Abstract
Objectives: Whilst patient input to Health Technology Assessment (HTA) has traditionally been of a qualitative nature, there is increasing interest to integrate quantitative evidence from patient preference studies into HTA decision-making. Preference data can be used to generate disease specific health utility data. We generated a health utility score for patients with Chronic Obstructive Pulmonary Disease (COPD) and consider its use within HTAs.
Methods: Based on qualitative research, six symptoms were identified as important to COPD patients: shortness of breath, exacerbations, chronic cough, mucus secretion, sleep disturbance and urinary incontinence. We employed a Discrete Choice Experiment (DCE) and the random parameter logistic (RPL) regression technique to estimate utility scores for all COPD health states. The relationship between patients’ COPD health utility score, self-perceived COPD severity, and EQ-5D-3L utility scores was analyzed, with data stratified according to disease severity and comorbidity subgroups.
Results: The COPD health utility score had face validity, with utility scores negatively correlated with patients’ self-perceived COPD severity. Correlation between the COPD health utility scores and EQ-5D-3L values was only moderate. Whilst patient EQ-5D-3L scores were impacted by comorbidities, the COPD health utility score was less impacted by comorbid conditions.
Conclusions: Our COPD utility measure derived from a DCE, provides a patient-centered health utility score, is more sensitive to the COPD health of the individual and less sensitive to other comorbidities. This disease-specific instrument should be considered alongside generic health-related quality of life instruments when valuing new COPD therapies in submissions to licensing and reimbursement agencies.
Methods: Based on qualitative research, six symptoms were identified as important to COPD patients: shortness of breath, exacerbations, chronic cough, mucus secretion, sleep disturbance and urinary incontinence. We employed a Discrete Choice Experiment (DCE) and the random parameter logistic (RPL) regression technique to estimate utility scores for all COPD health states. The relationship between patients’ COPD health utility score, self-perceived COPD severity, and EQ-5D-3L utility scores was analyzed, with data stratified according to disease severity and comorbidity subgroups.
Results: The COPD health utility score had face validity, with utility scores negatively correlated with patients’ self-perceived COPD severity. Correlation between the COPD health utility scores and EQ-5D-3L values was only moderate. Whilst patient EQ-5D-3L scores were impacted by comorbidities, the COPD health utility score was less impacted by comorbid conditions.
Conclusions: Our COPD utility measure derived from a DCE, provides a patient-centered health utility score, is more sensitive to the COPD health of the individual and less sensitive to other comorbidities. This disease-specific instrument should be considered alongside generic health-related quality of life instruments when valuing new COPD therapies in submissions to licensing and reimbursement agencies.
Original language | English |
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Article number | e30 |
Number of pages | 8 |
Journal | International Journal of Technology Assessment in Health Care |
Volume | 40 |
Issue number | 1 |
Early online date | 2 May 2024 |
DOIs | |
Publication status | Published - 2 May 2024 |
Bibliographical note
Open Access via the CUP AgreementAcknowledgements; We wish to acknowledge the support of Katie Mycock, Tom Gardner, Phil Mellor, Pam Hallworth, Kate Sully, Sophi Tatlock, Beyza Klein, Gerard Criner, Pierre-Régis Burgel, Olivier Le Rouzic, Kip Adams, Kirsten Phillips, Mike McKevitt, and Kazuko Toyama for their involvement and contributions to the design, conduct and analysis of this patient preference study, and Kevin Marsh for comments and suggestions for further analyses based on a manuscript draft.
Funding statement:
The study was funded in full by Novartis Pharma AG. Byron Jones and Nigel Cook are employees of Novartis Pharma AG. At the time of writing, Florian Gutzwiller was an employee of Novartis Pharma AG.
Data Availability Statement
No data availability statement.Keywords
- Disease-specific health utility score
- Chronic Obstructive Pulmonary Disease
- EQ56 5D-3L
- Patient preference