Using patient-reported outcomes in dose-finding oncology trials: surveys of key stakeholders and the National Cancer Research Institute Consumer Forum

Lai-Kwon Julia, Alyssa Vanderbeek, Anna Minchom, Olalekan Lee Aiyegbusi, Ogunleye Della, Richard Stephens, Melanie Calvert, Christina Yap

Research output: Contribution to journalArticlepeer-review

Abstract

Background
Patient-reported adverse events may be a useful adjunct for assessing a drug’s tolerability in dose-finding oncology trials (DFOT). We conducted surveys of international stakeholders and the National Cancer Research Institute (NCRI) Consumer Forum to understand attitudes about patient-reported outcome (PRO) use in DFOT.

Methods
A 35-question survey of clinicians, trial managers, statisticians, funders, and regulators of DFOT was distributed via professional bodies examining experience using PROs, benefits/barriers, and their potential role in defining tolerable doses. An 8-question survey of the NCRI Consumer Forum explored similar themes.

Results
International survey: 112 responses from 15 September–30 November 2020; 103 trialists [48 clinicians (42.9%), 38 statisticians (34.0%), 17 trial managers (15.2%)], 7 regulators (6.3%), 2 funders (1.8%)]. Most trialists had no experience designing (73, 70.9%), conducting (52, 50.5%), or reporting (88, 85.4%) PROs in DFOT. Most agreed that PROs could identify new toxicities (75, 67.0%) and provide data on the frequency (86, 76.8%) and duration (81, 72.3%) of toxicities. The top 3 barriers were lack of guidance regarding PRO selection (73/103, 70.9%), missing PRO data (71/103, 68.9%), and overburdening staff (68/103, 66.0%). NCRI survey: 57 responses on 21 March 2021. A total of 28 (49.1%) were willing to spend <15 min/day completing PROs. Most (55, 96.5%) preferred to complete PROs online. 61 (54.5%) trialists and 57 (100%) consumers agreed that patient-reported adverse events should be used to inform dose-escalation decisions.

Conclusion
Stakeholders reported minimal experience using PROs in DFOT but broadly supported their use. Guidelines are needed to standardize PRO selection, analysis, and reporting in DFOT.
Original languageEnglish
Article numberoyac117
Number of pages1
JournalThe Oncologist
DOIs
Publication statusPublished - 28 Jun 2022

Keywords

  • patient-reported outcomes
  • adverse events
  • drug development
  • cancer
  • quality of life
  • clinical trials

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