Health related quality of life aspects not captured by EQ-5D-5L: Results from an international survey of patients

doi:10.1016/j.healthpol.2018.12.003

Health Policy

Volume 123, Issue 2, February 2019, Pages 159-165

https://doi.org/10.1016/j.healthpol.2018.12.003 Get rights and content

Highlights

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EQ-5D-5L didn’t capture key QoL dimensions for more than half of the sample.
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Several QoL aspects, notably fatigue, were identified as overlooked by EQ-5D-5L.
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Around 60% of patients reported changes on key QoL aspects during their illness.
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It is unknown if other generic HRQoL measurement tools face the same challenges.

Abstract

Background

In this paper we discuss and present evidence on whether a generic Health Related Quality of Life (HRQoL) measurement tool, the EQ-5D-5L, captures the dimensions of quality of life (QoL) which patients consider significant.

Methods

An online survey, of individuals with a chronic condition, mainly breast cancer (BC), blood cancers (BLC), rheumatoid arthritis (RA), asthma, and rare diseases (RD) was conducted to collect data on HRQoL and important QoL aspects that respondents thought were not captured by the EQ-5D-5L. Patient organisations across 47 countries were invited to voluntarily share the survey tool with their membership network.

Results

767 responses from 38 countries showed that important QoL aspects were not captured by EQ-5D-5L for 51% of respondents, including fatigue (19%) and medication side effects (12%), among others. Fatigue (17%) was also the most commonly reported QoL aspect that changed over the course of patients’ illness, suggesting that the current version of the EQ-5D-5L might miss capturing significant clinical changes in important QoL domains.

Conclusions

Utilisation of the EQ-5D-5L in HRQoL measurement raises inconsistencies in capturing QoL attributes and changes in disease-specific patient populations. Further research is needed to clarify the extent to which other generic HRQoL measurement tools capture the aspects of health that really matter for patients.

Section snippets

Background

Health Technology Assessment (HTA) is used extensively by health care systems globally, to inform resource allocation decisions around the uptake of new health technologies [1]. In order to be efficient, HTA must harmonize the interests of the general population, healthcare systems and individual patient groups. Essentially, HTA should recommend the uptake of cost-effective technologies, which have a substantial benefit to patients at an affordable cost, while balancing societal preferences [2

Sample and research design

We conducted a web-based survey of an international patient population. Our sample comprised adult individuals diagnosed with at least one chronic condition. Participants were identified through a network of patients and patient associations’ representatives, held by the Medical Technology Research Group at the London School of Economics (LSE).

The survey was based on a multidimensional questionnaire comprising four sections, namely; a) patient demographic information and clinically relevant

Completion rate and sample size

Based on the invitations to 320 patient associations, a total of 1031 surveys were initiated from all 47 countries and 767 surveys were completed from 38 countries (completion rate of 74%), namely Armenia (n = 1), Australia (n = 5), Austria (n = 1), Belgium (n = 9), Bulgaria (n = 2), Canada (n = 2), Croatia (n = 12), Cyprus (n = 32), Czech Republic (n = 1), Denmark (n = 37), Estonia (n = 5), France (n = 39), Germany (n = 10), Gibraltar (n = 1), Greece (n = 55), Hungary (n = 1), Ireland (n = 7),

Discussion

We observed that EQ-5D-5L missed important health aspects in approximately 51% of an international, chronically ill population, although this percentage fluctuated according to disease area

Overall, there were 17 additional QoL aspects identified, as being important but not represented by the EQ-5D-5L. In this context, the most commonly reported missing aspect (as reported by 19% of the entire sample and 24% of the cancer sample) was that of fatigue and loss of energy. A Swiss study [18] using a

Conclusion

QoL is an important measure of disease burden, as unlike other measures, it takes into account patients’ subjective perceptions of wellbeing, treatment and overall QoL satisfaction. Understanding the factors which may reduce QoL outcomes is therefore important, for the implementation of more efficient disease management programmes, but also to pinpoint disease manifestations that may have previously been overlooked and accordingly, direct the development of new therapies. It is therefore

Conflicts of interest

None.

Acknowledgements

This study was supported by Advance-HTA, a research grants that has received funding from the European Commission, DG Research, 7th Framework Programme for Research (grant agreement No. 305983). The views expressed in this study are those of the authors and do not represent the views of the European Commission, DG Research.

We are grateful for the invaluable support of all European and international patient associations that were invited to participate in the study and voluntarily agreed to

References (35)

C.A. Marra et al.
A comparison of generic, indirect utility measures (the HUI2, HUI3, SF-6D, and the EQ-5D) and disease-specific instruments (the RAQoL and the HAQ) in rheumatoid arthritis
Social Science & Medicine
(2005)
K. Ahlberg et al.
Assessment and management of cancer-related fatigue in adults
Lancet
(2003)
P.F.M. Krabbe et al.
The effect of adding a cognitive dimension to the EuroQol multiattribute health-status classification system
Journal of Clinical Epidemiology
(1999)
P. Dolan et al.
Inconsistency and health state valuations
Social Science & Medicine
(1996)
C. Sorenson et al.
Ensuring value for money in health care: the role of health technology assessment in the European Union
(2008)
M.F. Drummond et al.
Assessing the economic challenges posed by orphan drugs
International Journal of Technology Assessment in Health Care
(2007)
D. Tordrup et al.
Responsiveness of the EQ-5D to clinical change: is the patient experience adequately represented?
International Journal of Technology Assessment in Health Care
(2014)
K. Facey et al.
Patients’ perspectives in health tech- nology assessment: a route to robust evidence and fair deliberation
International Journal of Technology Assessment in Health Care
(2010)
INAHTA
Involvement of consumers in the HTA activities of INAHTA members
(2011)
P. Dolan
How does NICE value health?
British Medical Journal
(2009)

S. Pietersma et al.

Domains of quality of life: results of a three-stage Delphi consensus procedure among patients, family of patients, clinicians, scientists and the general public

Quality of Life Research

(2014)

Brazier et al.

Nicedsu technical support document 11: alternatives to EQ-5D for generating health state utility values. Report by the decision support unit

(2011)

J. Brazier et al.

A comparison of the EQ-5D and SF-6D across seven patient groups

Journal of Health Economics

(2004)

L. Longworth et al.

Use of generic and condition-specific measures of health-related quality of life in NICE decision-making: a systematic review, statistical modelling and survey

Health Technology Assessment

(2014)

K.H. Jones et al.

How people with multiple sclerosis rate their quality of life: an EQ-5D survey via the UK MS register

PLoS One

(2013)

M.F. Janssen et al.

Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L across eight patient groups: a multi-country study

Quality of Life Research

(2013)

Parkin

Report of the OHE Commission on NHS outcomes, performance and productivity. 2008

(2008)

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View full text

Health related quality of life aspects not captured by EQ-5D-5L: Results from an international survey of patients

Highlights

Abstract

Background

Methods

Results

Conclusions

Section snippets

Background

Sample and research design

Completion rate and sample size

Discussion

Conclusion

Conflicts of interest

Acknowledgements

Social Science & Medicine

Lancet

Journal of Clinical Epidemiology

Social Science & Medicine

Ensuring value for money in health care: the role of health technology assessment in the European Union

Assessing the economic challenges posed by orphan drugs

International Journal of Technology Assessment in Health Care

Responsiveness of the EQ-5D to clinical change: is the patient experience adequately represented?

International Journal of Technology Assessment in Health Care

Patients’ perspectives in health tech- nology assessment: a route to robust evidence and fair deliberation

International Journal of Technology Assessment in Health Care

Involvement of consumers in the HTA activities of INAHTA members

How does NICE value health?

British Medical Journal

Domains of quality of life: results of a three-stage Delphi consensus procedure among patients, family of patients, clinicians, scientists and the general public

Quality of Life Research

Nicedsu technical support document 11: alternatives to EQ-5D for generating health state utility values. Report by the decision support unit

A comparison of the EQ-5D and SF-6D across seven patient groups

Journal of Health Economics

Use of generic and condition-specific measures of health-related quality of life in NICE decision-making: a systematic review, statistical modelling and survey

Health Technology Assessment

How people with multiple sclerosis rate their quality of life: an EQ-5D survey via the UK MS register

PLoS One

Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L across eight patient groups: a multi-country study

Quality of Life Research

Report of the OHE Commission on NHS outcomes, performance and productivity. 2008