National Cancer Institute Breast Cancer Steering Committee working group report on meaningful and appropriate end points for clinical trials in metastatic breast cancer Journal Article


Authors: Seidman, A. D.; Bordeleau, L.; Fehrenbacher, L.; Barlow, W. E.; Perlmutter, J.; Rubinstein, L.; Wedam, S. B.; Hershman, D. L.; Fallas Hayes, J.; Pearson Butler, L.; Smith, M. L.; Regan, M. M.; Beaver, J. A.; Amiri-Kordestani, L.; Rastogi, P.; Zujewski, J. A.; Korde, L. A.
Article Title: National Cancer Institute Breast Cancer Steering Committee working group report on meaningful and appropriate end points for clinical trials in metastatic breast cancer
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. Copyright © 2018 American Society of Clinical Oncology. All rights reserved.
Journal Title: Journal of Clinical Oncology
Volume: 36
Issue: 32
ISSN: 0732-183X
Publisher: American Society of Clinical Oncology  
Date Published: 2018-11-10
Start Page: 3259
End Page: 3268
Language: English
DOI: 10.1200/jco.18.00242
PROVIDER: scopus
PUBMED: 30212295
DOI/URL:
Notes: Article -- Export Date: 3 December 2018 -- Source: Scopus
Altmetric Score
MSK Authors
  1. Andrew D Seidman
    237 Seidman