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

Paul G. Kluetz, Bindu Kanapuru, Steven Lemery, Laura Lee Johnson, Mallorie H. Fiero, Karen Arscott, Yolanda Barbachano, Ethan Basch, Michelle Campbell, Joseph C. Cappelleri, David Cella, Charles Cleeland, Corneel Coens, Selena Daniels, Crystal S. Denlinger, Dianne L. Fairclough, James R. Hillard, Lori Minasian, Sandra A. Mitchell, Daniel O'ConnorSheetal Patel, Eric H. Rubin, Anna Ryden, Katherine Soltys, Rajeshwari Sridhara, Gita Thanarajasingam, Galina Velikova, Stephen Joel Coons

Research output: Contribution to journalArticlepeer-review

33 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)742-747
Number of pages6
JournalValue in Health
Issue number6
StatePublished - Jun 2018


  • drug safety
  • oncology
  • patient-reported outcomes
  • tolerability

ASJC Scopus subject areas

  • Health Policy
  • Public Health, Environmental and Occupational Health


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