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Harnessing the patient voice in real-world evidence: the essential role of patient-reported outcomes

Real-world evidence is increasingly valued by regulators and payers. Central to this evidence base is patient-reported outcome data describing the impact of drugs on quality of life, daily activities and symptoms. Here, we highlight key challenges with current real-world, patient-reported outcome data and describe collaborative next steps for international stakeholders to overcome these issues.
  1. Melanie J. Calvert

    1. Centre for Patient Reported Outcome Research (CPROR), Institute of Applied Health Research, and the National Institute for Health Research (NIHR), Birmingham Biomedical Research Centre, University of Birmingham, UK.

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  2. Daniel J. O’Connor

    1. Medicines and Healthcare products Regulatory Agency (MHRA), London, UK.

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  3. Ethan M. Basch

    1. Division of Hematology–Oncology, University of North Carolina, Chapel Hill, NC, USA.

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Data sources for real-world evidence include electronic health records, insurance claims and billing, registries or on-site medical chart review. However, information about how patients feel and function, as captured directly from patients themselves, is often missing1. As pressures from legislation, international regulatory authorities and patient groups promote more patient-centric drug development and evidence generation2, attention is now turning to the role of patient-reported outcomes (PROs) to provide the patient perspective in real-world data sets.

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Nature Reviews Drug Discovery 18, 731-732 (2019)

doi: https://doi.org/10.1038/d41573-019-00088-7

Acknowledgements

M.J.C. is director of the Centre for Patient Reported Outcome Research at the University of Birmingham and receives funding from the National Institute for Health Research (NIHR) Birmingham Biomedical Research Centre, the NIHR Surgical Reconstruction and Microbiology Research Centre at the University Hospitals Birmingham NHS Foundation Trust, Health Data Research UK, Innovate UK (part of UK Research and Innovation), Macmillan Cancer Support and the Patient-Centered Outcomes Research Institute (PCORI). D.J.O. is a UK government employee of the Medicines and Healthcare products Regulatory Agency (MHRA), UK. E.M.B. is an employee of the University of North Carolina, receives research funding from the US National Cancer Institute and PCORI, is on the board of the American Society of Clinical Oncology, is an associate editor for JAMA and is a scientific advisor to Carevive Systems, Sivan and Self Care Catalysts.

Disclaimer

This publication reflects the views of the individual authors and should not be construed to represent official views or policies of the UK Medicines and Healthcare products Regulatory Agency (MHRA), the National Health Service, the National Institute for Health Research, the Department of Health or the UK government. All authors are members of the SPIRIT-PRO, SISAQOL and PROTEUS consortia.

References

  1. Engel, P. et al. Lessons learned on the design and the conduct of post-authorization safety studies: review of 3 years of PRAC oversight. Br. J. Clin. Pharmacol. 83, 884–893 (2016).

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  2. Kluetz, P. G. et al. Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada. Lancet Oncol. 19, e267–e274 (2018).

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  3. Calvert, M. et al. Maximising the impact of patient reported outcome assessment for patients and society. BMJ 364, k5267 (2019).

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  4. Kyte, D. et al. Reflections on the national patient-reported outcome measures (PROMs) programme: where do we go from here? J. R. Soc. Med. 109, 441–445 (2016).

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  5. Calvert, M. et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: the SPIRIT-PRO extension. JAMA 319, 483–494 (2018).

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Supplementary Information

  1. Supplementary Table 1

Competing Interests

M.J.C. has received personal fees from Astellas Pharma, Takeda, Glaukos and Merck outside the submitted work.

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