Estimating Societal Cost of Illness and Patients' Quality of Life of Duchenne Muscular Dystrophy in Egypt.

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that causes substantial economic burden. This study aims to measure the DMD cost from societal perspective and the quality of life (QOL) of the Egyptian patients. We conducted interviews with caregivers of patients with DMD. The questionnaire included demographics, healthcare resource use, and nonmedical and indirect costs. Total disease burden was estimated with a bottom-up approach. QOL was measured with a disease-specific tool. Costs and utilities were stratified by the disease stage. Caregivers of 97 patients with DMD were interviewed. The mean annual per-patient cost of $17 485 (SD ± 9240) was estimated resulting in a total burden of $138 217 043 in Egypt. Nonmedical costs made up the largest category representing 54% followed by medical then indirect costs. Informal care made the greatest contribution of nonmedical costs whereas physiotherapy was the largest medical subcategory. Nonmedical costs were highest in stage 3 and lowest at early stages whereas medical costs were almost steady among all stages with differences in individual subcategories. Of all medical costs, 95% were out of pocket. The mean utility score was 0.43 (± 0.31), which decreases with disease progression. Our study quantified the huge economic burden of DMD on the society and how it differs in different stages. Almost the whole burden is paid by households resulting in catastrophic expenditures, which leads to reduced compliance and quality of care. QOL is also severely compromised. Our findings can inform future healthcare policies and economic evaluation of new DMD therapies. • Previously published cost of illness studies estimated a huge economic impact of Duchenne muscular dystrophy on societies and found that it greatly affects the quality of life of patients. The economic impacts increase as patients grow older and as the disease progresses. All the studies were from high-income countries in Europe and the United States. • To the best of our knowledge, this is the first cost of illness study on Duchenne muscular dystrophy from Egypt and Africa and the first that represents a low- to middle-income country. Based on this study, the disease causes a large economic burden on society with more than 90% of this burden falling on families. These catastrophic household expenditures lead to reduced compliance and quality of care for the patients. • Total costs are not increasing with disease progression in late stages unlike in previous studies likely due to limited resources. Nevertheless, patients' quality of life declines abruptly with disease progression. These findings could be used to inform the economic evaluation of new therapies that delay disease progression and in revisiting health policies aiming at improving the quality of care for this patient population. [ABSTRACT FROM AUTHOR]