The US Food and Drug Administration (FDA) published the draft guidance Monday and is seeking comments through October 26, 2019.
“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,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research (CDER).
Due to this rarity, men have historically not been included in clinical trials for breast cancer treatment, he noted, though this has led to a lack of data and treatment based on studies 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 labeling indications for men,” explained Pazdur in the announcement.
According to the FDA, the final recommendations will provide clarity as to how various trial designs using different data sources – including small-single arm trials and real-world data (RWD) – can support the treatment of male patients with breast cancer.
“Clinical trials are key to making progress against breast cancer, and we want everyone to benefit from the advances that are made. When clinical trials aren’t inclusive, it’s hard to tell if the resulting knowledge or breakthroughs will be applicable to all breast cancer patients," said Kimberly A. Sabelko, PhD, senior director, scientific strategy and programs, Susan G. Komen.
"That’s why it’s so important that clinical trials reflect the diversity of breast cancer patients, and why we support the FDA’s efforts to make breast cancer clinical trials more inclusive," she told us. "While rare, men do get breast cancer and their participation in clinical trials is critical for developing new treatments for male breast cancer.”
Developed by Novartis, Kisqali (ribociclib) was the first CDK4/6 inhibitor to be studied in men with advanced breast cancer, explained Novartis spokesperson Julie Masow.
“Male breast cancer is a rare disease, with approximately 2,550 new diagnoses and 480 deaths estimated in 2018, but according to the American Cancer Society the incidence of breast cancer in men has increased approximately 26% over the last 25 years,” said Masow speaking to the guidance publication.
The company’s open-label Phase IIIb CompLEEment-1 trial was designed to enroll a diverse group of patients “reflective of those seen in real-world settings, including men,” she added.
Said Masow, “Preliminary findings from the interim safety analysis were presented at ESMO 2018 showing the safety profile of Kisqali plus letrozole in men is consistent to that observed in women.”
Novartis also included men in its SOLAR-1 clinical trial protocol for Piqray (alpelisib), which was approved by the FDA in May as the first and only treatment specifically for postmenopausal women, and men, with HR+/HER2- advanced breast cancer with a PIK3CA mutation.
