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Informing the Tolerability of Cancer Treatments Using Patient-Reported Outcome Measures: Summary of an FDA and Critical Path Institute Workshop.

  • Kluetz, Paul G1
  • Kanapuru, Bindu2
  • Lemery, Steven2
  • Johnson, Laura Lee2
  • Fiero, Mallorie H2
  • Arscott, Karen3
  • Barbachano, Yolanda4
  • Basch, Ethan5
  • Campbell, Michelle2
  • Cappelleri, Joseph C6
  • Cella, David7
  • Cleeland, Charles8
  • Coens, Corneel9
  • Daniels, Selena2
  • Denlinger, Crystal S10
  • Fairclough, Dianne L11
  • Hillard, James R12
  • Minasian, Lori13
  • Mitchell, Sandra A13
  • O'Connor, Daniel4
  • And 8 more
  • 1 Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, USA. Electronic address: [email protected]
  • 2 Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
  • 3 Geisinger Commonwealth School of Medicine, Scranton, PA, USA.
  • 4 Medicines and Healthcare Products Regulatory Agency, Victoria, London, UK.
  • 5 Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC, USA.
  • 6 Pfizer Inc., Groton, CT, USA.
  • 7 Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL, USA.
  • 8 Department of Symptom Research, Division of Internal Medicine, UT MD Anderson Cancer Center, Houston, TX, USA.
  • 9 European Organisation for Research and Treatment of Cancer (EORTC) HQ, Quality of Life Department, Brussels, Belgium. , (Belgium)
  • 10 Fox Chase Cancer Center, Philadelphia, PA, USA.
  • 11 Department of Biostatistics and Informatics, Colorado School of Public Health, Aurora, CO, USA.
  • 12 Patient Representative, East Lansing, MI, USA.
  • 13 National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
  • 14 Genentech, South San Francisco, CA, USA.
  • 15 Merck Research Laboratories, Merck & Co., Kenilworth, NJ, USA.
  • 16 AstraZeneca, Gothenburg, Sweden. , (Sweden)
  • 17 Therapeutic Products Directorate, Health Products and Food Branch, Health Canada, Ottawa, ON, Canada. , (Canada)
  • 18 Division of Hematology, Mayo Clinic, Rochester, MN, USA.
  • 19 Leeds Institute of Cancer and Pathology, University of Leeds, St James's Institute of Oncology, Leeds, UK.
  • 20 Patient-Reported Outcome Consortium, Critical Path Institute, Tucson, AZ, USA.
Published Article
Value in Health
Publication Date
Jun 01, 2018
DOI: 10.1016/j.jval.2017.09.009
PMID: 29909880


The US Food and Drug Administration and the Critical Path Institute's Patient-Reported Outcome (PRO) Consortium convened a cosponsored workshop on the use of PRO measures to inform the assessment of safety and tolerability in cancer clinical trials. A broad array of international stakeholders involved in oncology drug development and PRO measurement science provided perspectives on the role of PRO measures to provide complementary clinical data on the symptomatic side effects of anticancer agents. Speakers and panelists explored the utility of information derived from existing and emerging PRO measures, focusing on the PRO version of the National Cancer Institute's Common Terminology Criteria for Adverse Events. Panelists and speakers discussed potential ways to improve the collection, analysis, and presentation of PRO data describing symptomatic adverse events to support drug development and better inform regulatory and treatment decisions. Workshop participants concluded the day with a discussion of possible approaches to the patient-reported assessment of an investigational drug's overall side effect burden as a potential clinical trial end point. The Food and Drug Administration reiterated its commitment to collaborate with international drug development stakeholders to identify rigorous methods to incorporate the patient perspective into the development of cancer therapeutics. Copyright © 2018 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.

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