OBJECTIVES: This study aims to evaluate the benefit of givinostat in addition to stable corticosteroids, on the disease progression of DMD patients estimated from the cost-effectiveness model. This study estimates the impact of extending patients’ lives in the earlier phases of the disease on both patient and caregivers.

METHODS: The EPIDYS trial of ambulant DMD patients aged 6 and above on stable corticosteroid treatment and receiving givinostat in addition to stable corticosteroids, and the open label extension study of givinostat, were integrated into Project Hercules’ Markov model. The relative clinical effectiveness was obtained from a published analysis of givinostat versus CNRG-DNHS (Cooperative International Neuromuscular Research Group Duchene Natural History Study) and imaging DMD, and compared versus a base-case comparator from the University of Leicester’s natural history model. The model simulated the long-term impact of givinostat over 50 years. Health benefit was discounted at 3.5% annually. The main outcome was quality-adjusted life years (QALYs) gained for patients. Additional outcomes included life years (LYs) gained and health state utility values for patients and caregivers.

RESULTS: Givinostat, in addition to stable corticosteroids, extended the median age that patients remained in the ambulatory health states. Median utility values were over double for early and late ambulatory states (0.79 and 0.64) compared to non-ambulatory states (0.314 to 0.211). Along with the additional 2.77 LYs and patients remaining longer in the early stages of the disease with higher utility values, givinostat treatment resulted in an additional 4.55 QALYs compared to natural history. The quality of life of caregiver was also higher for givinostat treated group when dependents were in early and late ambulatory states (0.86 and 0.84, respectively) compared to non-ambulatory disease states (from 0.78 to 0.81).

CONCLUSIONS: Model results showed that givinostat treatment slows disease progression, improving both patient and caregiver quality of life and resulting in QALY gains.