TY - JOUR
T1 - National Cancer Institute Breast Cancer Steering Committee working group report on meaningful and appropriate end points for clinical trials in metastatic breast cancer
AU - Seidman, Andrew D.
AU - Bordeleau, Louise
AU - Fehrenbacher, Louis
AU - Barlow, William E.
AU - Perlmutter, Jane
AU - Rubinstein, Lawrence
AU - Wedam, Suparna B.
AU - Hershman, Dawn L.
AU - Fallas Hayes, Jennifer
AU - Pearson Butler, Lynn
AU - Smith, Mary Lou
AU - Regan, Meredith M.
AU - Beaver, Julia A.
AU - Amiri-Kordestani, Laleh
AU - Rastogi, Priya
AU - Zujewski, Jo Anne
AU - Korde, Larissa A.
N1 - Funding Information:
Supported by the Coordinating Center for Clinical Trials, Office of the Director, National Cancer Institute. The contributions of S.B.W., L.A.K., and J.A.B. represent their opinions and not those of the US FDA. We thank Allen Melemed, MD, MBA (Eli Lilly), and Maria Koehler, MD, PhD (Pfizer), for providing a pharmaceutic/biotechnology industry perspective. We also thank Lori Minasian, MD (National Cancer Institute), and Paul Kluetz, MD (US FDA), for valuable input about PROs. Finally, we thank Amy Abernethy, MD, PhD (Flatiron Health), for sharing a unique perspective on how real-world evidence and big data analyses may inform clinical trial design in the future.
Publisher Copyright:
Copyright © 2018 American Society of Clinical Oncology. All rights reserved.
PY - 2018/11/10
Y1 - 2018/11/10
N2 - 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.
AB - 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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U2 - 10.1200/JCO.18.00242
DO - 10.1200/JCO.18.00242
M3 - Article
C2 - 30212295
AN - SCOPUS:85056582667
SN - 0732-183X
VL - 36
SP - 3259
EP - 3268
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
IS - 32
ER -