Abstract
BackgroundThe 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.MethodsLupusQoL 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.ResultsFor 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.ConclusionsThe 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.
Highlights
During phase III clinical trials, the effect of an intervention is assessed by comparing differences in endpoints between the intervention and control groups to determine if the intervention provides treatment benefit
An in-depth summary of the evaluation results for target population, concepts measured, measurement properties, and documentation are presented for each Systemic Lupus Erythematosus (SLE)-measure separately in the Additional file 1: Appendix 3
Measurement properties The description of methods and results for measurement properties were assessed to determine if measurement properties included all expected attributes [6]
Summary
During phase III clinical trials, the effect of an intervention is assessed by comparing differences in endpoints between the intervention and control groups to determine if the intervention provides treatment benefit. It may be evaluated using tools that directly or indirectly measure how patients feel, function, or survive [1, 2]. Direct evidence of treatment benefit is a measure of a meaningful health aspect, such as survival or a direct report from patients regarding feelings and functions in their daily activities living with their condition.. 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 Admin‐ istration (FDA) 2009 patient-reported outcome (PRO) guidance
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
