Past and current practice of patient-reported outcome measurement in randomized cancer clinical trials: a systematic review

Johannes M. Giesinger, Fabio Efficace, Neil Aaronson, Melanie Calvert, Derek Kyte, Francesco Cottone, David Cella, Eva-Maria Gamper

Research output: Contribution to journalReview articlepeer-review


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.

Original languageEnglish
Pages (from-to)585-591
Number of pages7
JournalValue in Health
Issue number4
Early online date10 Feb 2021
Publication statusPublished - Apr 2021

Bibliographical note

Conflict of Interest Disclosures: Drs Giesinger and Gamper reported receiving a research grant from the European Organisation for Research and Treatment of Cancer (EORTC) for an unrelated project. Dr Efficace reported receiving personal fees from Abbvie, Bristol Myers Squibb, Orsenix, and Takeda outside the submitted work; grants and personal fees from Amgen outside the submitted work; and a grant from the EORTC Quality of Life Group outside the submitted work. Dr Calvert is a National Institute for Health Research (NIHR) Senior Investigator and reported receiving grants from the NIHR, Health Data Research UK, Macmillan Cancer Support, UCB Pharma, and Innovate UK outside the submitted work; and personal fees from Astellas, Takeda, Merck, GlaxoSmithKline, Daiichi Sankyo, and Glaukos outside the submitted work. Dr Kyte reported receiving grants from NIHR, Innovate UK, NIHR Birmingham Biomedical Research Centre, NIHR Surgical Reconstruction and Microbiology Research Centre at the University of Birmingham, University Hospitals Birmingham National Health Service Foundation Trust, the Macmillan Cancer Support, and Kidney Research UK outside the submitted work; and personal fees from Merck and GlaxoSmithKline outside the submitted work. Dr Cella is the president of No other disclosures were reported.

Funding/Support: The authors received no financial support for this research.

Acknowledgment: The views expressed in this article are those of the authors and not necessarily those of the NIHR, NHS or the Department of Health and Social Care.

Funding Information:
Of the 537 registered trials, 278 (51.8%) trials were funded without industry involvement, and 259 (48.2%) were funded, at least in part, by industry. The EORTC measures were used in 59.7% of the non–industry-funded trials and 73.0% of the trials with industry funding. For the other PRO measures this ratio was as follows: FACIT measures 27.7% (non-industry) versus 23.9% (industry), SF-36 7.6% (non-industry) versus 1.9% (industry), EQ-5D 22.3% (non-industry) versus 23.9% (industry), PROMIS 2.9% (non-industry) versus 0.4% (industry), PRO-CTCAE 2.9% (non-industry) versus 1.5% (industry), and MDASI 0.4% (non-industry) versus 1.9% (industry). For the EORTC QLQ-C30 additional questionnaire modules were used in 63.3% of non-industry trials and 59.8% of industry-funded trials. The FACT-G was supplemented in 94.8% of non-industry trials and in 95% of industry-funded trials. For those trials with industry involvement, the type of sponsor and funding source were congruent for all but 5 trials, and thus no additional analysis was conducted by sponsor.

Publisher Copyright:
© 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research


  • cancer
  • patient-reported outcome
  • questionnaire
  • randomized-controlled trial
  • systematic review


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