Health Utility and Indirect Costs of Neuromuscular Disease Through a Patient-Centered Lens: A National Patient and Caregiver Survey
Author(s)
Alyssa Grant, MSc, PhD1, Ian C. Smith, PhD2, Lola Lessard3, Homira Osman, PhD4, Hanns Lochmuller, MD5, Hugh McMillan, MD6, Gerald Pfeffer, MD7, Lawrence Korngut, MD8, Cynthia Gagnon, MD9, Stacey Lintern, RN10, Kathy Selby, MD11, Kednapa Thavorn, PhD2, Jodi Warman-Chardon, MD PhD5.
1Post-doctoral fellow, Ottawa Hospital Research Institute, Ottawa, ON, Canada, 2Ottawa Hospital Research Institute, Ottawa, ON, Canada, 3France, 4Muscular Dystrophy Canada, East Gwillimbury, ON, Canada, 5The Ottawa Hospital, Ottawa, ON, Canada, 6Faculty of Medicine, University of Ottawa, Ottawa, ON, Canada, 7Department of Clinical Neurosciences, University of Calgary,, Calgary, AB, Canada, 8Department of Clinical Neurosciences, University of Calgary, Calgary, AB, Canada, 9École de réadaptation site Saguenay/clinique des maladies neuromusculaires, Jonquière, QC, Canada, 10Muscular Dystrophy Canada, Toronto, ON, Canada, 11British Columbia Children’s Hospital, Vancouver, BC, Canada.
1Post-doctoral fellow, Ottawa Hospital Research Institute, Ottawa, ON, Canada, 2Ottawa Hospital Research Institute, Ottawa, ON, Canada, 3France, 4Muscular Dystrophy Canada, East Gwillimbury, ON, Canada, 5The Ottawa Hospital, Ottawa, ON, Canada, 6Faculty of Medicine, University of Ottawa, Ottawa, ON, Canada, 7Department of Clinical Neurosciences, University of Calgary,, Calgary, AB, Canada, 8Department of Clinical Neurosciences, University of Calgary, Calgary, AB, Canada, 9École de réadaptation site Saguenay/clinique des maladies neuromusculaires, Jonquière, QC, Canada, 10Muscular Dystrophy Canada, Toronto, ON, Canada, 11British Columbia Children’s Hospital, Vancouver, BC, Canada.
OBJECTIVES: Health state utility values and cost estimates are essential for economic evaluations of interventions for neuromuscular diseases (NMDs), yet robust reference data remain scarce. This study aimed to (1) describe health utility scores and both direct non-medical and indirect costs among individuals with inherited and acquired NMDs, and (2) examine sociodemographic, clinical, and economic predictors of health utility.
METHODS: We conducted a national cross-sectional survey of 1,454 included individuals with NMD or their caregivers registered with Muscular Dystrophy Canada. Participants completed the Health Utilities Index Mark 3 (HUI3; range: -0.36 to 1), Work Productivity and Activity Impairment or Caregiver Indirect and Informal Care Cost Assessment Questionnaires. For minors, parent-proxy responses were obtained using the Pediatric Quality of Life Inventory (PedsQL) 3.0 Neuromuscular Module (range: 0-100). Self-reported direct non-medical costs included medications, assistive devices, home/vehicle modifications, and transportation. Costs were presented in 2024 Canadian Dollars. Multivariable linear regression was used to identify predictors of health utility.
RESULTS: The mean HUI3 score was 0.27 (standard deviation (SD): 0.31) among adults and caregivers with NMD. For minors, the mean PedsQL was 45.9 (SD: 18.7). Among adults, lower health utility scores were independently associated with longer disease duration, specific NMD subtypes, inability to work due to NMD, being single, widowed, divorced, or separated, and greater financial toxicity (p<0.05). In minors, lower health utility was significantly associated with financial toxicity and school absenteeism (p<0.01). Mean annual direct non-medical costs were $3,270, while productivity losses were $25,688 for patients and $48,698 and caregivers.
CONCLUSIONS: NMDs impose substantial health and economic burdens. Reduced utility scores and high non-medical and productivity-related costs underscore the importance of incorporating disease subtype, duration, financial strain, and social determinants into future economic evaluations.
METHODS: We conducted a national cross-sectional survey of 1,454 included individuals with NMD or their caregivers registered with Muscular Dystrophy Canada. Participants completed the Health Utilities Index Mark 3 (HUI3; range: -0.36 to 1), Work Productivity and Activity Impairment or Caregiver Indirect and Informal Care Cost Assessment Questionnaires. For minors, parent-proxy responses were obtained using the Pediatric Quality of Life Inventory (PedsQL) 3.0 Neuromuscular Module (range: 0-100). Self-reported direct non-medical costs included medications, assistive devices, home/vehicle modifications, and transportation. Costs were presented in 2024 Canadian Dollars. Multivariable linear regression was used to identify predictors of health utility.
RESULTS: The mean HUI3 score was 0.27 (standard deviation (SD): 0.31) among adults and caregivers with NMD. For minors, the mean PedsQL was 45.9 (SD: 18.7). Among adults, lower health utility scores were independently associated with longer disease duration, specific NMD subtypes, inability to work due to NMD, being single, widowed, divorced, or separated, and greater financial toxicity (p<0.05). In minors, lower health utility was significantly associated with financial toxicity and school absenteeism (p<0.01). Mean annual direct non-medical costs were $3,270, while productivity losses were $25,688 for patients and $48,698 and caregivers.
CONCLUSIONS: NMDs impose substantial health and economic burdens. Reduced utility scores and high non-medical and productivity-related costs underscore the importance of incorporating disease subtype, duration, financial strain, and social determinants into future economic evaluations.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR109
Topic
Clinical Outcomes, Epidemiology & Public Health, Patient-Centered Research
Topic Subcategory
Health State Utilities
Disease
Neurological Disorders