Evaluating New Zealanders’ Values for Drug Coverage Decision Making: Trade-Offs between Treatments for Rare and Common Conditions

Abstract

Background

New Zealand’s near static healthcare budget limits access to expensive medications including those for rare conditions. As such, it is necessary to know the public’s priority for values in the drug funding decision-making process.

Objectives

The objectives of this study were to measure the relative societal importance of values of New Zealanders in informing drug funding decisions and to determine how New Zealanders trade off funding in various scenarios between common and rare diseases.

Methods

An online survey was conducted between 17 April and 17 May, 2019 on a sample of 500 New Zealanders aged ≥ 18 years. Participants ranked 13 values using an analytical hierarchy process. Participants were then presented with different trade-off scenarios to measure their attitudes towards funding drugs for common and rare diseases.

Results

The values ranked in the top five by most were potential effect on quality of life (71.8%), ability of the drug to work (57.6%), severity (57.6%), safety (57%), and potential to extend life (56%). Adherence and rarity held the lowest and second lowest ranking. Most believe that resources should be allocated towards drugs that have been proven to work and have the greatest health benefits. In trade-offs between access to an expensive drug therapy for a rare disease with uncertain benefits or receive a fixed cash payment, the overwhelming consensus was to receive the cash payment.

Conclusions

New Zealanders ultimately value drug-related factors (e.g. quality of life and efficacy) and disease-related factors (e.g. severity of disease and equity) the most but did not value disease rarity.

This is a preview of subscription content, log in to check access.

Fig. 1
Fig. 2
Fig. 3
Fig. 4

References

  1. 1.

    Leopold C, Morgan SG, Wagner AK. A rapidly changing global medicines environment: how adaptable are funding decision-making systems? Health Policy. 2017;121(6):637–43.

    Article  PubMed  PubMed Central  Google Scholar 

  2. 2.

    EiCM L. The price of drugs for chronic myeloid leukemia (CML) is a reflection of the unsustainable prices of cancer drugs: from the perspective of a large group of CML experts. Blood. 2013;121(22):4439–42.

    Article  Google Scholar 

  3. 3.

    Gammie T, Seyfoddin A, Hasan SS, Curley LE. Patient access to medicines in two countries with similar health systems and differing medicines policies: implications from a comprehensive literature review. Res Soc Adm Pharm. 2019;15(3):231–43.

    Article  Google Scholar 

  4. 4.

    Trooskin SB, Reynolds H, Kostman JR. Access to costly new hepatitis C drugs: medicine, money, and advocacy. Clin Infect Dis. 2015;61(12):1825–30.

    Article  PubMed  Google Scholar 

  5. 5.

    Daniel H. Stemming the escalating cost of prescription drugs: a position paper of the American College of Physicians. Ann Intern Med. 2016;165(1):50–2.

    Article  PubMed  Google Scholar 

  6. 6.

    Cumming J, Mays N, Daubé J. How New Zealand has contained expenditure on drugs. BMJ. 2010;340:c2441.

    Article  PubMed  Google Scholar 

  7. 7.

    Davis P. “Tough but fair”? The active management of the New Zealand drug benefits scheme by an independent Crown agency. Aust Health Rev. 2004;28(2):171–81.

    Article  PubMed  Google Scholar 

  8. 8.

    PHARMAC. Combined pharmaceutical budget [CPB]. PHARMAC; 2020. https://pharmac.govt.nz/medicine-funding-and-supply/the-funding-process/setting-and-managing-the-combined-pharmaceutical-budget-cpb/. Accessed 2 Nov 2020.

  9. 9.

    PHARMAC. Introduction to PHARMAC. PHARMAC; 2020. https://pharmac.govt.nz/about/what-we-do/welcome-to-pharmac/. Accessed 2 Nov 2020.

  10. 10.

    PHARMAC. Pharmaceutical Management Agency annual report for the year ended 30 June 2018. 2018. https://www.pharmac.govt.nz/assets/annual-report-2017-2018.pdf. Accessed 20 Oct 2020.

  11. 11.

    PHARMAC. Making funding decisions. 2018. https://www.pharmac.govt.nz/assets/factsheet-04-making-funding-decisions.pdf. Accessed 20 Oct 2020.

  12. 12.

    PHARMAC. 1 Background. PHARMAC; 2020. https://pharmac.govt.nz/medicine-funding-and-supply/the-funding-process/policies-manuals-and-processes/economic-analysis/prescription-for-pharmacoeconomic-analysis-methods-for-cost-utility-analysis/1-background/. Accessed 2 Nov 2020.

  13. 13.

    PHARMAC. 2 Health economic analysis at PHARMAC. PHARMAC; 2018. https://pharmac.govt.nz/medicine-funding-and-supply/the-funding-process/policies-manuals-and-processes/economic-analysis/prescription-for-pharmacoeconomic-analysis-methods-for-cost-utility-analysis/2-health-economic-analysis-at-pharmac/. Accessed 2 Nov 2020.

  14. 14.

    PHARMAC. Cost-utility analysis (CUA) explained. 2018. https://www.pharmac.govt.nz/assets/cost-utility-analysis-explained-2015-08.pdf. Accessed 20 Oct 2020.

  15. 15.

    Taylor C, Wonder M. Exploring the implications of a fixed budget for new medicines: a study of reimbursement of new medicines in Australia and New Zealand. Aust Health Rev. 2015;39(4):455–61.

    Article  Google Scholar 

  16. 16.

    Schuller Y, Hollak C, Biegstraaten M. The quality of economic evaluations of ultra-orphan drugs in Europe: a systematic review. Orphanet J Rare Dis. 2015;10:92.

    CAS  Article  PubMed  PubMed Central  Google Scholar 

  17. 17.

    Richter T, Nestler-Parr S, Babela R, Khan ZM, Tesoro T, Molsen E, Hughes DA. Rare disease terminology and definitions: a systematic global review: report of the ISPOR Rare Disease Special Interest Group. Value Health. 2015;18(6):906–14.

    Article  PubMed  PubMed Central  Google Scholar 

  18. 18.

    Hughes DA, Poletti-Hughes J. Profitability and market value of orphan drug companies: a retrospective, propensity-matched case-control study. PLoS ONE. 2016;11(10):e0164681.

    Article  PubMed  PubMed Central  Google Scholar 

  19. 19.

    PHARMAC. Funding for rare disorders. 2018. https://www.pharmac.govt.nz/medicines/how-medicines-are-funded/medicines-for-rare-disorders/. Accessed 30 Oct 2020.

  20. 20.

    PHARMAC. High cost medicines for rare disorders. PHARMAC; 2018. https://pharmac.govt.nz/news-and-resources/research/high-cost-of-medicines/. Accessed 2 Nov 2020.

  21. 21.

    Gericke CA, Riesberg A, Busse R. Ethical issues in funding orphan drug research and development. J Med Ethics. 2005;31(3):164–8. https://doi.org/10.1136/jme.2003.007138.

    CAS  Article  PubMed  PubMed Central  Google Scholar 

  22. 22.

    Cannizzo S, Lorenzoni V, Palla I, Pirri S, Triulzi I, Turchetti G. Rare diseases under different levels of economic analysis: current activities, challenges and perspectives. RMD Open. 2018;4(Suppl. 1):e000794.

    Article  PubMed  PubMed Central  Google Scholar 

  23. 23.

    Rizzardo S, Bansback N, Dragojlovic N, Douglas C, Li KH, Mitton C, et al. Evaluating Canadians’ values for drug coverage decision making. Value Health. 2019;22(3):362–9.

    Article  PubMed  Google Scholar 

  24. 24.

    Desser AS, Gyrd-Hansen D, Olsen JA, Grepperud S, Kristiansen IS. Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67. BMJ. 2010;341:c4715.

    Article  PubMed  PubMed Central  Google Scholar 

  25. 25.

    Dionne F, Mitton C, Dempster B, Lynd LD. Developing a multi-criteria approach for drug reimbursement decision making: an initial step forward. J Popul Ther Clin Pharmacol. 2015;22(1):e68-77.

    PubMed  Google Scholar 

  26. 26.

    Friedmann C, Levy P, Hensel P, Hiligsmann M. Using multi-criteria decision analysis to appraise orphan drugs: a systematic review. Expert Rev Pharmacoecon Outcomes Res. 2018;18(2):135–46.

    Article  PubMed  Google Scholar 

  27. 27.

    Paulden M, Stafinski T, Menon D, McCabe C. Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework. Pharmacoeconomics. 2015;33(3):255–69.

    Article  PubMed  Google Scholar 

  28. 28.

    Bourke SM, Plumpton CO, Hughes DA. Societal preferences for funding orphan drugs in the United Kingdom: an application of person trade-off and discrete choice experiment methods. Value Health. 2018;21:538–46.

    Article  PubMed  Google Scholar 

  29. 29.

    Aotearoa Stats NZ Tatauranga Aotearoa. National population estimates: at 30 June 2018. 2018. https://www.stats.govt.nz/information-releases/national-population-estimates-at-30-june-2018. Accessed 30 Oct 2020.

  30. 30.

    Schmidt K, Aumann I, Hollander I, Damm K, von der Schulenburg J-MG. Applying the analytic hierarchy process in healthcare research: a systematic literature review and evaluation of reporting. BMC Med Inform Decis Mak. 2015;15:112. https://doi.org/10.1186/s12911-015-0234-7.

    Article  PubMed  PubMed Central  Google Scholar 

  31. 31.

    Dolan JG. Shared decision-making: transferring research into practice: the analytic hierarchy process (AHP). Patient Educ Couns. 2008;73(3):418–25.

    Article  PubMed  PubMed Central  Google Scholar 

  32. 32.

    Dolan JG, Isselhardt BJ, Cappuccio JD. The analytic hierarchy process in medical decision making: a tutorial. Med Decis Mak. 1989;9(1):40–50.

    CAS  Article  Google Scholar 

  33. 33.

    Green C. Investigating public preferences on ‘severity of health’ as a relevant condition for setting healthcare priorities. Soc Sci Med. 2009;68(12):2247–55.

    Article  PubMed  PubMed Central  Google Scholar 

  34. 34.

    Lim MK, Bae EY, Choi S-E, Lee EK, Lee T-J. Eliciting public preference for health-care resource allocation in South Korea. Value Health. 2012;15(1 Suppl.):S91–4.

    Article  PubMed  PubMed Central  Google Scholar 

  35. 35.

    Sullivan T, Hansen P. Determining criteria and weights for prioritizing health technologies based on the preferences of the general population: a New Zealand pilot study. Value Health. 2017;20(4):679–86. https://doi.org/10.1016/j.jval.2016.12.008 (Epub 27 Jan 2017).

    Article  PubMed  PubMed Central  Google Scholar 

Download references

Author information

Affiliations

Authors

Corresponding author

Correspondence to Carlo A. Marra.

Ethics declarations

Funding

This research was funded by the University of Otago.

Conflicts of Interest/Competing Interests

Linda Yamoah, Nick Dragojlovic, Alesha Smith, Larry D. Lynd and Carlo A. Marra have no conflicts of interest that are directly relevant to the content of this study.

Ethics Approval

This study was approved by the University of Otago Human Ethics Committee (D19/048).

Consent to Participate

Informed consent was sought prior to completion of the online survey.

Consent for Publication

Informed consent for publication was sought.

Availability of Data and Material

As consent was not sought for sharing the data, we are unable to share the data from the survey.

Code Availability

We can share the R code for the analytic hierarchy process upon request.

Authors’ Contributions

Linda Yamoah was a BPharm(Hons) student and adapted the survey from the Canadian study, analysed the data and drafted the manuscript. This manuscript is an adaptation of her dissertation. Nick Dragojlovic was an investigator on the Canadian survey and helped with the data analysis, reviewed the manuscript for publication and helped respond to reviewers’ comments. Alesha Smith was a co-supervisor of Linda Yamoah and reviewed the manuscript. Larry Lynd was the primary investigator of the Canadian study and reviewed the manuscript and helped with reviewers’ comments. Carlo Marra was the primary investigator of the study, supervised Linda Yamoah, helped analyse the data, reviewed the manuscript and responded to the reviewers’ comments.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Yamoah, L., Dragojlovic, N., Smith, A. et al. Evaluating New Zealanders’ Values for Drug Coverage Decision Making: Trade-Offs between Treatments for Rare and Common Conditions. PharmacoEconomics (2020). https://doi.org/10.1007/s40273-020-00974-8

Download citation