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Health and Quality of Life Outcomes
Published in: Health and Quality of Life Outcomes 1/2016

Open Access 01-12-2016 | Research

Interpreting change from patient reported outcome (PRO) endpoints: patient global ratings of concept versus patient global ratings of change, a case study among osteoporosis patients

Authors: Annabel Nixon, Helen Doll, Cicely Kerr, Russel Burge, April N. Naegeli

Published in: Health and Quality of Life Outcomes | Issue 1/2016

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Abstract

Background

Regulatory guidance recommends anchor-based methods for interpretation of treatment effects measured by PRO endpoints. Methodological pros and cons of patient global ratings of change vs. patient global ratings of concept have been discussed but empirical evidence in support of either approach is lacking. This study evaluated the performance of patient global ratings of change and patient global ratings of concept for interpreting patient stability and patient improvement.

Methods

Patient global ratings of change and patient global ratings of concept were included in a psychometric validation study of an osteoporosis-targeted PRO instrument (the OPAQ-PF) to assess its ability to detect change and to derive responder definitions. 144 female osteoporosis patients with (n = 37) or without (n = 107) a recent (within 6 weeks) fragility fracture completed the OPAQ-PF and global items at baseline, 2 weeks (no recent fracture), and 12 weeks (recent fracture) post-baseline.

Results

Results differed between the two methods. Recent fracture patients reported more improvement while patients without recent fracture reported more stability on ratings of change than ratings of concept. However, correlations with OPAQ-PF score change were stronger for ratings of concept than ratings of change (both groups). Effect sizes for OPAQ-PF score change increased consistently with level of change in ratings of concept but inconsistently with ratings of change, with the mean AUC for prediction of a one-point change being 0.72 vs. 0.56.

Conclusions

This study provides initial empirical support for methodological and regulatory recommendations to use patient global ratings of concept rather than ratings of change when interpreting change captured by PRO instruments in studies evaluating treatment effects. These findings warrant being confirmed in a purpose-designed larger scale analysis.
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Metadata
Title
Interpreting change from patient reported outcome (PRO) endpoints: patient global ratings of concept versus patient global ratings of change, a case study among osteoporosis patients
Authors
Annabel Nixon
Helen Doll
Cicely Kerr
Russel Burge
April N. Naegeli
Publication date
01-12-2016
Publisher
BioMed Central
Published in
Health and Quality of Life Outcomes / Issue 1/2016
Electronic ISSN: 1477-7525
DOI
https://doi.org/10.1186/s12955-016-0427-5

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