males and females based on the mechanism of action of a drug. The use of extrapolation should be supported by data from earlier stages of development (e.g., nonclinical testing), literature, or both.
  
  
  • Further data may be necessary to support the extrapolation of findings to support an FDA-approved indication for male patients with breast cancer where there is a concern for differential efficacy or safety between males and females. In breast cancer, this may be relevant when a drug results in or relies upon manipulation of the hormonal axis, as with endocrine therapy. The 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 small-single arm trials and studies using real-world data sources.
  
  
  
  

SOURCE: Male Breast Cancer: Developing Drugs for Treatment