Recommendations to address respondent burden associated with patient-reported outcome assessment

The paper addresses the problem of respondent burden associated with completing patient-reported outcomes (PROs), defined as the perceived difficulty, time consumption or emotional stress of participation. While PROs provide vital patient-centered data for research, regulatory and clinical decisions, excessive burden can lead to poor completion rates, missing data and participant withdrawal, undermining data quality and representativeness. With no existing international guidance focused on minimizing respondent burden, the study’s objective was to develop consensus-based recommendations to help researchers and clinicians balance the value of PRO data with the burden imposed on patients across healthcare research and clinical practice.

An initial comprehensive literature review (published in 2022) informed the generation of candidate recommendations. PubMed was searched on 11/22/2021 without restrictions on study design or language. Two reviewers independently screened titles, abstracts and full texts. Qualitative data were analyzed using the framework method. The review identified key factors influencing respondent burden (including patient characteristics, measure features, mode and frequency of administration) and provided an evidence base for 26 initial candidate recommendations.

Study design followed COMET Handbook v1.0 guidance, overseen by a steering group. Ethical approval was obtained from the University of Birmingham Ethical Review Board (ERN_22-0276). The process included: 1) Generation of 26 candidate recommendations from the prior literature review. 2) Two-round international online Delphi survey (DelphiManager v5.0) among multi-stakeholder participants (trialists, PRO researchers/statisticians, patients/advocates, clinicians, journal editors, policymakers, industry and other PRO implementers). Round 1: 127 participants rated the importance of 26 recommendations on a 9-point scale (1–3 not important; 4–6 important but not critical; 7–9 important and critical) and provided qualitative feedback and new suggestions. Seven additional recommendations were added for Round 2 (total 33). Round 2: 106 participants (83% of Round 1) completed ratings; Round 1 responses were retained for those not completing Round 2. Pre-specified inclusion criteria required ≥70% of Round 2 participants rating 7–9 and ≤15% rating 1–3. Items meeting overall or stakeholder-group-specific thresholds were taken forward to a consensus meeting. 3) International consensus meeting (September 2023, online via Zoom) with 36 delegates (28 voters; 8 non-voting steering group/overlapping-institution experts) representing key stakeholder groups (12 trialists/academic researchers/statisticians, 7 industry, 6 regulators/policymakers, 5 healthcare professionals, 5 patients/advocates/public, 1 journal editor). Delegates discussed wording and inclusion of recommendations and voted anonymously via Zoom Poll (include/exclude/further discussion). Post-meeting, a final consultation sought comments on wording and approval of the final version. Through discussion and merging of overlapping items, the final consensus comprised 19 recommendations across three domains: rationale/schedule for assessment, measure selection and measure delivery.

• Process outcomes: 26 initial candidates from literature; 7 additional items proposed in Delphi Round 1; Round 1 participants n=127; Round 2 participants n=106 (83% of Round 1); consensus meeting delegates n=36 (28 voters). Pre-specified consensus threshold: ≥70% rating 7–9 and ≤15% rating 1–3 in Round 2. Final outcome: 19 recommendations after merging.
• Final 19 recommendations (organized by domain): Rationale and schedule for PRO assessment

1. Involve patients, clinicians and other stakeholders in formulating PRO research questions/clinical objectives to ensure relevance.
2. Balance respondent burden with the quantity and quality of required data.
3. Involve patients, clinicians and other stakeholders in decisions about assessment schedules and frequency. Measure selection
4. Review literature to identify relevant concepts of interest.
5. Use qualitative and quantitative methods to obtain input from patients and clinicians when selecting or developing fit-for-purpose measures.
6. Consider the complexity of measure format and instructions.
7. Consider respondents’ literacy levels.
8. Ensure cultural and linguistic relevance, with appropriate translation and linguistic validation.
9. Consider measure length and justify use of longer measures when appropriate.
10. When using multiple measures, avoid overlapping constructs.
11. Consider recall periods; longer timeframes may burden some respondents. Measure delivery
12. Ensure respondents understand why data are collected, who will access them and how they will be used.
13. Provide clear instructions, training and support for respondents.
14. Provide training/guidance for research staff and clinicians on PRO value and respondent burden.
15. Specify allowable support for respondents during completion.
16. Offer flexible modes of administration to meet needs, including underserved groups.
17. Where possible, consider ePROs to reduce burden, balanced with preferences and digital access.
18. In co-designing or modifying ePRO systems, involve patients and clinicians, including diverse target-population representatives.
19. Explore ePRO functionality with diverse target-population representatives (e.g., usability features, reminders, adaptive design).

• Additional data points and context: Evidence indicates measure length is not always associated with higher perceived burden; alignment with meaningful outcomes, clarity of purpose, appropriate scheduling and supportive delivery can improve completion and data quality.

The recommendations directly address the mechanisms by which respondent burden undermines PRO data quality, completeness and representativeness. By engaging stakeholders in defining meaningful objectives and assessment schedules, selecting relevant and comprehensible measures, and optimizing delivery (including training, support and flexible modes), the guidance seeks to maintain high-quality, inclusive PRO data while minimizing patient burden. The statement is intended for use by trialists, researchers, clinicians and healthcare providers and may inform protocol development, ethical/scientific review and clinical implementation. Although focused on adult PROs in research and clinical practice, the principles may extend to other contexts (e.g., pediatrics, patient-reported experience measures) with additional considerations. The recommendations complement existing resources and regulatory guidance and aim to promote equitable, inclusive PRO strategies.

This international Delphi and consensus effort produced 19 practical, consensus-based recommendations to minimize respondent burden in PRO assessments across healthcare research and clinical practice. Organized across rationale/scheduling, measure selection and delivery, these recommendations support stakeholder engagement, appropriate instrument and schedule choices, and inclusive, flexible implementation (including ePROs). Broad adoption may improve PRO completion, data quality and representativeness, thereby enhancing patient-centered decision-making. Future research should evaluate the impact of implementing these recommendations across different settings, populations and disease contexts and refine approaches for specific use cases.

• The initial evidence synthesis that generated candidate recommendations searched only PubMed, so relevant studies may have been missed and some candidate recommendations not identified; however, Delphi Round 1 allowed participants to suggest additional items (seven added).
• The recommendations focus on respondent burden from the patient perspective and do not address burden experienced by research or clinical staff; separate work is needed for these stakeholders.
• As with any consensus process, findings reflect participating stakeholder views and may not represent all perspectives globally.