Medical costs for Duchenne muscular dystrophy (DMD) rise sharply with advancing disease stages, according to a report published in the Orphanet Journal of Rare Diseases.
An analysis of German health care claims data revealed that direct medical costs escalate as patients’ conditions progress. The increased costs are driven by the need for orthopedic surgeries, physical therapy, pharmaceutical prescriptions and more specialized medical equipment such as leg braces, walkers, wheelchairs and ventilators.
The authors identified 134 patients with DMD between 2016 and 2021 for whom they could determine the disease stage. Their aim was to estimate the prevalence of DMD in Germany and assess healthcare resource use, disease burden and direct costs.
The authors assigned each patient to one of four stages based on disease progression. Stage 4 (late nonambulatory) was the largest group, with about 47% of the identified patients; stage 3 (early nonambulatory) included 37% of patients; stage 2 (late ambulatory) included 34%; and stage 1 (early ambulatory) included just 4% of patients.
The team noted that in stage 2, health care claims codes reflected the need for mobility support and orthopedic surgeries. In stage 3, bedsore prevention emerged and increased to about 50% of stage 4 patients. Average direct costs per patient per year significantly increased with advancing disease stage, with mobility aids accounting for about half of all costs in stages 3 and 4.
“The economic cost of DMD increases drastically as patients begin to lose ambulation and require more intensive care, often around age 8–12,” the authors wrote. “The very high use of medical aids in our disease stage 4 group (including decubitus prevention aids and ventilation) reflects the severity of a DMD disease course.”
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The authors noted that U.S. families often face greater out-of-pocket costs and gaps in equipment coverage than individuals covered by Germany’s public health insurance system.
While this study focused on direct costs, the authors noted that indirect costs — including caregiver time, loss of productivity and reduced quality of life — can be even higher than direct costs, and both increase substantially as the disease advances. In fact, one German study reported mean per-patient annual direct costs in 2012 of about $11,000 per year, but indirect costs amounted to more than $45,000.
The team concluded that planning care based on the stage of DMD is essential, both for managing symptoms and for preparing families for the increasing support their child will need.
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