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Ongoing validation of health-related quality of life instruments
Trials volume 16, Article number: O8 (2015)
Background
Validated health-related quality of life (HRQOL) instruments may require updating as they begin to be used in randomised controlled trials (RCTs) containing patient populations and novel drugs that did not form part of the original validation study. Multiple myeloma (MM) is one example where new classes of drugs are rapidly emerging. There are a number of commonly used HRQOL instruments used, some generic and some MM specific but it is unclear how valid existing instruments will be in RCTs of novel agents. We aim to make recommendations for ongoing validation of HRQOL instruments in RCTs applicable to many disease areas using empirical evidence from the case of MM.
Methods
We searched MEDLINE for RCT's in MM measuring HRQOL conducted in the last 10 years. We also surveyed instrument publications and websites in order to determine whether the instrument was validated for use in the RCT population, how long ago and whether there are provisions for ongoing validation.
Results
Our search returned 146 abstracts. Validation studies sometimes contained a small sample of sub-populations of a disease (e.g. the EORTC QLQ-MY20 validation sample had 225 newly diagnosed and only 15 relapsed patients).
Conclusions
Health-related quality of life instruments should be subjected to ongoing validation to ensure they remain relevant in their field and for specific clinical trial patient populations. We discuss simple psychometrics that can be incorporated into the RCT statistical analysis plan and adding debrief questions to the instrument could provide additional insight, particularly in phase II studies of novel drugs.
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This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
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Cocks, K., Tharmanathan, P. Ongoing validation of health-related quality of life instruments. Trials 16, O8 (2015). https://doi.org/10.1186/1745-6215-16-S2-O8
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Keywords
- Multiple Myeloma
- Myeloma
- Validation Study
- Analysis Plan
- Validation Sample
