Recommendations to address respondent burden associated with patient-reported outcome assessment

Olalekan Lee Aiyegbusi^{1

2

3

4

5}, Samantha Cruz Rivera^{6

7}, Jessica Roydhouse^{8

9}, Paul Kamudoni¹⁰, Yvonne Alder⁶, Nicola Anderson^{6

11

12

13}, Robert Mitchell Baldwin¹⁴, Vishal Bhatnagar¹⁵, Jennifer Black¹⁴, Andrew Bottomley¹⁶, Michael Brundage¹⁷, David Cella¹⁸, Philip Collis⁶, Elin-Haf Davies¹⁹, Alastair K Denniston^{11

13}, Fabio Efficace²⁰, Adrian Gardner^{21

22}, Ari Gnanasakthy²³, Robert M Golub²⁴, Sarah E Hughes^{6

11

12

25}, Flic Jeyes⁶, Scottie Kern²⁶, Bellinda L King-Kallimanis²⁷, Antony Martin²⁸, Christel McMullan^{6

25}, Rebecca Mercieca-Bebber²⁹, Joao Monteiro³⁰, John Devin Peipert¹⁸, Juan Carlos Quijano-Campos^{31

32

33}, Chantal Quinten³⁴, Khadija Rerhou Rantell³⁵, Antoine Regnault³⁶, Maxime Sasseville¹⁴, Liv Marit Valen Schougaard³⁷, Roya Sherafat-Kazemzadeh³⁸, Claire Snyder³⁹, Angela M Stover^{40

41}, Rav Verdi⁶, Roger Wilson^{6

42}, Melanie J Calvert^{6

11

12

25

7}

Affiliations

¹ Centre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
² National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC), University Hospital Birmingham and University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
³ National Institute for Health and Care Research (NIHR) Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
⁴ NIHR Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
⁵ Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
⁶ Centre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
⁷ Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.
⁸ Menzies Institute for Medical Research, University of Tasmania, Hobart, Tasmania, Australia.
⁹ Department of Health Services Policy and Practice, Brown University School of Public Health, Providence, RI, USA.
¹⁰ Merck KGaA, Darmstadt, Germany.
¹¹ National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC), University Hospital Birmingham and University of Birmingham, Birmingham, UK.
¹² National Institute for Health and Care Research (NIHR) Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK.
¹³ University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
¹⁴ Health Canada, Ottawa, Ontario, Canada.
¹⁵ Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, USA.
¹⁶ Bottomley Consulting Group, Belgium, Belgium.
¹⁷ Queen's University, Kingston, Ontario, Canada.
¹⁸ Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
¹⁹ Aparito, Wrexham, UK.
²⁰ Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
²¹ The Royal Orthopaedic Hospital NHS Foundation Trust, Birmingham, UK.
²² Aston University, Birmingham, UK.
²³ RTI Health Solutions, Durham, NC, USA.
²⁴ Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
²⁵ NIHR Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK.
²⁶ Critical Path Institute, Tucson, AZ, USA.
²⁷ LUNGevity Foundation, Bethesda, MD, USA.
²⁸ QC Medica, Liverpool, UK.
²⁹ The NHMRC Clinical Trials Centre, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.
³⁰ Nature Medicine, New York, NY, USA.
³¹ William Harvey Research Institute, Queen Mary University of London, London, UK.
³² St Bartholomew's Hospital, Barts Health NHS Trust, London, UK.
³³ Royal Papworth Hospital NHS Foundation Trust, Cambridge, UK.
³⁴ European Medicines Agency, Amsterdam, Netherlands.
³⁵ Medicines and Healthcare products Regulatory Agency, London, UK.
³⁶ Modus Outcomes, Lyon, France.
³⁷ AmbuFlex, Centre for Patient-reported Outcomes, Gødstrup Hospital, Herning, Denmark.
³⁸ Mapi Research Trust, Lyon, France.
³⁹ Johns Hopkins Schools of Medicine and Public Health, Baltimore, MD, USA.
⁴⁰ University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
⁴¹ Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC, USA.
⁴² Cancer Research Advocacy Forum, London, UK.

PMID: 38424214
DOI: 10.1038/s41591-024-02827-9

Review

Recommendations to address respondent burden associated with patient-reported outcome assessment

Olalekan Lee Aiyegbusi et al. Nat Med. 2024 Mar.

. 2024 Mar;30(3):650-659.

doi: 10.1038/s41591-024-02827-9. Epub 2024 Feb 29.

Authors

Affiliations

¹ Centre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
² National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC), University Hospital Birmingham and University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
³ National Institute for Health and Care Research (NIHR) Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
⁴ NIHR Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
⁵ Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK. O.L.Aiyegbusi@bham.ac.uk.
⁶ Centre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
⁷ Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.
⁸ Menzies Institute for Medical Research, University of Tasmania, Hobart, Tasmania, Australia.
⁹ Department of Health Services Policy and Practice, Brown University School of Public Health, Providence, RI, USA.
¹⁰ Merck KGaA, Darmstadt, Germany.
¹¹ National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC), University Hospital Birmingham and University of Birmingham, Birmingham, UK.
¹² National Institute for Health and Care Research (NIHR) Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK.
¹³ University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
¹⁴ Health Canada, Ottawa, Ontario, Canada.
¹⁵ Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, USA.
¹⁶ Bottomley Consulting Group, Belgium, Belgium.
¹⁷ Queen's University, Kingston, Ontario, Canada.
¹⁸ Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
¹⁹ Aparito, Wrexham, UK.
²⁰ Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
²¹ The Royal Orthopaedic Hospital NHS Foundation Trust, Birmingham, UK.
²² Aston University, Birmingham, UK.
²³ RTI Health Solutions, Durham, NC, USA.
²⁴ Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
²⁵ NIHR Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK.
²⁶ Critical Path Institute, Tucson, AZ, USA.
²⁷ LUNGevity Foundation, Bethesda, MD, USA.
²⁸ QC Medica, Liverpool, UK.
²⁹ The NHMRC Clinical Trials Centre, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.
³⁰ Nature Medicine, New York, NY, USA.
³¹ William Harvey Research Institute, Queen Mary University of London, London, UK.
³² St Bartholomew's Hospital, Barts Health NHS Trust, London, UK.
³³ Royal Papworth Hospital NHS Foundation Trust, Cambridge, UK.
³⁴ European Medicines Agency, Amsterdam, Netherlands.
³⁵ Medicines and Healthcare products Regulatory Agency, London, UK.
³⁶ Modus Outcomes, Lyon, France.
³⁷ AmbuFlex, Centre for Patient-reported Outcomes, Gødstrup Hospital, Herning, Denmark.
³⁸ Mapi Research Trust, Lyon, France.
³⁹ Johns Hopkins Schools of Medicine and Public Health, Baltimore, MD, USA.
⁴⁰ University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
⁴¹ Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC, USA.
⁴² Cancer Research Advocacy Forum, London, UK.

PMID: 38424214
DOI: 10.1038/s41591-024-02827-9

Abstract

Patient-reported outcomes (PROs) are increasingly used in healthcare research to provide evidence of the benefits and risks of interventions from the patient perspective and to inform regulatory decisions and health policy. The use of PROs in clinical practice can facilitate symptom monitoring, tailor care to individual needs, aid clinical decision-making and inform value-based healthcare initiatives. Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete PROs, with potential impact on the completeness and quality of the data for decision-making. We therefore conducted an initial literature review to generate a list of candidate recommendations aimed at reducing respondent burden. This was followed by a two-stage Delphi survey by an international multi-stakeholder group. A consensus meeting was held to finalize the recommendations. The final consensus statement includes 19 recommendations to address PRO respondent burden in healthcare research and clinical practice. If implemented, these recommendations may reduce PRO respondent burden.

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References

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1. Calvert, M., Kyte, D., Price, G., Valderas, J. M. & Hjollund, N. H. Maximising the impact of patient-reported outcome assessment for patients and society. BMJ 364, k5267 (2019). - PubMed - DOI

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Recommendations to address respondent burden associated with patient-reported outcome assessment

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Recommendations to address respondent burden associated with patient-reported outcome assessment

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