International perspectives on suboptimal patient‐reported outcome trial design and reporting in cancer clinical trials: A qualitative study

Ameeta Retzer,Michael Brundage,Rachel M Taylor,Melanie Calvert,Khaled Ahmed,Fabio Efficace,Diana M Greenfield,Madeleine T King,Thomas Keeley,Julia M Brown,Adam W Glaser,Derek Kyte,Jo Armes,Anna Gavin,Anne Lanceley,Rebecca Mercieca‐Bebber,Galina Velikova,Lynn Calman

doi:10.1002/cam4.4111

Abstract

PurposeEvidence suggests that the patient‐reported outcome (PRO) content of cancer trial protocols is frequently inadequate and non‐reporting of PRO findings is widespread. This qualitative study examined the factors influencing suboptimal PRO protocol content, implementation, and reporting, and use of PRO data during clinical interactions.MethodsSemi‐structured interviews were conducted with four stakeholder groups: (1) trialists and chief investigators; (2) people with lived experience of cancer; (3) international experts in PRO cancer trial design; (4) journal editors, funding panelists, and regulatory agencies. Data were analyzed using directed thematic analysis with an iterative coding frame.ResultsForty‐four interviews were undertaken. Several factors were identified that could influenced effective integration of PROs into trials and subsequent findings. Participants described (1) late inclusion of PROs in trial design; (2) PROs being considered a lower priority outcome compared to survival; (3) trialists’ reluctance to collect or report PROs due to participant burden, missing data, and perceived reticence of journals to publish; (4) lack of staff training. Strategies to address these included training research personnel and improved communication with site staff and patients regarding the value of PROs. Examples of good practice were identified.ConclusionMisconceptions relating to PRO methodology and its use may undermine their planning, collection, and reporting. There is a role for funding, regulatory, methodological, and journalistic institutions to address perceptions around the value of PROs, their position within the trial outcomes hierarchy, that PRO training and guidance is available, signposted, and readily accessible, with accompanying measures to ensure compliance with international best practice guidelines.

Highlights

Patient-reported outcomes (PROs) enable the assessment of cancer, its impact, and treatment, from the patient's perspective, and are collected using validated self-reported questionnaires
There has to be some responsibility obviously on the patient, it has to be a shared task so improved tech partnership working is what is so important that the patient feels a part of the team and your data is incredibly valuable to us because it will inform research and clinical practice you know as an ongoing issue and it may be years down the line before you may see the benefit but you will be contributing to this so you are part of our team [009, Lived Experience]
I think it is another problem with patient-reported outcomes that you have these issues to deal with; the key items that everybody wants to know about or the key questionnaires and there is a whole raft of other things that you have collected

Summary

Introduction

Patient-reported outcomes (PROs) enable the assessment of cancer, its impact, and treatment, from the patient's perspective, and are collected using validated self-reported questionnaires. Patient-reported outcome data support informed decision- making by patients from diagnosis and throughout treatment.[2] Its value has been recognized by key stakeholders including clinicians, funders, regulators, and policy-m akers.3–5 the growing literature suggests that the quality of PRO data may. | 3 be compromised[6] through the omission of PRO-related content in trial protocols.[7,8] PRO data are often poorly reported9–11 or not included in trial publications.[12] Despite this evidence, a substantial, and growing, number of randomized controlled trials (RCTs) across all cancer types include PROs as primary and/or secondary endpoints.[13,14] Poor PRO protocol content and subsequent poor outcome reporting reduce the extent to which PRO results reach and inform clinical interactions and decision making, while simultaneously devaluing the contribution of trial participants providing this information. There is limited qualitative research exploring the factors affecting PRO trial design, data collection and reporting, or patients’ and clinicians’ access to PRO data to inform decision making

Objectives

Methods

Results

Discussion

Conclusion