Konrad Maruszczyk1, Olalekan Lee Aiyegbusi1-4 , Thomas Keeley1,5 , Melanie J. Calvert1-4 

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 NIHR Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK
5 GlaxoSmithKline (GSK), Patient Centered Outcomes, Value Evidence and Outcomes, Brentford, UK

PROs are increasingly collected as part of real world (RW) studies. They provide valuable information on the long-term effectiveness and tolerability of therapeutics from the patient’s perspective. Data collected outside the constraints of randomised clinical trials (RCTs) should be more generalisable to real world target patient populations. Moreover, sourcing data as part of routine care provision allows for reaching patient groups that are typically hesitant to participate in research. Nevertheless, collecting PROs in this setting poses many challenges to RW researchers. Varying levels of PRO expertise within functions that are accountable for delivery of RWE studies might be one of the barriers that hampers their successful implementation.

In 2021, Konrad Maruszczyk began his PhD programme at the University of Birmingham, which focused on understanding the use of PROs in RWE research. One of the first steps of this PhD project was to identify available guidance facilitating the collection and use of real world patient-reported outcomes (RW-PROs) for regulatory, reimbursement and health policy decision-making. As a result, we published a systematic review on this topic in the Journal of Patient-Reported Outcomes (JPRO), which received the 2023 ISOQOL Outstanding Article of the Year Award for JPRO. The full article is available here.

Our review showed that current guidance is fragmented and no international guidelines directly address the use of PROs in RWE generation. The RWE space is characterised by substantial data sources and study design heterogeneity. Thus, future RW-PRO guidelines should be tailored to the specific type of studies or context for its use. Additionally, all relevant stakeholders should be considered to inform guideline development, including researchers, clinicians, patient groups, regulators, payers, and industry.

RWE is a rapidly evolving field, and since the publication of our work, various publications that provide some level of relevant recommendations have become available. Some guidance documents focusing on various aspects of RWE were published recently, and their transferability to RW-PROs should be investigated. These documents are linked below for further reading. Nevertheless, particular attention should be drawn to guidance for PRO collection as part of registries, such as this article published in BMC Health Services Research, and the PROTEUS Clinical Practice PRO Guide.

The limited availability of guidance and success stories depicting best practices for collecting and utilising RW-PROs indicates that further work is needed. Joint efforts of the broad scientific community are crucial to advance the field. With time, broadly accepted standards for using RW-PROs should emerge. Regulatory bodies and payers could incorporate some of this learning into guidance documents. This, in turn, should de-risk the industry’s investments in conducting these types of studies, as they would know what requirements must be fulfilled to produce regulatory-grade data.

One of the initiatives aiming to advance the field is the recently accepted ISPOR Task Force. It seeks to identify emerging good practices using PROs in prospective RW study design and will be informed by this research project. Initiatives such as this will help address challenges associated with different RW-PRO use cases.

Disclaimer: The Centre for Patient Reported Outcome Research at the University of Birmingham has an ongoing collaboration with GSK aimed at investigating the current and future use of patient-reported outcomes for real world evidence (RWE) generation.

Additional Resources

  1. NICE real-world evidence framework
  2. Guideline on Using Real-World Data to Generate Real-World Evidence” from the National Medical Products Administration (NMPA)
  3. Guideline on registry-based studies” from the European Medicines Agency (EMA)
  4. Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products” from the US Food and Drug Administration (FDA)
  5. Guidance for Reporting Real-World Evidence” from Canada’s Drug and Health Technology Agency (CADTH)

This newsletter editorial represents the views of the author and does not necessarily reflect the views of ISOQOL. 

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