OBJECTIVES: DMD is a rare, fatal, X-linked recessive genetic disorder. Corticosteroids (CS) are the standard of care for treatment, with Emflaza (deflazacort) being the first and only FDA-approved CS treatment for DMD in the US. Over half of the patients with DMD are not currently treated with CS. An economic model was built to estimate the budget impact of deflazacort for the treatment of DMD.

METHODS: The model estimates the costs that may be associated with deflazacort for the treatment of DMD from managed care decision maker’s perspective. Costs include pharmacy and medical costs, and offsets for delays in scoliosis, loss of ambulation, nocturnal ventilation, and cardiomyopathy. Deflazacort is compared with no treatment. The budget impact is based on the total costs of therapy for a 1,000,000 member health plan. The budget impact results are calculated based on a 10% uptake of deflazcort in year 1, a 15% uptake in year 2, and a 20% uptake in year 3.

RESULTS: For a commercial plan with 1,000,000 members, 16 patients aged 5-24 years were estimated to have DMD. The estimated incremental PMPM pharmacy cost due to deflazacort was $0.008/$0.012/$0.0116 for years 1/2/3. Due to lower costs associated with delays in scoliosis surgery, loss of ambulation and onset of cardiomyopathy, and less need for nocturnal ventilation, the total annual cost per patient treated with deflazacort was ~$17,000 less than patients who were not treated. The deflazacort budget impact was a savings of $28,299/$42,262/$56,226 in years 1/2/3, with 3-yr cumulative total budget savings of $126,786; corresponding total PMPM budget impact was -$0.002, -$0.004, -$0.005 for years 1, 2, and 3.

CONCLUSIONS: Deflazacort is a less costly alternative to no treatment. The introduction of deflazacort for the treatment of DMD results in modest budget impact savings.