Lunan M, Hartley L, Pearson I, Doyle S. Targeted literature review of the burden of illness in late-stage Duchenne Muscular Dystrophy (DMD). Poster presented at the Virtual ISPOR Europe 2021; November 3, 2021. [abstract] Value Health. 2021 Dec; 24(12):S2.

OBJECTIVES : DMD is a rare, fatal genetic disease typically diagnosed before 5 years of age and characterized by motor dysfunction. In later stages of the disease, cardiac management and respiratory support are typically needed. Although the average age of death has increased with evolving clinical management, most adults with DMD will not live beyond the fifth decade of life. This research aimed to establish the unmet need and burden of illness in late-stage DMD.

METHODS : We conducted targeted literature searches of the Embase database (2010-2020) and international health technology assessment and conference websites (2018-2020). Searches for late-stage (ie, advanced, terminal, nonambulatory, or adult/adolescent) DMD were combined with search strings for resource use and costs and clinical and humanistic burden. English-language publications including children or adults were selected, regardless of geography. Animal studies, commentaries, letters, and studies not reporting the outcomes of interest were excluded.

RESULTS : Of 1,727 records identified, data were extracted from 80 studies. There was considerable literature on the economic (n=13 studies) and humanistic (n=23 studies) burden of DMD. Costs were generally shown to increase steadily by age group for health systems, with high variability of care and per-patient costs across different geographical regions (eg, Eastern vs Western Europe). Costs and healthcare resource use increased with disease progression. Loss of ambulation and need for ventilation support were associated with substantial economic and humanistic consequences, including high costs for ventilation support, formal and informal care, mobility tools (eg, wheelchairs, vehicle modifications), and home modifications, as well as impaired patient quality of life.

CONCLUSIONS : Late-stage DMD is associated with a substantial impact on patients, their families, and society. Although life expectancy is increasing, better treatments are needed to address the burden and unmet need in DMD.

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