OBJECTIVES: Real-world evidence (RWE) generation can be enhanced by collecting and using patient-reported outcomes (PROs), which can provide valuable information on the long-term effectiveness, safety, and tolerability of health interventions from the patient perspective. Methods for collecting and using this data type in the real-world setting are currently underdeveloped. There is a lack of international guidance specific to RWE generation in the context of use for regulatory decision-making, reimbursement, and health policy. This study aimed to explore stakeholder perspectives on the current use, barriers and facilitators, and future opportunities for the use of PROs in RWE.

METHODS: One-to-one, online semi-structured interviews were conducted with international stakeholders: patients, regulators, payers, clinicians, academic researchers and individuals employed by the pharmaceutical industry or consultancies specialising in RWE. Interviews were transcribed verbatim and analysed thematically with QSR NVivo 20 based on the Consolidated Framework for Implementation Research (CFIR). To date, 16 interviews have been conducted, including seven academic researchers, four regulators, three pharma industry or consultancy employees, one patient advocate and one payer.

RESULTS: Interviewees acknowledged the potential value of PRO collection and utilisation for RWE generation. Perceived benefits included providing data from the patient perspective on real world effectiveness and tolerability of treatment from the diverse populations. The most frequently mentioned barriers that hamper full implementation of PROs in RWE generation included: the limited collection of PROs in routine care, the burden on clinicians and patients, lack of consensus about methods for data analysis and exemplars of successful implementation.

CONCLUSIONS: The use of PROs in RWE generation is in its infancy. Setting standards for PRO data collection, analysis, and use in the real world would maximise its benefits. The needs of various stakeholder groups (including patients, health care professionals, regulators, payers, and industry) should be considered when developing future guidelines.