Cost/effectiveness of elivaldogene autotemcel vs. hematopoietic stem cells transplantation in patients with cerebral adrenoleukodystrophy (CALD)

Cerebral adrenoleukodystrophy (CALD) is a severe, rare neurological disease with a progressive course and poor prognosis. Allogeneic hematopoietic stem cell transplantation (HSCT) is still considered the standard of care for CALD. Elivaldogene autotemcel is a novel gene therapy for the treatment of childhood-onset CALD. Since many novel gene therapies are associated with high costs, our aim was to compare costs and effectiveness of elivaldogene autotemcel with the costs and effectiveness of allogeneic HSCT treatment of childhood-onset CALD. The analysis used an 80-year horizon and the perspective of the Serbian Republic Health Insurance Fund. Quality-adjusted life years (QALYs) gained with elivaldogene autotemcel and HSCT, as well as direct treatment costs, were our major outcomes. For the purposes of the economic analysis, we constructed and simulated a discrete-event simulation model to calculate the outcomes. We obtained the results of the model using a Monte Carlo simulation, which we conducted on 1000 virtual CALD patients. The analysis indicated that elivaldogene autotemcel was not cost-effective when compared to HSCT, and the net monetary benefit was negative. The base case incremental cost/effectiveness ratio was 259,848,512 RSD/QALY. Elivaldogene autotemcel is not more cost-effective than HSCT for the treatment of CALD from the perspective of the Serbian Republic Health Insurance Fund, according to classic pharmacoeconomic analysis and criteria. To ensure that the treatment is affordable for all patients for whom it is prescribed, a differentiated pricing strategy with lower drug prices in developing nations seems to be required.