The scope for this technology appraisal includes avatrombopag. NICE cannot release any recommendations on avatrombopag until it has an agreed list price in the UK.
1.1 Lusutrombopag is recommended, within its marketing authorisation, as an option for treating severe thrombocytopenia (that is, a platelet count of below 50,000 platelets per microlitre of blood) in adults with chronic liver disease having planned invasive procedures.
Why the committee made these recommendations
People with chronic liver disease often have low blood platelet levels. This means that they are more likely to bleed during invasive medical procedures, including surgery. Currently, they have a platelet transfusion before invasive procedures to help reduce their chances of bleeding.
Avatrombopag and lusutrombopag are oral therapies that raise platelet levels, the aim being to reduce (but not eliminate) the chances of a patient needing a platelet transfusion. Platelet transfusions rely on donors and are given intravenously, so the possibility of replacing them with an oral treatment is an improvement. The drugs have several other benefits, including:
the convenience of fewer transfusions
fewer hospital stays
a decreased chance of having transfusion-related complications.
In addition, platelets are a limited resource and can only be stored for a short time. This means that there can be problems getting them to people in time for their procedure, which can delay surgery. On the other hand, avatrombopag and lusutrombopag need to be taken more than a week before a procedure, so can be used only for planned procedures.
Clinical trial evidence shows that fewer people need a platelet transfusion if they have avatrombopag or lusutrombopag rather than a placebo treatment. But whether the drugs improve survival compared with platelet transfusions has not been measured. There is also no clinical evidence that either drug is better than the other.
The economic modelling does not fully account for the benefits for patients and service delivery when using avatrombopag and lusutrombopag. If these are considered, using lusutrombopag would likely save the NHS money. So, lusutrombopag can be recommended for treating thrombocytopenia in people with chronic liver disease who need planned invasive procedures. It is not possible for NICE to make a recommendation for avatrombopag because the drug does not have a UK price.