Considerations for Maximizing the Research Value of PRO Data Collected in Clinical Care

OBJECTIVES: Real-world evidence (RWE) plays an increasing role in drug development, regulatory review and reimbursement. Patient-Reported Outcomes (PROs) provide a unique perspective about how patients feel and function. Apart from generating RWE in prospective real-world studies, research can be informed by re-using PROs collected in clinical care primarily to facilitate individual patient management. In this concept paper we sought to identify challenges and opportunities for collecting research-quality PRO data in the clinical care context.

METHODS: We discuss the potential for secondary use of PRO data collected in routine clinical care and the key considerations for the generation of valuable research data. This discussion is presented using the PROTEUS Guide to Implementing Patient-Reported Outcomes in Clinical Practice as a framework to map.

RESULTS: Applications with significant potential for secondary use of PROs include: characterizing disease trajectories, burden of disease, or impact of PRO concepts and traditional clinical attributes on outcomes of care. Considerations for generation of valuable research data from PROs collected within clinical care include: PRO instrument selection, participation and engagement (motivation to report), minimization of burden to patients and healthcare professionals, collaboration with RWE end-users, PRO administration, collection and management (context of healthcare events, frequency of collection, linking data across sources).

CONCLUSIONS: Methods for collecting and using PRO data from clinical care to inform research are currently underdeveloped, with no guidance for considerations which would enhance its use in the research context. Setting standards for the secondary use of PRO data collected in healthcare settings would maximize its benefit. Cooperation between stakeholders responsible for implementing PROs and conducting RW evaluations is needed to maximize the rigour of collected data.