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. 2022 Sep 2;27(9):768-777.
doi: 10.1093/oncolo/oyac117.

Using Patient-Reported Outcomes in Dose-Finding Oncology Trials: Surveys of Key Stakeholders and the National Cancer Research Institute Consumer Forum

Julia Lai-Kwon  1 , Alyssa M Vanderbeek  2 , Anna Minchom  1 , Olalekan Lee Aiyegbusi  3 , Della Ogunleye  4 , Richard Stephens  4 , Melanie Calvert  3   5   6 , Christina Yap  2
Affiliations

Using Patient-Reported Outcomes in Dose-Finding Oncology Trials: Surveys of Key Stakeholders and the National Cancer Research Institute Consumer Forum

Julia Lai-Kwon et al. Oncologist. .

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.

Keywords: adverse events; cancer; clinical trials; drug development; patient-reported outcomes; quality of life.

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Figures

Figure 1.
Figure 1.
Attitudes toward PROs for dose–escalation decisions: (a) By work role and (b) by number of years of work experience.
Figure 2.
Figure 2.
NCRI consumer forum—attitudes toward using PROs in dose-finding oncology trials.
Figure 3.
Figure 3.
NCRI consumer forum—a consumer perspective (Della Ogunleye and Richard Stephens).
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