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Capturing the patient experience in systemic lupus erythematosus: Are widely used measures fit-for-purpose and adherent to FDA PRO guidance recommendations?

BACKGROUND: The 2009 Food and Drug Administration (FDA) patient-reported outcome (PRO) guidance outlines characteristics of rigorous PRO-measure development. There are a number of widely used PRO measures for Systemic Lupus Erythematosus (SLE), but it is unknown how well the development processes of...

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Autores principales: Majercak, Kayleigh R., Perfetto, Eleanor M., Villalonga-Olives, Ester
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8777546/
https://www.ncbi.nlm.nih.gov/pubmed/35061149
http://dx.doi.org/10.1186/s41687-022-00411-8
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author Majercak, Kayleigh R.
Perfetto, Eleanor M.
Villalonga-Olives, Ester
author_facet Majercak, Kayleigh R.
Perfetto, Eleanor M.
Villalonga-Olives, Ester
author_sort Majercak, Kayleigh R.
collection PubMed
description BACKGROUND: The 2009 Food and Drug Administration (FDA) patient-reported outcome (PRO) guidance outlines characteristics of rigorous PRO-measure development. There are a number of widely used PRO measures for Systemic Lupus Erythematosus (SLE), but it is unknown how well the development processes of SLE PRO measures align with FDA guidance; including updated versions. The objective of this study was to assess how well the LupusQoL and LupusPRO, and corresponding updated versions, LupusQoL-US and LupusPROv1.8, align with Food and Drug Administration (FDA) 2009 patient-reported outcome (PRO) guidance. METHODS: LupusQoL and LupusPRO were selected as the most widely studied and used Lupus PROs in the UK and US. Original (LupusQoL (2007) and LupusQoL-US (2010)) and revised (LupusPROVv1.7 (2012) and LupusPROv1.8 (2018)) versions were reviewed. We used FDA PRO guidance to create evaluation criteria for key components: target population, concepts measured, measurement properties, documentation across the phases of content validity (item-generation and cognitive interviewing, separately) and other psychometric-property testing. Two reviewers abstracted data independently, compared results, and resolved discrepancies. RESULTS: For all measures, the target population was unclear as population characteristics (e.g., ethnicity, education, disease severity) varied, and/or were not consistently reported or not considered across the three phases (e.g., LupusQoL item-generation lacked male involvement, LupusPRO cognitive-interviewing population characteristics were not reported). The item-generation phase for both original measures was conducted with concepts elicited via patient-engagement interviews and item derivation from experts. Cognitive interviewing was conducted via patient feedback with limited item-tracking for original measures. In contrast, the revised measures assumed content validity. Other psychometric testing recommendations (reliability, construct validity, ability to detect change) were reported for both original and revised measures, except for ability to detect change for revised measures. CONCLUSIONS: The SLE PRO measures adhere to some but not all FDA PRO guidance recommendations. Limitations in processes and documentation of the study population, make it unclear for which target population(s) the current Lupus measures are fit-for-purpose. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s41687-022-00411-8.
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spelling pubmed-87775462022-02-02 Capturing the patient experience in systemic lupus erythematosus: Are widely used measures fit-for-purpose and adherent to FDA PRO guidance recommendations? Majercak, Kayleigh R. Perfetto, Eleanor M. Villalonga-Olives, Ester J Patient Rep Outcomes Research BACKGROUND: The 2009 Food and Drug Administration (FDA) patient-reported outcome (PRO) guidance outlines characteristics of rigorous PRO-measure development. There are a number of widely used PRO measures for Systemic Lupus Erythematosus (SLE), but it is unknown how well the development processes of SLE PRO measures align with FDA guidance; including updated versions. The objective of this study was to assess how well the LupusQoL and LupusPRO, and corresponding updated versions, LupusQoL-US and LupusPROv1.8, align with Food and Drug Administration (FDA) 2009 patient-reported outcome (PRO) guidance. METHODS: LupusQoL and LupusPRO were selected as the most widely studied and used Lupus PROs in the UK and US. Original (LupusQoL (2007) and LupusQoL-US (2010)) and revised (LupusPROVv1.7 (2012) and LupusPROv1.8 (2018)) versions were reviewed. We used FDA PRO guidance to create evaluation criteria for key components: target population, concepts measured, measurement properties, documentation across the phases of content validity (item-generation and cognitive interviewing, separately) and other psychometric-property testing. Two reviewers abstracted data independently, compared results, and resolved discrepancies. RESULTS: For all measures, the target population was unclear as population characteristics (e.g., ethnicity, education, disease severity) varied, and/or were not consistently reported or not considered across the three phases (e.g., LupusQoL item-generation lacked male involvement, LupusPRO cognitive-interviewing population characteristics were not reported). The item-generation phase for both original measures was conducted with concepts elicited via patient-engagement interviews and item derivation from experts. Cognitive interviewing was conducted via patient feedback with limited item-tracking for original measures. In contrast, the revised measures assumed content validity. Other psychometric testing recommendations (reliability, construct validity, ability to detect change) were reported for both original and revised measures, except for ability to detect change for revised measures. CONCLUSIONS: The SLE PRO measures adhere to some but not all FDA PRO guidance recommendations. Limitations in processes and documentation of the study population, make it unclear for which target population(s) the current Lupus measures are fit-for-purpose. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s41687-022-00411-8. Springer International Publishing 2022-01-21 /pmc/articles/PMC8777546/ /pubmed/35061149 http://dx.doi.org/10.1186/s41687-022-00411-8 Text en © The Author(s) 2022 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) .
spellingShingle Research
Majercak, Kayleigh R.
Perfetto, Eleanor M.
Villalonga-Olives, Ester
Capturing the patient experience in systemic lupus erythematosus: Are widely used measures fit-for-purpose and adherent to FDA PRO guidance recommendations?
title Capturing the patient experience in systemic lupus erythematosus: Are widely used measures fit-for-purpose and adherent to FDA PRO guidance recommendations?
title_full Capturing the patient experience in systemic lupus erythematosus: Are widely used measures fit-for-purpose and adherent to FDA PRO guidance recommendations?
title_fullStr Capturing the patient experience in systemic lupus erythematosus: Are widely used measures fit-for-purpose and adherent to FDA PRO guidance recommendations?
title_full_unstemmed Capturing the patient experience in systemic lupus erythematosus: Are widely used measures fit-for-purpose and adherent to FDA PRO guidance recommendations?
title_short Capturing the patient experience in systemic lupus erythematosus: Are widely used measures fit-for-purpose and adherent to FDA PRO guidance recommendations?
title_sort capturing the patient experience in systemic lupus erythematosus: are widely used measures fit-for-purpose and adherent to fda pro guidance recommendations?
topic Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8777546/
https://www.ncbi.nlm.nih.gov/pubmed/35061149
http://dx.doi.org/10.1186/s41687-022-00411-8
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