OBJECTIVES: Multiple myeloma (MM) is a disease of the bone marrow characterized by the uncontrolled proliferation of immunoglobulin-producing plasma cells. It accounts for 1,2% of all cancers diagnosed in Italy and is associated with a significant burden of illness. Daratumumab monotherapy was recently approved for the treatment of adults with relapsed and refractory MM, whose prior therapy included a proteasome inhibitor and an immunomodulatory agent, and who have demonstrated progression on the last therapy. The aim of the study is to assess the cost-effectiveness of daratumumab in this setting compared to pomalidomide and low-dosed dexamethasone (LDD) in Italy.

METHODS: The study is based on a survival partition model comparing treatment costs and outcomes from the Italian NHS perspective. The model was adapted using trial data, literature and local sources. Drug list prices and national tariffs were used to estimate treatment costs over a 10 year-timeframe with a 3,5% discount rate. Treatments were compared by incremental cost-effectiveness ratio (ICER) per life year (LY) gained. As Italy does not have a formalized cost-effectiveness threshold, a literature value of 60€k/LY was adopted (Messori, 2004).

RESULTS: The results demonstrate the cost-effectiveness of daratumumab’s new immunotherapeutic approach compared to pomalidomide and LDD in the target population, providing an additional 1.1 LY with incremental direct costs of 36,681€. The analysis established an ICER of 34,151€ per LY gained versus pomalidomide and LDD, falling within the acceptability threshold of 60€k/LY. The results were confirmed through deterministic and probability sensitivity analyses, as in 94% (DSA) and 95% (PSA) of cases, the ICER lies within the above threshold.

CONCLUSIONS: This study proves that daratumumab is a cost-effective therapy for the treatment of MM in the Italian NHS, compared to SOC for patients who have received ≥2 prior lines of therapy, including a PI or an IMiD, or who were double refractory to both.