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New HTA Decisions in England
December 2020
Drug name
CABLIVI® (caplacizumab)
Decision date
Therapeutic area
Blood and immune system conditions
Therapeutic sub area
Blood conditions
Caplacizumab with plasma exchange and immunosuppression is recommended, within its marketing authorisation, as an option for treating an acute episode of acquired thrombotic thrombocytopenic purpura (TTP) in adults, and in young people aged 12 years and over who weigh at least 40 kg. Treatment should be started and supervised by physicians experienced in managing thrombotic microangiopathies.
Decision Detail
It is recommended only if the company provides caplacizumab according to the commercial arrangement (a simple discount patient access scheme).
HERCULES and TITAN (double-blind placebo-controlled trials) provided the clinical evidence for caplacizumab. Trial results show that, compared with standard care alone, caplacizumab plus standard care reduces the time it takes to bring platelet levels back to normal, the number of plasma exchange treatments needed and time in hospital and intensive care. Adding caplacizumab likely reduces the long-term complications of acquired TTP and risk of death around the time of an acute episode, but it is unclear by how much. Using the committee's preferred modelling assumptions resulted in an ICER of £29,537 per QALY gained. Overall, the estimates are within the range normally considered a cost-effective use of NHS resources, so caplacizumab is recommended for treating acute acquired TTP.