Patient-reported outcome measures in MS: Do development processes and patient involvement support valid quantification of clinically important variables?

Trishna Bharadia; Jo Vandercappellen; Tanuja Chitnis; Piet Eelen; Birgit Bauer; Giampaolo Brichetto; Andrew Lloyd; Hollie Schmidt; Miriam King; Jennifer Fitzgerald; Thomas Hach; Jeremy Hobart

doi:10.1177/20552173221105642

Patient-reported outcome measures in MS: Do development processes and patient involvement support valid quantification of clinically important variables?

Trishna Bharadia, Jo Vandercappellen, Tanuja Chitnis, Piet Eelen, Birgit Bauer, Giampaolo Brichetto, Andrew Lloyd, Hollie Schmidt, Miriam King, Jennifer Fitzgerald, Thomas Hach, Jeremy Hobart^*

^*Corresponding author for this work

Peninsula Medical School

Research output: Contribution to journal › Article › peer-review

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Abstract

Background Patient-reported outcomes (PROs) are widely measured in multiple sclerosis (MS) studies. However, the quality of instrument development processes varies, raising concerns about the meaningfulness of associated data. Objectives To review the development of selected PROs commonly used in MS studies, including definitions of the concepts measured, use of conceptual frameworks, and degree of input from people living with MS (PlwMS). To gain insights and recommendations from PlwMS on their experience with these PROs. Methods We assessed 6 PROs (FSIQ-RMS, modified-FIS, MSQoL-54, Leeds 8-item MSQoL, MSIS-29 and EQ-5D) for alignment with regulatory and scientific requirements on PRO structure/development. PlwMS evaluated the degree to which the PROs reflect disease aspects they perceive important. Results Definitions, clarifications and conceptualisations of the measurement variables were often lacking. PlwMS were variably involved in PRO development. Ethnic diversity was rarely documented. PlwMS identified individualisation, ease of understanding, time burden, and mode of administration as factors affecting PRO usability. Conclusions To date, the PRO development process has consistently lacked clear definitions of concepts of interest, use of conceptual frameworks and patient involvement, thereby compromising the validity of data they generate. PRO instrument development must be conducted more robustly to maximise the value of pivotal clinical trials.

Original language	English
Pages (from-to)	205521732211056-205521732211056
Number of pages	0
Journal	Multiple Sclerosis Journal - Experimental, Translational and Clinical
Volume	8
Issue number	2
Early online date	22 Jun 2022
DOIs	https://doi.org/10.1177/20552173221105642
Publication status	Published - 22 Jun 2022

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10.1177/20552173221105642

20552173221105642

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Bharadia, T., Vandercappellen, J., Chitnis, T., Eelen, P., Bauer, B., Brichetto, G., Lloyd, A., Schmidt, H., King, M., Fitzgerald, J., Hach, T., & Hobart, J. (2022). Patient-reported outcome measures in MS: Do development processes and patient involvement support valid quantification of clinically important variables? Multiple Sclerosis Journal - Experimental, Translational and Clinical, 8(2), 205521732211056-205521732211056. https://doi.org/10.1177/20552173221105642

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title = "Patient-reported outcome measures in MS: Do development processes and patient involvement support valid quantification of clinically important variables?",

abstract = "Background Patient-reported outcomes (PROs) are widely measured in multiple sclerosis (MS) studies. However, the quality of instrument development processes varies, raising concerns about the meaningfulness of associated data. Objectives To review the development of selected PROs commonly used in MS studies, including definitions of the concepts measured, use of conceptual frameworks, and degree of input from people living with MS (PlwMS). To gain insights and recommendations from PlwMS on their experience with these PROs. Methods We assessed 6 PROs (FSIQ-RMS, modified-FIS, MSQoL-54, Leeds 8-item MSQoL, MSIS-29 and EQ-5D) for alignment with regulatory and scientific requirements on PRO structure/development. PlwMS evaluated the degree to which the PROs reflect disease aspects they perceive important. Results Definitions, clarifications and conceptualisations of the measurement variables were often lacking. PlwMS were variably involved in PRO development. Ethnic diversity was rarely documented. PlwMS identified individualisation, ease of understanding, time burden, and mode of administration as factors affecting PRO usability. Conclusions To date, the PRO development process has consistently lacked clear definitions of concepts of interest, use of conceptual frameworks and patient involvement, thereby compromising the validity of data they generate. PRO instrument development must be conducted more robustly to maximise the value of pivotal clinical trials. ",

author = "Trishna Bharadia and Jo Vandercappellen and Tanuja Chitnis and Piet Eelen and Birgit Bauer and Giampaolo Brichetto and Andrew Lloyd and Hollie Schmidt and Miriam King and Jennifer Fitzgerald and Thomas Hach and Jeremy Hobart",

year = "2022",

month = jun,

day = "22",

doi = "10.1177/20552173221105642",

language = "English",

volume = "8",

pages = "205521732211056--205521732211056",

journal = "Multiple Sclerosis Journal - Experimental, Translational and Clinical",

issn = "2055-2173",

publisher = "SAGE Publications Inc.",

number = "2",

}