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 

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 

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 

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 

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

Article  PubMed  Google Scholar 

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

Article  CAS  PubMed  Google Scholar 

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 

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 

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 

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 

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 

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 

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 

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 

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.

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.

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.

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.

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 

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 

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 

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 

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 

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 

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 

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 

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 

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 

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

Article  PubMed  Google Scholar 

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 

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 

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 

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

Article  PubMed  Google Scholar