Paving the way for patient centricity in real-world evidence (RWE): Qualitative interviews to identify considerations for wider implementation of patient-reported outcomes in RWE generation

Konrad Maruszczyk; Christel McMullan; Olalekan Lee Aiyegbusi; Thomas Keeley; Roger Wilson; Philip Collis; Catherine Bottomley; Melanie J Calvert

doi:10.1016/j.heliyon.2023.e20157

Paving the way for patient centricity in real-world evidence (RWE): Qualitative interviews to identify considerations for wider implementation of patient-reported outcomes in RWE generation

Heliyon. 2023 Sep 14;9(9):e20157. doi: 10.1016/j.heliyon.2023.e20157. eCollection 2023 Sep.

Authors

Konrad Maruszczyk¹, Christel McMullan^{1

2}, Olalekan Lee Aiyegbusi^{1

3}, Thomas Keeley^{1

4}, Roger Wilson^{1

5}, Philip Collis^{1

5}, Catherine Bottomley⁶, Melanie J Calvert^{1

7}

Affiliations

¹ Centre for Patient Reported Outcome Research and Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.
² NIHR Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK.
³ NIHR Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK.
⁴ GlaxoSmithKline (GSK), Patient Centered Outcomes, Value Evidence and Outcomes, Brentford, UK.
⁵ Patient partner, UK.
⁶ Vitaccess Ltd., Oxford, UK.
⁷ NIHR Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK.

Abstract

Objectives: Real-world evidence (RWE) generation can be enhanced by including patient-reported outcomes (PROs). Methods for collecting and using PRO data in the real-world setting are currently underdeveloped and there is no international guidance specific to its use in this context. This study explored stakeholders' perspectives and needs for using PROs in RWE generation. Barriers, facilitators, and opportunities for wider use of PROs in real-world studies were also investigated.

Methods: Online semi-structured interviews were conducted with international stakeholders: patients, patient advocates, regulators, payers, clinicians, academic researchers, and industry experts. Interviews were recorded, transcribed verbatim and analysed using NVivo 20. Thematic analysis was conducted based on the updated Consolidated Framework for Implementation Research (CFIR).

Results: Twenty-three interviews were conducted. Participants confirmed that the use of PROs in RWE generation is not yet well established. Participants expressed a mixed level of confidence in the value of PROs collected in a real-world setting. Operational challenges associated with collecting routine PRO data to inform care delivery at the individual level (e.g., setting up infrastructure) need to be addressed. Methodological and other challenges (e.g., financing research) associated with collecting prospective de novo data in a real-world setting should be considered to facilitate PRO utilisation in real-world studies.

Conclusions: Several opportunities and challenges were identified regarding the broader use of PROs in RWE research. Joint efforts from different stakeholders are needed to maximise PRO implementation, with consideration given to each stakeholders' specific needs (e.g., by developing good practices).