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The value of patient-reported outcomes in early-phase clinical trials

Nature Medicine volume 28, pages 18–20 (2022)Cite this article

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If used correctly, patient-reported outcomes can provide preliminary evidence of efficacy and tolerability from a patient perspective, as well as supporting regulatory review.

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Fig. 1: Patient-reported outcomes in early-phase trials.
Fig. 2: Strategy for patient-reported outcome assessment.

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Acknowledgements

We thank A. Filer, P. Trivedi, D. Chanouzas, S. Ghosh and A. Dueck for their advice and suggestions during this project. The author(s) disclose receipt of the following financial support for the research, authorship and/or publication of this article: This work was supported by the National Institute for Health Research (NIHR) Birmingham Biomedical Research Centre, Midlands-Wales Advanced Therapies Treatment Centre (MW-ATTC) program grant from Innovate UK to a consortium of partners including Health Technology Wales, the Welsh Blood Service and the University of Birmingham (grant number: IUK: 104232) and Innovate UK (part of UK Research and Innovation) grant Patient-reported outcomes assessment to support accelerated access to advanced cell and gene therapies: PROmics (grant number: 104777). The funder was not involved in any aspect of the research work. O.L.A., A.R. and M.C. receive funding from the NIHR Applied Research Collaboration, West Midlands (ARC), and the Brain Tumour Charity. O.L.A, receives funding from the NIHR Birmingham Biomedical Research Centre (BRC, West Midlands at the University of Birmingham) and University Hospitals Birmingham NHS Foundation, Innovate UK (part of UK Research and Innovation), Gilead Sciences Ltd. and Janssen Pharmaceuticals Inc. MC is Director of the Birmingham Health Partners Centre for Regulatory Science and Innovation, Director of the Centre for Patient Reported Outcomes Research and is a National Institute for Health Research (NIHR) Senior Investigator. She receives funding from the NIHR, UK Research and Innovation (UKRI), NIHR Birmingham Biomedical Research Centre, the NIHR Surgical Reconstruction and Microbiology Research Centre and UK SPINE at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, Health Data Research UK, Innovate UK (part of UKRI), Macmillan Cancer Support, UCB, Gilead, Janssen and GSK Pharma. The views expressed in this article are those of the authors and not necessarily those of the NIHR, the Department of Health and Social Care or the MHRA.

Author information

Authors and Affiliations

  1. Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK

    Ameeta Retzer, Olalekan Lee Aiyegbusi & Melanie Calvert

  2. National Institute for Health Research Applied Research Centre West Midlands, University of Birmingham, Birmingham, UK

    Ameeta Retzer, Olalekan Lee Aiyegbusi & Melanie Calvert

  3. National Institute for Health Research Birmingham Biomedical Research Centre at University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK

    Olalekan Lee Aiyegbusi, Anna Rowe, Philip N. Newsome, Sheeba Khan & Melanie Calvert

  4. Birmingham Health Partners Centre for Regulatory Science and Innovation, Birmingham, UK

    Olalekan Lee Aiyegbusi & Melanie Calvert

  5. Cancer Research UK Clinical Trials Unit, University of Birmingham, Birmingham, UK

    Anna Rowe & Jessica Douglas-Pugh

  6. Centre for Liver and Gastrointestinal Research, Institute of Immunology and Immunotherapy, University of Birmingham, Birmingham, UK

    Philip N. Newsome & Sheeba Khan

  7. Liver Unit, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK

    Sheeba Khan

  8. University of Birmingham, Birmingham, UK

    Saloni Mittal

  9. NCRI Consumer Forum National Cancer Research Institute, London, UK

    Roger Wilson

  10. Medicines and Healthcare Products Regulatory Agency (MHRA), London, UK

    Daniel O’Connor & Lisa Campbell

  11. Outcomes Research Branch, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, MD, USA

    Sandra A. Mitchell

  12. National Institute for Health Research Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, Birmingham, UK

    Melanie Calvert

  13. UK SPINE, University of Birmingham, Birmingham, UK

    Melanie Calvert

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  1. Ameeta Retzer
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Corresponding author

Correspondence to Melanie Calvert.

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Competing interests

O.L.A. declares personal fees from Gilead Sciences Ltd., GlaxoSmithKline (GSK) and Merck outside the submitted work. M.C. has received personal fees from Astellas, Aparito Ltd., CIS Oncology, Takeda, Merck, Daiichi Sankyo, Glaukos, GSK and the Patient-Centered Outcomes Research Institute (PCORI) outside the submitted work. The other authors declare no competing interests.

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Retzer, A., Aiyegbusi, O.L., Rowe, A. et al. The value of patient-reported outcomes in early-phase clinical trials. Nat Med 28, 18–20 (2022). https://doi.org/10.1038/s41591-021-01648-4

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