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Using real-world evidence for label expansion: Use of palbociclib in male breast cancer patients


January 2022


Kraus, A.L., Yu-Kite, M., Mardekian, J., Cotter, M.J., Kim, S., Decembrino, J., Snow, T., Carson, K.R., Motyl Rockland, J., Gossai, A., Wilner, K., Wang, D.D., Huang Bartlett, C., Oharu, N., Schnell, P., VanArsdale, T., Lu, D.R. and Tursi, J.M. Real-World Data of Palbociclib in Combination With Endocrine Therapy for the Treatment of Metastatic Breast Cancer in Men. Clinical Pharmacology & Therapeutics. 2021

Our summary

Real-world evidence helped provide data of the use of palbociclib in males with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer. Analysis of de-identified claims data and de-identified EHR-derived Flatiron data on men with HR+, HER2- metastatic breast cancer treated in combination with palbociclib in the real world supported the FDA’s expansion of the palbociclib approval, previously only approved in women, to include men with HR+, HER2- metastatic breast cancer.

Why this matters

The advent of CDK inhibitors such as palbociclib has dramatically changed the way breast cancer is treated. However, the original approvals did not include male patients, an extremely rare patient population. The traditional standards of prospective clinical evidence would be nearly impossible to meet in order to secure a regulatory approval for male patients with breast cancer. This study shows how rigorous deployment of RWE could fit into the clinical evidence paradigm, complementing clinical trials where needed, to ultimately bring valuable therapeutic advances to patients in need.

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