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Economic Evaluations of Treatments for Duchenne Muscular Dystrophy: The Caregiver QALY Trap

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References

  1. Adelman RD, Tmanova LL, Delgado D, Dion S, Lachs MS. Caregiver burden: a clinical review. JAMA. 2014;311:1052–60.

    Article  CAS  PubMed  Google Scholar 

  2. Schulz R, Beach SR. Caregiving as a risk factor for mortality: the Caregiver Health Effects Study. JAMA. 1999;282:2215–9.

    Article  CAS  PubMed  Google Scholar 

  3. Gouin JP, Hantsoo L, Kiecolt-Glaser JK. Immune dysregulation and chronic stress among older adults: a review. NeuroImmunoModulation. 2008;15:251–9.

    Article  CAS  PubMed  Google Scholar 

  4. Lee S, Colditz GA, Berkman LF, Kawachi I. Caregiving and risk of coronary heart disease in U.S. women: a prospective study. Am J Prev Med. 2003;24:113–9.

    Article  PubMed  Google Scholar 

  5. Hirst M. Carer distress: a prospective, population-based study. Soc Sci Med. 2005;61(3):697–708.

    Article  PubMed  Google Scholar 

  6. Emery AE. The muscular dystrophies. Lancet. 2002;359(9307):687–95.

    Article  CAS  PubMed  Google Scholar 

  7. Landfeldt E, Thompson R, Sejersen T, McMillan HJ, Kirschner J, Lochmüller H. Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis. Eur J Epidemiol. 2020;35(7):643–53.

    Article  PubMed  PubMed Central  Google Scholar 

  8. McDonald CM, Henricson EK, Abresch RT, Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP, Cnaan A, Gordish-Dressman H, CINRG Investigators. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet. 2018;391(10119):451–61.

    Article  CAS  PubMed  Google Scholar 

  9. Landfeldt E, Edström J, Buccella F, Kirschner J, Lochmüller H. Duchenne muscular dystrophy and caregiver burden: a systematic review. Dev Med Child Neurol. 2018;60(10):987–96.

    Article  PubMed  Google Scholar 

  10. Landfeldt E, Lindgren P, Bell CF, Guglieri M, Straub V, Lochmüller H, Bushby K. Quantifying the burden of caregiving in Duchenne muscular dystrophy. J Neurol. 2016;263(5):906–15.

    Article  PubMed  PubMed Central  Google Scholar 

  11. Landfeldt E, Lindgren P, Bell CF, Schmitt C, Guglieri M, Straub V, Lochmüller H, Bushby K. The burden of Duchenne muscular dystrophy: an international, cross-sectional study. Neurology. 2014;83(6):529–36.

    Article  PubMed  PubMed Central  Google Scholar 

  12. Pennington B, Eaton J, Hatswell AJ, Taylor H. Carers’ health-related quality of life in global health technology assessment: guidance, case studies and recommendations. Pharmacoeconomics. 2022;40(9):837–50.

    Article  PubMed  PubMed Central  Google Scholar 

  13. Lamsal R, Yeh EA, Pullenayegum E, Ungar WJ. A systematic review of methods used by pediatric cost-utility analyses to include family spillover effects. Pharmacoeconomics. 2024;42(2):199–217.

    Article  PubMed  Google Scholar 

  14. Scope A, Bhadhuri A, Pennington B. Systematic Review of Cost-Utility Analyses That Have Included Carer and Family Member Health-Related Quality of Life. Value Health. 2022;25(9):1644–53.

    Article  PubMed  Google Scholar 

  15. Canada's Drug and Health Technology Agency (CADTH). Guidelines for the Economic Evaluation of Health Technologies: Canada – 4th Edition. Available from: https://www.cadth.ca/guidelines-economic-evaluation-health-technologies-canada-4th-edition. Accessed 22 Dec 2023.

  16. Zorginstituut Nederlands (ZIN). Guidelines for economic evaluations in Healthcare. Available from: https://english.zorginstituutnederland.nl/about-us/publications/reports/2016/06/16/guideline-for-economic-evaluations-in-healthcare. Accessed 22 Dec 2023.

  17. National Institute for Health and Care Excellence (NICE). Health technology evaluations: the manual. Available from: https://www.nice.org.uk/process/pmg36/chapter/economic-evaluation. Accessed 22 Dec 2023.

  18. Institute for Clinical and Economic Review (ICER). 2020-2023 Value Assessment Framework. Available from: https://icer.org/wp-content/uploads/2020/10/ICER_2020_2023_VAF_102220.pdf. Accessed 22 Dec 2023.

  19. Mott DJ, Schirrmacher H, Al-Janabi H, Guest S, Pennington B, Scheuer N, Shah KK, Skedgel C. Modelling spillover effects on informal carers: the Carer QALY Trap. Pharmacoeconomics. 2023;41(12):1557–61.

    Article  PubMed  PubMed Central  Google Scholar 

  20. Landfeldt E. Gene therapy for neuromuscular diseases: health economic challenges and future perspectives. J Neuromuscul Dis. 2022;9(6):675–88.

    Article  PubMed  PubMed Central  Google Scholar 

  21. Landfeldt E, Alfredsson L, Straub V, Lochmüller H, Bushby K, Lindgren P. Economic evaluation in Duchenne muscular dystrophy: model frameworks for cost-effectiveness analysis. Pharmacoeconomics. 2017;35(2):249–58.

    Article  PubMed  Google Scholar 

  22. Song J, Floyd FJ, Seltzer MM, Greenberg JS, Hong J. Long-term Effects of Child Death on Parents’ Health Related Quality of Life: A Dyadic Analysis. Fam Relat. 2010;59(3):269–82.

    Article  PubMed  PubMed Central  Google Scholar 

  23. Song J, Mailick MR, Greenberg JS, Floyd FJ. Mortality in parents after the death of a child. Soc Sci Med. 2019;239: 112522.

    Article  PubMed  PubMed Central  Google Scholar 

  24. Rogers CH, Floyd FJ, Seltzer MM, Greenberg J, Hong J. Long-term effects of the death of a child on parents’ adjustment in midlife. J Fam Psychol. 2008;22(2):203–11.

    Article  PubMed  PubMed Central  Google Scholar 

  25. Donnelly CM, Quinlivan RM, Herron A, Graham CD. A systematic review and qualitative synthesis of the experiences of parents of individuals living with Duchenne muscular dystrophy. Disabil Rehabil. 2023;45(8):1285–98.

    Article  PubMed  Google Scholar 

  26. Tilford JM, Tarlan A. The carer QALY trap and altruism in economic evaluations. Pharmacoeconomics. 2023;41(12):1553–5. https://doi.org/10.1007/s40273-023-01324-0. (Epub 2023 Nov 2 PMID: 37917418).

    Article  PubMed  Google Scholar 

  27. Riley AG, Mulé CM, Lerner D, Belter L, O’Toole CM, Kowal S, Fox D, Shapouri S, Vesel T, Lavelle TA. Assessing the impact of grief on quality of life, work productivity, and health outcomes for parents bereaved from SMA: A study protocol. Cost Eff Resour Alloc. 2023;21(1):55.

    Article  PubMed  PubMed Central  Google Scholar 

  28. Landfeldt E. Extending life in duchenne muscular dystrophy: implications for appraisals of cost-effectiveness. Pharmacoecon Open. 2019;3(3):279–80.

    Article  PubMed  Google Scholar 

  29. Brouwer WB. Too important to ignore: informal caregivers and other significant others. Pharmacoeconomics. 2006;24(1):39–41.

    Article  PubMed  Google Scholar 

  30. Koopmanschap MA, van Exel JN, van den Berg B, Brouwer WB. An overview of methods and applications to value informal care in economic evaluations of healthcare. Pharmacoeconomics. 2008;26(4):269–80.

    Article  PubMed  Google Scholar 

  31. van den Berg B, Brouwer WB, Koopmanschap MA. Economic valuation of informal care. An overview of methods and applications. Eur J Health Econ. 2004;5(1):36–45.

    Article  PubMed  Google Scholar 

  32. Al-Janabi H, van Exel J, Brouwer W, Coast J. A framework for including family health spillovers in economic evaluation. Med Decis Mak. 2016;36:176–86.

    Article  Google Scholar 

  33. Brouwer WBF. The inclusion of spillover effects in economic evaluations: not an optional extra. Pharmacoeconomics. 2019;37:451–6.

    Article  PubMed  Google Scholar 

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Correspondence to Erik Landfeldt.

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Dr Erik Landfeldt and Mr Hera Sandhu have conducted research for pharmaceutical companies marketing treatments for DMD.

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Landfeldt, E., Sandhu, H. Economic Evaluations of Treatments for Duchenne Muscular Dystrophy: The Caregiver QALY Trap. PharmacoEconomics 42, 475–478 (2024). https://doi.org/10.1007/s40273-024-01367-x

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