Abstract
Background
Cost-effectiveness models in Parkinson’s disease often include health states based on Hoehn and Yahr (H&Y) and time in ‘off’. Few studies have investigated utilities in these health states.
Objective
The aim of this study was firstly to explore utilities in health states based on H&Y and off-time, and secondly to investigate to what extent H&Y and off-time correlated with EQ-5D dimensions.
Methods
Patients with idiopathic Parkinson’s disease in the National Parkinson’s Disease Patient Registry (PARKreg) in Sweden with observations of EQ-5D-3L, H&Y and off-time were included. Correlations with EQ-5D dimensions were analyzed. The relationship between the EQ-5D-3L and H&Y and off-time were estimated by a linear mixed-model with random intercept.
Results
Among patients in PARKreg, 1823 observations fulfilled inclusion criteria. The dimensions ‘self-care’, ‘mobility’ and ‘usual activities’ correlated moderately with H&Y (rs = 0.45, rs = 0.46, rs = 0.45). Weak correlations were found for ‘anxiety/depression’ and ‘pain/discomfort’ (rs = 0.24, rs = 0.22) (p values < 0.001). All dimensions correlated weakly with off-time. The fitted model included H&Y, time in ‘off’, and sex. All H&Y stages were found to be significant and had large and monotonous impact on EQ-5D. Off-time was not significant, but improved the model goodness of fit. Predicted values ranged from 0.733 to − 0.106.
Conclusion
This study provides utilities for health states reflecting the current modeling practice of interventions targeting motor symptoms in Parkinson’s disease. Future research should investigate patient utilities in health states that also capture non-motor symptoms of the disease, as the management of and options for treatments targeting these symptoms increases.
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Funding
The research has received financial support from AbbVie, Medtronic and Nordic Infucare (Air Liquide Healthcare). The sponsors had no access to data. The authors had full independence regarding data collection, manuscript preparation, decision to publish, study design, interpretation, and analysis.
Conflict of interest
PO has received honoraria for lectures and expert advice from AbbVie, Bial, Britannia, Global Kinetics, Lobsor, Nordic Infucare, PD Neurotechnology and Zambon. KK and JMN are employees at the Swedish Institute for Health Economics (IHE), which provides consulting services for a broad range of health care stakeholders, including national authorities, healthcare providers, branch organizations, and manufacturers.
Ethics approval
The study was performed in accordance with the Declaration of Helsinki and with approval from the Lund ethical review board. All patients have provided their informed consent to participate in PARKreg.
Consent to participate
Informed consent was obtained from all patients when enrolled in the PARKreg register. Patients can opt out from the register at any time.
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Not applicable.
Availability of data and material
The data that support the findings of this study were retrieved from PARKreg. Restrictions apply to the availability of these data, which were used under license for the current study, and so are not publicly available.
Code availability
Code from R is available from the authors upon request.
Author contributions
JMN, KK and PO contributed to the conceptional framework and the study design. KK carried out the statistical analyses with contribution from JMN. JMN and KK wrote the manuscript with input from PO.
Acknowledgements
We would like to thank patients, health care professionals, Swedish Movement Disorder Society and SWEPAR-net for using and advancing PARKreg.
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Norlin, J.M., Kellerborg, K. & Odin, P. Patient Utilities in Health States Based on Hoehn and Yahr and Off-Time in Parkinson’s Disease: A Swedish Register-Based Study in 1823 Observations. PharmacoEconomics 39, 1141–1149 (2021). https://doi.org/10.1007/s40273-021-01056-z
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DOI: https://doi.org/10.1007/s40273-021-01056-z