Cost-effectiveness analysis of caplacizumab in the new standard of care for immune Thrombotic Thrombocytopenic Purpura in Italy

Abstract


 Objectives: To evaluate the cost-effectiveness analysis (CEA) of caplacizumab in combination with plasmapheresis (PEX) and immunosuppression compared to PEX and immunosuppression in the treatment of acute episodes of iTTP.
 Methods: A Markov model was used to conduct the CEA from the perspective of the hospital, over a lifetime horizon. Clinical data derived from HERCULES trial and a systematic literature review. Economic input included direct costs only. Utility and disutility values were obtained from literature. Data on healthcare resources and costs were retrieved from HERCULES trial, literature, TTP guidelines and Italian tariffs. A sensitivity analysis was conducted. The cost-effectiveness probability was tested for several options of discount levels considering a suggested willingness to pay (WTP) threshold of € 60,000 in Italy.
 Results: The use of caplacizumab in combination with PEX and immunosuppression is associated with a positive difference in survival of 3.27 life years (24.53 vs 21.26) and in quality of life of 3.06 QALY (22.01 vs 18.96) when compared to PEX and immunosuppression. Caplacizumab leads to an ICER per life years of € 41,653 and an ICER per QALY of € 44,572. For the suggested WTP threshold, the probability of caplacizumab being cost-effective is 82.4% (no discount), 92.8% (15% discount), 95.3% (20% discount), 96.9% (25% discount) and 98.2% (30% discount).
 Conclusions: Caplacizumab in addition to PEX and immunosuppression is cost-effective, allowing the hospital to achieve greater efficiency in managing the burden of a life-threatening disease such as iTTP.

The standard of care for immune thrombotic thrombocytopenic purpura today

“…Most recent studies have demonstrated that adding caplacizumab to TPE and immunosuppressives is cost-effective. 69,80 It allows the hospital to achieve a greater efficiency in managing a life-threatening disease like iTTP.…”

“…Thus, it is important to stop microvascular thrombosis with caplacizumab as early as possible, while trying to address the underlying autoantibody‐mediated inhibition of ADAMTS‐13 activity with immunosuppressives, which may take weeks to months. Most recent studies have demonstrated that adding caplacizumab to TPE and immunosuppressives is cost‐effective 69,80 . It allows the hospital to achieve a greater efficiency in managing a life‐threatening disease like iTTP.…”

Global Health Resource Utilization and Cost-Effectiveness of Therapeutics and Diagnostics in Immune Thrombotic Thrombocytopenic Purpura (TTP)

“…Cost-effectiveness: Cost is one concern with the use of caplacizumab, which has been examined in several cost-effectiveness analyses [58][59][60]. In December 2020, after initial rejection, a series of meetings and re-appraisal of Sanofi's modeling at their fourth meeting, the National Institute for Health and Care Excellence (NICE) officially recommended caplacizumab use with TPE and immunosuppression for patients with immune TTP [58].…”

“…A 2021 Italian health technology assessment conducted a cost-effectiveness analysis of capalcizumab added to the standard of care versus the standard of care at a lifetime time-horizon, an Italian hospital perspective and at a willingness-to-pay threshold of 60,000 EUR/QALY previously suggested in Italy [59]. The authors reported an expected increase in 3.27 life years and 3.06 quality-adjusted life years with caplacizumab use at an incremental cost-effectiveness ratio of 44,600 EUR/QALY.…”