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

Research output: Contribution to journalArticlepeer-review

Authors

  • JM Giesinger
  • Fabio Efficace
  • Neil Aaronson
  • F Cottone
  • D Cella
  • EM Gamper

Colleges, School and Institutes

External organisations

  • Italian Group for Adult Hematologic Diseases (GIMEMA)
  • Health Outcomes Research Unit

Abstract

Objective: 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 tumour types (breast, lung, colorectal, prostate, bladder, and gynaecological cancers) and either published 2004-2018 or registered on clinicaltrials.gov and initiated 2014-2019. Frequency of use of individual PRO measures was assessed overall, over time, and by cancer site.
Results: Screening of 42,095 database records and 3,425 registered trials identified 480 published and 537 registered trials meeting inclusion criteria. Among published trials, the EORTC measures were used most often (54.8% of trials), followed by the FACIT measures (35.8%), the EQ-5D (10.2%), the SF-36 (7.3%), and the 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 PRO-CTCAE (2.2%), the 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, health care 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.

Details

Original languageEnglish
JournalValue in Health
Publication statusAccepted/In press - 7 Nov 2020