Skip to main content

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

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.

This is a preview of subscription content, access via your institution.

Fig. 1
Fig. 2

References

  1. Jenkinson C, Fitzpatrick R, Peto V, Greenhall R, Hyman N. The PDQ-8: Development and validation of a short-form parkinson’s disease questionnaire. Psychol Health. 1997;12(6):805–14.

    Article  Google Scholar 

  2. Jenkinson C, Fitzpatrick R, Peto V, Greenhall R, Hyman N. The Parkinson’s Disease Questionnaire (PDQ-39): development and validation of a Parkinson’s disease summary index score. Age Ageing. 1997;26(5):353–7.

    CAS  PubMed  Article  Google Scholar 

  3. Schrag A, Jahanshahi M, Quinn N. How does Parkinson’s disease affect quality of life? A comparison with quality of life in the general population. Mov Disord. 2000;15(6):1112–8.

    CAS  PubMed  Article  Google Scholar 

  4. Keranen T, Kaakkola S, Sotaniemi K, Laulumaa V, Haapaniemi T, Jolma T, et al. Economic burden and quality of life impairment increase with severity of PD. Parkinsonism Relat Disord. 2003;9(3):163–8.

    CAS  PubMed  Article  Google Scholar 

  5. Dodel RC, Berger K, Oertel WH. Health-related quality of life and healthcare utilisation in patients with Parkinson’s disease: impact of motor fluctuations and dyskinesias. Pharmacoeconomics. 2001;19(10):1013–38.

    CAS  PubMed  Article  Google Scholar 

  6. Hechtner MC, Vogt T, Zollner Y, Schroder S, Sauer JB, Binder H, et al. Quality of life in Parkinson’s disease patients with motor fluctuations and dyskinesias in five European countries. Parkinsonism Relat Disord. 2014;20(9):969–74.

    PubMed  Article  Google Scholar 

  7. Martinez-Martin P, Rodriguez-Blazquez C, Kurtis MM, Chaudhuri KR, Group NV. The impact of non-motor symptoms on health-related quality of life of patients with Parkinson’s disease. Mov Disord. 2011;26(3):399–406.

    PubMed  Article  Google Scholar 

  8. Martinez-Martin P. Nonmotor symptoms and health-related quality of life in early Parkinson’s disease. Mov Disord. 2014;29(2):166–8.

    PubMed  Article  Google Scholar 

  9. Chaudhuri KR, Sauerbier A, Rojo JM, Sethi K, Schapira AH, Brown RG, et al. The burden of non-motor symptoms in Parkinson’s disease using a self-completed non-motor questionnaire: a simple grading system. Parkinsonism Relat Disord. 2015;21(3):287–91.

    PubMed  Article  Google Scholar 

  10. Seppi K, Ray Chaudhuri K, Coelho M, Fox SH, Katzenschlager R, Perez Lloret S, et al. Update on treatments for nonmotor symptoms of Parkinson’s disease-an evidence-based medicine review. Mov Disord. 2019;34(2):180–98.

    PubMed  PubMed Central  Article  Google Scholar 

  11. Barone P, Erro R, Picillo M. Quality of life and nonmotor symptoms in Parkinson’s disease. Int Rev Neurobiol. 2017;133:499–516.

    PubMed  Article  Google Scholar 

  12. Dams J, Bornschein B, Reese JP, Conrads-Frank A, Oertel WH, Siebert U, et al. Modelling the cost effectiveness of treatments for Parkinson’s disease: a methodological review. Pharmacoeconomics. 2011;29(12):1025–49.

    PubMed  Article  Google Scholar 

  13. Afentou N, Jarl J, Gerdtham UG, Saha S. Economic evaluation of interventions in Parkinson’s disease: a systematic literature review. Mov Disord Clin Pract. 2019;6(4):282–90.

    PubMed  PubMed Central  Article  Google Scholar 

  14. Nuijten M, van Iperen P, Palmer C, van Hilten B, Snyder E. Cost-effectiveness analysis of entacapone in Parkinson’s disease: a Markov process analysis. Value Health. 2001;4(4):316–28.

    CAS  PubMed  Article  Google Scholar 

  15. Palmer C, Nuijten M, Schmier J, Subedi P, Snyder E. Cost effectiveness of treatment of Parkinson’s disease with entacapone in the United States. Pharmacoeconomics. 2002;20(9):617–28.

    PubMed  Article  Google Scholar 

  16. Lowin J, Bergman A, Chaudhuri KR, Findley LJ, Roeder C, Schifflers M, et al. A cost-effectiveness analysis of levodopa/carbidopa intestinal gel compared to standard care in late stage Parkinson’s disease in the UK. J Med Econ. 2011;14(5):584–93.

    PubMed  Article  Google Scholar 

  17. Lowin J, Sail K, Baj R, Jalundhwala YJ, Marshall TS, Konwea H, et al. The cost-effectiveness of levodopa/carbidopa intestinal gel compared to standard care in advanced Parkinson’s disease. J Med Econ. 2017;20(11):1207–15.

    PubMed  Article  Google Scholar 

  18. Dams J, Siebert U, Bornschein B, Volkmann J, Deuschl G, Oertel WH, et al. Cost-effectiveness of deep brain stimulation in patients with Parkinson’s disease. Mov Disord. 2013;28(6):763–71.

    PubMed  Article  Google Scholar 

  19. Hoehn MM, Yahr MD. Parkinsonism: onset, progression and mortality. Neurology. 1967;17(5):427–42.

    CAS  PubMed  Article  Google Scholar 

  20. Martinez-Martin P, Jeukens-Visser M, Lyons KE, Rodriguez-Blazquez C, Selai C, Siderowf A, et al. Health-related quality-of-life scales in Parkinson’s disease: critique and recommendations. Mov Disord. 2011;26(13):2371–80.

    PubMed  Article  Google Scholar 

  21. Dodel R, Jonsson B, Reese JP, Winter Y, Martinez-Martin P, Holloway R, et al. Measurement of costs and scales for outcome evaluation in health economic studies of Parkinson’s disease. Mov Disord. 2014;29(2):169–76.

    PubMed  Article  Google Scholar 

  22. Schrag A, Selai C, Jahanshahi M, Quinn N. The EQ-5D—a generic quality of life measure-is a useful instrument to measure quality of life in patients with Parkinson’s disease. J Neurol Neurosurg Psychiatry. 2000;69(1):67–73.

    CAS  PubMed  PubMed Central  Article  Google Scholar 

  23. Siderowf A, Ravina B, Glick H. Preference-based quality-of-life in patients with Parkinson’s disease. Neurology. 2002;59(1):103–8.

    PubMed  Article  Google Scholar 

  24. Garcia-Gordillo MA, Del Pozo-Cruz B, Adsuar JC, Cordero-Ferrera JM, Abellan-Perpinan JM, Sanchez-Martinez FI. Validation and comparison of Eq-5d-3l and Sf-6d instruments in a Spanish Parkinson S disease population sample. Nutr Hosp. 2015;32(6):2808–21.

    PubMed  Google Scholar 

  25. Schrag A, Spottke A, Quinn NP, Dodel R. Comparative responsiveness of Parkinson’s disease scales to change over time. Mov Disord. 2009;24(6):813–8.

    PubMed  Article  Google Scholar 

  26. Brooks R. EuroQol: the current state of play. Health Policy. 1996;37(1):53–72.

    CAS  PubMed  Article  Google Scholar 

  27. EuroQol Group. EuroQol-a new facility for the measurement of health-related quality of life. Health Policy. 1990;16(3):199–208.

    Article  Google Scholar 

  28. Dolan P. Modeling valuations for EuroQol health states. Med Care. 1997;35(11):1095–108.

    CAS  PubMed  Article  Google Scholar 

  29. Burstrom K, Sun S, Gerdtham UG, Henriksson M, Johannesson M, Levin LA, et al. Swedish experience-based value sets for EQ-5D health states. Qual Life Res. 2014;23(2):431–42.

    PubMed  Article  Google Scholar 

  30. Willis M, Persson U, Zoellner Y, Gradl B. Reducing uncertainty in value-based pricing using evidence development agreements: the case of continuous intraduodenal infusion of levodopa/carbidopa (duodopa(R)) in sweden. Appl Health Econ Health Policy. 2010;8(6):377–86.

    PubMed  Article  Google Scholar 

  31. Swinscow TDV, Campbell MJ. Statistics at square one, vol 8. 10th ed. London: BMJ Publishing Group; 2002. p. 158.

    Google Scholar 

  32. Johnson PC. Extension of Nakagawa and Schielzeth’s R(2)(GLMM) to random slopes models. Methods Ecol Evol. 2014;5(9):944–6.

    PubMed  PubMed Central  Article  Google Scholar 

  33. R Core Team. R: a language and environment for statistical computing. R Foundation for Statistical Computing, Vienna, Austria. 2017. https://www.R-project.org/. Accessed 24 Jan 2020.

  34. Kiadaliri AA, Eliasson B, Gerdtham UG. Does the choice of EQ-5D tariff matter? A comparison of the Swedish EQ-5D-3L index score with UK, US, Germany and Denmark among type 2 diabetes patients. Health Qual Life Outcomes. 2015;13:145.

    PubMed  PubMed Central  Article  Google Scholar 

  35. Aronsson M, Husberg M, Kalkan A, Eckard N, Alwin J. Differences between hypothetical and experience-based value sets for EQ-5D used in Sweden: implications for decision makers. Scand J Public Health. 2015;43(8):848–54.

    PubMed  Article  Google Scholar 

  36. Ubel PA, Loewenstein G, Jepson C. Whose quality of life? A commentary exploring discrepancies between health state evaluations of patients and the general public. Qual Life Res. 2003;12(6):599–607.

    PubMed  Article  Google Scholar 

  37. Martinez-Martin P, Rodriguez-Blazquez C, Forjaz MJ, Alvarez-Sanchez M, Arakaki T, Bergareche-Yarza A, et al. Relationship between the MDS-UPDRS domains and the health-related quality of life of Parkinson’s disease patients. Eur J Neurol. 2014;21(3):519–24.

    CAS  PubMed  Article  Google Scholar 

  38. Shearer J, Green C, Counsell CE, Zajicek JP. The impact of motor and non motor symptoms on health state values in newly diagnosed idiopathic Parkinson’s disease. J Neurol. 2012;259(3):462–8.

    PubMed  Article  Google Scholar 

  39. Vossius C, Nilsen OB, Larsen JP. Health state values during the first year of drug treatment in early-stage Parkinson’s disease: a prospective, population-based, cohort study. Drugs Aging. 2009;26(11):973–80.

    PubMed  Article  Google Scholar 

  40. Reuther M, Spottke EA, Klotsche J, Riedel O, Peter H, Berger K, et al. Assessing health-related quality of life in patients with Parkinson’s disease in a prospective longitudinal study. Parkinsonism Relat Disord. 2007;13(2):108–14.

    CAS  PubMed  Article  Google Scholar 

  41. Antonini A, Barone P, Marconi R, Morgante L, Zappulla S, Pontieri FE, et al. The progression of non-motor symptoms in Parkinson’s disease and their contribution to motor disability and quality of life. J Neurol. 2012;259(12):2621–31.

    PubMed  Article  Google Scholar 

  42. Chandler C, Alvarez P, Folse H, Ward A. The association between utilities and disease severity for Parkinson’s disease: a systematic literature review. In: Presented at ISPOR European Conference November 10–14, 2018; Barcelona, Spain, 2018.

  43. Palmer C, Schmier J, Snyder E, Scott B. Patient preferences and utilities for ‘off-time’ outcomes in the treatment of Parkinson’s disease. Qual Life Res. 2000;9(7):819–27.

    CAS  PubMed  Article  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Jenny M. Norlin.

Ethics declarations

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.

Consent for publication

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.

Supplementary Information

Below is the link to the electronic supplementary material.

Supplementary file1 (PDF 116 kb)

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

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

Download citation

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s40273-021-01056-z