FDA: Include premenopausal women in breast cancer trials

Premenopausal women should be considered for inclusion in breast cancer clinical trials, according to new draft guidance from the US Food and Drug Administration (FDA). “Menopausal status should not be the basis of exclusion from any breast cancer clinical trial,” says the draft guidance, which was made available for a 60-day consultation period on 7 October.
 
The draft guidance puts forward recommendations to help move clinical breast cancer research away from the historical practice of commonly excluding premenopausal women from clinical trials.
 
"Today, the U.S. Food and Drug Administration issued draft guidance encouraging the inclusion of premenopausal women in breast cancer clinical trials that investigate the efficacy of hormonal drug and biological products,” said Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence, in announcing the availability of the draft guidance.
 
“When finalized, the guidance will provide recommendations for industry to generate additional data that will support the efficacy and safety of drugs and biologics for premenopausal women with breast cancer,” said Pazdur, who is also acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research (CDER).
 
Previously, some sponsors of drugs to treat hormone receptor (HR)-positive breast cancer have run completely separate clinical trials for premenopausal women with HR-positive cancer, delaying when these hormonal treatments would be available to premenopausal women.
 
Pazdur noted that the guidance observes that premenopausal women who have sufficient estrogen suppression should see efficacy and safety similar to that seen in postmenopausal women. “Therefore, premenopausal women should be included in these trials,” he said.  
 
The guidance specifically recommends equal inclusion of premenopausal women with adequate estrogen suppression in clinical trials for drugs or combinations that manipulate the hormonal axis. Also, the guidance points out that menopausal status does not affect the efficacy of chemotherapy, immunotherapy and targeted therapy.
 
However, the guidance acknowledges that safety and efficacy considerations may still play into a decision to stratify the randomization of trial enrollees by menopausal status. The guidance specifically excludes issues of fertility and fertility preservation from the scope of issues it addresses.
 
Earlier this year, FDA also finalized a guidance document that addresses developing drugs to treat male breast cancer. (RELATED: FDA finalizes recommendations for enrolling males in breast cancer trials, Regulatory Focus 12 August 2020)
 
 
  
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