Dataset Information

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.

SUBMITTER: Bharadia T

PROVIDER: S-EPMC9228659 | biostudies-literature | 2022 Apr-Jun

REPOSITORIES: biostudies-literature

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Publications

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

Bharadia Trishna T Vandercappellen Jo J Chitnis Tanuja T Eelen Piet P Bauer Birgit B Brichetto Giampaolo G Lloyd Andrew A Schmidt Hollie H King Miriam M Fitzgerald Jennifer J Hach Thomas T Hobart Jeremy J

Multiple sclerosis journal - experimental, translational and clinical 20220401 2

<h4>Background</h4>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.<h4>Objectives</h4>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 ...[more]

PMID: 35755007

Similar Datasets

Project description:ImportancePatient-reported outcome measures (PROMs) allow clinicians and researchers to assess health-related information from a patient's perspective. These measures have been used more frequently over the last several decades, but an associated minimal clinically important difference (MCID) is needed to optimize their utility. This narrative review identified the top 100 most-cited otolaryngology-related PROM development and validation publications and assessed the presence and characteristics of the PROMs' associated MCID.ObservationsIn this narrative review, a literature search in Scopus and Web of Science was conducted on June 29, 2022, using keywords related to PROM development and validation studies in otolaryngology and reference lists. Studies that met the definition of a PROM and assessed an otolaryngologic disorder or study population were included for full-text review. After full-text review of 188 articles, the top 100 most-cited PROM development and validation publications, resulting in 106 total PROMs, were chosen for review. A total of 39 (37%) of the identified PROMs had an associated MCID. Of those reporting an MCID, 14 (35.9%) used an anchor-based method, 12 (30.8%) used a distribution-based method, 10 (25.6%) used both, and 3 (7.7%) did not specify or used neither method. Rhinology had the greatest number of PROMs with an associated MCID (16 of 24, 66%), and pediatrics had the fewest (1 of 13, 7.7%). The median number of citations of PROMs with an MCID was higher than those without an MCID.Conclusions and relevanceThe majority of the most-cited PROMs in otolaryngology lack an associated MCID. These data indicated that there are a multitude of PROMs that have been cited hundreds of times and used for decades without the ability to identify whether a particular change in score on the instrument is clinically meaningful. There is a need to determine and validate MCIDs for commonly used PROMs to aid clinical research and trial interpretation.

Dataset Information

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

Background

Objectives

Methods

Results

Conclusions

Publications

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

Similar Datasets

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