Past and Current Practice of Patient-Reported Outcome Measurement in Randomized Cancer Clinical Trials: A Systematic Review

Value Health. 2021 Apr;24(4):585-591. doi: 10.1016/j.jval.2020.11.004. Epub 2021 Feb 10.


Objectives: In our systematic review, we assessed past and current practice of patient-reported outcome (PRO) measurement in cancer randomized, controlled trials (RCTs).

Methods: We included RCTs with PRO endpoints evaluating conventional medical treatments, conducted in patients with the most prevalent solid tumor types (breast, lung, colorectal, prostate, bladder, and gynecological cancers) and either published in 2004 to 2018 or registered on and initiated in 2014 to 2019. Frequency of use of individual PRO measures was assessed overall, over time, and by cancer site.

Results: Screening of 42 095 database records and 3425 registered trials identified 480 published and 537 registered trials meeting inclusion criteria. Among published trials, the European Organisation for Research and Treatment of Cancer (EORTC) measures were used most often (54.8% of trials), followed by the Functional Assessment of Chronic Illness Therapy (FACIT) measures (35.8%), the EQ-5D (10.2%), the SF-36 (7.3%), and the MD Anderson Symptom Inventory (MDASI; 2.5%). Among registered trials, the EORTC measures were used in 66.1% of the trials, followed by the FACIT measures (25.9%), the EQ-5D (23.1%), the SF-36 (4.8%), the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE; 2.2%), the Patient-Reported Outcomes Measurement Information System (PROMIS) measures (1.7%), and the MDASI measures (1.1%).

Conclusion: The PRO measures most frequently used in RCTs identified in our review differ substantially in terms of content and domains, reflecting the ongoing debate among the scientific community, healthcare providers, and regulators on the type of PRO to be measured. Current findings may contribute to better informing the development of an internationally agreed core outcome set for future cancer trials.

Keywords: cancer; patient-reported outcome; questionnaire; randomized-controlled trial; systematic review.

Publication types

  • Comparative Study
  • Systematic Review

MeSH terms

  • Humans
  • Neoplasms / psychology*
  • Neoplasms / therapy*
  • Patient Reported Outcome Measures*
  • Quality of Life*
  • Randomized Controlled Trials as Topic
  • Surveys and Questionnaires