OBJECTIVES: Letermovir has demonstrated efficacy as a prophylactic treatment of cytomegalovirus (CMV) reactivation and disease in adult CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT) in a recent Phase III clinical trial. The objective of this analysis was to assess the cost-effectiveness of letermovir compared with pre-emptive therapy in Portugal.

METHODS: A decision analytic model estimated the total cost and lifetime quality adjusted life years (QALY’s) by incorporating data from the phase III clinical trial at 24 weeks post-transplant. This study was conducted according to a Portuguese societal perspective, with no productivity costs included. All data related to demographics and costs were derived from Portuguese sources. The model used an annual discount rate of 5% for both costs and outcomes.

RESULTS: In the base case analysis, letermovir reduced the number of clinically significant CMV infections compared to the standard of care (SoC), at 24 weeks post-transplant. This treatment partially offset costs through a reduction in utilization of pre-emptive therapy and fewer CMV-related hospitalizations when compared to SoC. Prophylaxis with letermovir resulted in an incremental 0.43 life years gained and 0.41 QALYs gained per patient treated while increasing total cost. The incremental cost-effectiveness ratio (ICER) was €35,604€ per QALY gained.

CONCLUSIONS: The results of this model are robust and would suggest that letermovir prophylaxis is a cost-effective strategy at commonly accepted ICERs for orphan drugs in Portugal.The clinical and economic outcomes of letermovir could result in benefit to the Portuguese healthcare system by addressing a major unmet need and shifting CMV prevention strategies from preemptive therapy to prophylaxis in adult CMV-seropositive patients in the post-HSCT context.