The Burden-of-Illness of Duchenne Muscular Dystrophy in Japan: A Socioeconomic Survey

Speaker(s)

Shimizu-Motohashi Y1, Merla V2, Nagano M3, Shima D4, Posner N2, Evans J5
1National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan, 2Pfizer Inc., New York, NY, USA, 3Pfizer Japan Inc., Shibuya-ku, 13, Japan, 4Pfizer Japan Inc., Tokyo, Japan, 5HCD Economics, Knutsford, UK

OBJECTIVES: The objective of this study was to estimate the average per-patient cost, including direct medical, direct non-medical, and indirect cost of Duchenne muscular dystrophy (DMD) from a societal perspective in Japan, as well as to estimate the health-related quality of life (HRQoL) impact of DMD in Japan on patients and caregivers.

METHODS: Neuromuscular specialists (surveyed during March 2023) completed an electronic case report form (eCRF) capturing demographic, clinical, and direct medical costs over the last 12 months for each patient. Patients and their caregivers were recruited via the Japan Muscular Dystrophy Association to complete Patient Public Involvement Engagement questionnaires (PPIE), which collected demographic information as well as direct non-medical (e.g alternative therapies, home adaptations) and indirect costs over the past 12 months, and cross-sectional Patient Reported Outcome Measure (PROMs), including HRQoL via the EQ5D, DMD-QoL, and CarerQoL.

RESULTS: Physicians completed eCRFs for 63 patients, with 38 PPIE questionnaires also completed. The mean age and proportion of patients ambulatory was 15.9 years and 35% respectively for the eCRF sample; and 17.5 years and 47% for the PPIE sample Mean direct medical, direct non-medical, and indirect cost (both patient and caregiver) were estimated at ¥814,492 ($5,818), ¥5,849,454 ($41,783), and ¥1,136,346 ($8,117) respectively. Mean societal cost of DMD over the past 12 months was ¥7,800,292 ($49,771) and was highly influenced by informal care, comprising 61% of all cost. Mean HRQoL index score was reported at 0.79 (EQ-5D-Y proxy), 0.75 (EQ-5D-Y), 0.33 (EQ-5D-5L), 0.70 (DMD-QoL proxy), and 0.50 (DMD-QoL patient). Caregivers reported mean HRQoL index scores of 0.78 (EQ5D-5L), and 68.11 (CarerQoL).

CONCLUSIONS: This is the most recent and most detailed study exploring the burden of DMD in Japan, and highlights the important contribution patient and caregiver costs have on societal cost. HRQoL results also demonstrate the low levels of HRQoL of DMD patients, particularly during adulthood.

Code

EE472

Topic

Economic Evaluation, Patient-Centered Research, Study Approaches

Topic Subcategory

Electronic Medical & Health Records, Patient-reported Outcomes & Quality of Life Outcomes, Work & Home Productivity - Indirect Costs

Disease

Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Neurological Disorders, Rare & Orphan Diseases