Andrew D Seidman1, Louise Bordeleau1, Louis Fehrenbacher1, William E Barlow1, Jane Perlmutter1, Lawrence Rubinstein1, Suparna B Wedam1, Dawn L Hershman1, Jennifer Fallas Hayes1, Lynn Pearson Butler1, Mary Lou Smith1, Meredith M Regan1, Julia A Beaver1, Laleh Amiri-Kordestani1, Priya Rastogi1, Jo Anne Zujewski1, Larissa A Korde1. 1. Andrew D. Seidman, Memorial Sloan Kettering Cancer Center; Dawn L. Hershman, Columbia University, New York, NY; Louise Bordeleau, Hamilton Health Sciences and Canadian Cancer Trials Group, Hamilton, Ontario, Canada; Louis Fehrenbacher, Kaiser Permanente, Vallejo, CA; William E. Barlow, Cancer Research and Biostatistics, Seattle, WA; Jane Perlmutter, Gemini Group, Ann Arbor, MI; Lawrence Rubinstein, Jennifer Fallas Hayes, and Larissa A. Korde, National Cancer Institute; Lynn Pearson Butler, The Emmes Corporation, Rockville; Suparna B. Wedam, Julia A. Beaver, and Laleh Amiri-Kordestani, US Food and Drug Administration, Silver Spring; Jo Anne Zujewski, JZ Oncology, Bethesda, MD; Mary Lou Smith, Research Advocacy Network, Plano, TX; Meredith M. Regan, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA; and Priya Rastogi, University of Pittsburgh Medical Center, Pittsburgh, PA.
Abstract
PURPOSE: To provide evidence-based consensus recommendations on choice of end points for clinical trials in metastatic breast cancer, with a focus on biologic subtype and line of therapy. METHODS: The National Cancer Institute Breast Cancer Steering Committee convened a working group of breast medical oncologists, patient advocates, biostatisticians, and liaisons from the Food and Drug Administration to conduct a detailed curated systematic review of the literature, including original reports, reviews, and meta-analyses, to determine the current landscape of therapeutic options, recent clinical trial data, and natural history of four biologic subtypes of breast cancer. Ongoing clinical trials for metastatic breast cancer in each subtype also were reviewed from ClinicalTrials.gov for planned primary end points. External input was obtained from the pharmaceutic/biotechnology industry, real-world clinical data specialists, experts in quality of life and patient-reported outcomes, and combined metrics for assessing magnitude of clinical benefit. RESULTS: The literature search yielded 146 publications to inform the recommendations from the working group. CONCLUSION: Recommendations for appropriate end points 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.
PURPOSE: To provide evidence-based consensus recommendations on choice of end points for clinical trials in metastatic breast cancer, with a focus on biologic subtype and line of therapy. METHODS: The National Cancer Institute Breast Cancer Steering Committee convened a working group of breast medical oncologists, patient advocates, biostatisticians, and liaisons from the Food and Drug Administration to conduct a detailed curated systematic review of the literature, including original reports, reviews, and meta-analyses, to determine the current landscape of therapeutic options, recent clinical trial data, and natural history of four biologic subtypes of breast cancer. Ongoing clinical trials for metastatic breast cancer in each subtype also were reviewed from ClinicalTrials.gov for planned primary end points. External input was obtained from the pharmaceutic/biotechnology industry, real-world clinical data specialists, experts in quality of life and patient-reported outcomes, and combined metrics for assessing magnitude of clinical benefit. RESULTS: The literature search yielded 146 publications to inform the recommendations from the working group. CONCLUSION: Recommendations for appropriate end points 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.
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