UAT Around 4,000 people with sickle cell disease could benefit from a new treatment - regression
Improved deal means the treatment can now be recommended as a cost-effective use of NHS resources..
Voxelotor treats haemolytic anaemia caused by sickle cell disease (SCD) and is recommended for use on the NHS following a reduction in its price.
The price change has mitigated some of the uncertainties in the evidence identified by the independent appraisal committee in its previous draft guidance and means the treatment can now be recommended as a cost-effective use of NHS resources.
Our final draft guidance (published Friday 3 May 2024), recommends voxelotor (also called Oxbryta and made by Pfizer) with or without another treatment for SCD called hydroxycarbamide.
Voxelotor will be funded immediately via NHS England’s Innovative Medicines Fund and means around 4,000 people with SCD in England are set to benefit from the treatment straight away.
The committee agreed that, in this case, greater uncertainty in the estimates of clinical effectiveness, which supported the economic modeling for voxelotor, could be accepted. This was in light of the substantial disadvantages experienced by people with sickle cell disease. So, a higher cost-effectiveness estimate than usual could be considered acceptable.