OBJECTIVES:

Duchenne muscular dystrophy (DMD) is a rare degenerative neuromuscular disease resulting in progressive muscle weakness, loss of functional ability, and premature death. Disease progression, especially loss of ambulation, impacts work productivity for patients with DMD and caregivers, particularly in relation to loss of income and work years. The objective of this study was to quantify the lifetime loss of income and work years for patients with DMD and their caregivers based on the natural history progression of DMD.

METHODS:

A lifetime cost model was developed using a 5-state partitioned survival model to characterize progression of DMD. Health states included early and late ambulatory, early and late non-ambulatory, and death. Work productivity of patients with DMD and their caregivers was calculated and compared with that of the general US population to estimate lifetime income and work years lost. Salaries were adjusted for annual salary growth by 2.72%, and a discount rate of 3% per annum was applied to potential earnings.

RESULTS:

Patients with DMD had a substantially reduced number of work years relative to the general population (0.86 vs 35.78 years), resulting in an estimated lifetime loss of income of $1.9 million. Caregivers lost more than 4 work years over the patients’ lifetime, incurring lifetime earning potential losses of approximately $165,000 on average. The results were robust, with scenario and sensitivity analyses suggesting that work opportunity loss is driven by early progression to loss of ambulation as well as mortality.

CONCLUSIONS:

These results underscore the significant negative impact on work productivity and the associated financial burden that DMD has on patients and caregivers.