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New HTA Decisions in England
May 2021
Drug name
ULTOMIRIS® (ravulizumab)
Alexion Pharmaceuticals
Decision date
Therapeutic area
Blood and immune system conditions
Therapeutic sub area
Blood conditions
Recommended with restrictions
Ravulizumab is recommended, within its marketing authorisation, as an option for treating paroxysmal nocturnal haemoglobinuria in adults.
Decision Detail
It is recommended for adults: • with haemolysis with clinical symptoms suggesting high disease activity, or; • whose disease is clinically stable after having eculizumab for at least 6 months, and; • the company provides it according to the commercial arrangement (there is a simple discount patient access scheme for ravulizumab).
The company presented data from ALXN1210-PNH-301 and ALXN1210-PNH-302, two non-inferiority phase 3 trials comparing the safety and effectiveness of ravulizumab with eculizumab. The committee concluded that ravulizumab and eculizumab are similarly effective and that adverse events with ravulizumab are likely to be similar to eculizumab. Ravulizumab is given less often than eculizumab so there is some benefit on quality of life and it may save costs because people need to have it less often. In the committee's preferred analysis and all the other cost effectiveness analyses, ravulizumab was more effective and less costly than eculizumab. Therefore, ravulizumab is recommended as a treatment option for paroxysmal nocturnal haemoglobinuria.