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NCI Breast Cancer Working Group Report on Appropriate Endpoints for Clinical Trials in Metastatic Breast Cancer

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As reported in the Journal of Clinical Oncology by Seidman et al, the National Cancer Institute (NCI) Breast Cancer Steering Committee Working Group has issued a report on meaningful and appropriate endpoints for clinical trials in metastatic breast cancer.

With the goal of providing evidence-based consensus recommendations on choice of endpoints for clinical trials in metastatic breast cancer focusing on biologic subtype and line of therapy, the NCI convened a working group comprising breast medical oncologists, patient advocates, biostatisticians, and liaisons from the U.S. Food and Drug Administration to conduct a systematic review of the literature. External input was obtained from the pharmaceutic/biotechnology industry, real-world clinical data specialists, and experts in quality of life and patient-reported outcomes (PROs). The literature search yielded 146 publications to inform the working group recommendations.

Key Recommendations

Key recommendations for preferred clinical trial endpoints in metastatic disease based on assessment of magnitude of benefit representing meaningful clinical benefit are summarized/reproduced below.

  • Triple-negative breast cancer: Primary endpoints are overall survival (OS) for first-, second-, and ≥ third-line therapy. Secondary endpoints are response rate (RR) and PRO for all lines of therapy.
  • Hormone receptor (HR)–negative/HER2-positive disease: Primary endpoints are progression-free survival (PFS) and OS for all lines of therapy. Secondary endpoints are RR and PRO for all lines of therapy.
  • HR-positive/HER2-positive disease: Primary endpoints are PFS and OS for all lines of therapy. Secondary endpoints are RR and PRO for all lines of therapy.
  • HR-positive/HER2-negative disease: Primary endpoints are PFS for first and second lines of therapy and PFS and OS for ≥ third-line therapy. Secondary endpoints are OS, RR, and PRO for first and second lines of therapy and RR and PRO for ≥ third-line therapy.

The authors concluded, “Recommendations for appropriate endpoints for metastatic breast cancer clinical trials focus on biologic subtype and line of therapy and the magnitude of absolute and relative gains that would represent meaningful clinical benefit.”

The work was supported by the Coordinating Center for Clinical Trials, Office of the Director, National Cancer Institute.

Andrew D. Seidman, MD, of Memorial Sloan Kettering Cancer Center, is the corresponding author for the Journal of Clinical Oncology article.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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