Using Patient-Reported Outcomes (PROs) in Dose-finding Oncology Trials (DFOT) – results from a global stakeholder survey and the National Cancer Research Institute (NCRI) Consumer Forum


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Session type:

Julia Lai-Kwon1, Christina Yap1, Alyssa Vanderbeek1, Anna Minchom1, Olalekan Lee Aiyegbusi2, Melanie Calvert2
1Institute of Cancer Research (ICR), 2University of Birmingham

Abstract

Background

Patient-reported adverse events may be a useful adjunct for assessing a drug’s tolerability in DFOT.  However, PRO use is limited and the reasons for this are unknown.  We conducted a global online stakeholder survey and a survey at the NCRI Consumer Forum to understand attitudes to PROs in DFOT.

Method

A 35-question survey of clinicians, trial managers, statisticians, funders and regulators of DFOT from hospitals, academia or industry was distributed via professional bodies. Questions examined prior experience of using PROs, benefits/barriers to PRO use and potential role in defining tolerable doses. An 8-question survey of the NCRI Consumer Forum explored similar themes with patients and carers. 

Results

Global survey: 112 responses from September-November 2020; 103 trialists [48(42.9%) clinicians, 38 (34.0%) statisticians , 17 (15.2%) trial managers], seven (6.3%) regulators , two (1.8%) funders.  Most trialists had no prior experience designing (73, 70.9%), conducting (52, 50.5%) or reporting (88, 85.4%) PROs in DFOT.  Most agreed PROs could identify new toxicities (75, 67.0%) and provide data on frequency (86, 76.8%) and duration (81, 72.3%) of toxicities.  The top three barriers were lack of guidance regarding PRO selection (73/103, 70.9%), missing PRO data (79/112, 70.5%), and overburdening staff (68/103, 66.0%).  Qualitative analysis of free-text responses will be presented.  

NCRI survey: 57 responses on 21/03/2021.  27 (48.2%) were willing to spend <15 minutes/day completing PROs.  Most (56, 98.2%) preferred to complete PROs online.   There was broad support for using PROs to inform selection of tolerable doses.   


 

Strongly agreed/agreed (n, %)

 

Global (n=112)

NCRI Consumer Forum (n=57)

PROs should be communicated in real-time to investigators

62 (55.3%)

54 (94.7%)

PROs on adverse events (with clinician- assessed CTCAE data) should be used to inform:

 

 

           Dose-escalation decisions

61 (54.5%)

57 (100%)

           Maximum tolerated dose

63 (56.3%)

Not-assessed

           Recommended phase 2 dose

76 (67.9%)

54 (94.7%)


Conclusion

Trialists and consumers reported minimal prior experience using PROs in DFOT but supported their use in defining tolerable doses. Guidelines are needed to standardise PRO selection, analysis and reporting in DFOT. 

Impact statement

This survey highlights there is high support for the use of PROs in DFOT and future guidance is required to increase its adoption.