OBJECTIVES

: Duchenne muscular dystrophy (DMD) is a rare, fatal genetic neuromuscular disease. To date there is a paucity of published information regarding the cost-effectiveness of deflazacort versus prednisone for these patients. This study provides an evidence-based cost-effectiveness assessment of deflazacort relative to prednisone.

METHODS

: A Partitioned Survival Model (PartSA) was developed based on a previous research effort that measured time to loss of ambulation and death from DMD patients on corticosteroids using Kaplan-Meier curves, and on previous survey-based evidence regarding supportive care costs and health utility for ambulatory and non-ambulatory DMD patients. As a conservative assumption, a highly favorable but still evidence-based treatment effect that shifted both the ambulation survival curve and mortality curve by two years was used to estimate changes in quality-adjusted life years (QALYs) and direct medical costs for deflazacort. The analysis took a United States health sector perspective and a modified societal perspective over a lifetime time horizon and used a 3% discount rate. Deterministic sensitivity analysis (DSA) and probabilistic sensitivity analyses (PSA) were also performed.

RESULTS

: Even with very favorable assumptions regarding treatment effects for deflazacort, the incremental cost effectiveness ratios (ICERs) were $790,000/QALY gained and $829,000/QALY gained for the health sector perspective and modified societal perspective, respectively. Treatment effect, deflazacort drug cost, and ambulatory health utility were the most sensitive in the DSA. In the PSA, deflazacort had a 0% probability of being cost-effective at a willingness to pay threshold of $150,000/QALY gained and a 2.79% probability at $500,000/QALY gained.

CONCLUSIONS

: The evidence for evaluating the cost-effectiveness of treatments for DMD is sparse. However, available evidence suggests deflazacort at current prices is highly likely to have ICERs exceeding standard thresholds when compared to prednisone.