Improved deal signals NICE recommendation of sickle cell treatment voxelotor

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mitigated some of the uncertainties in the evidence identified by the independent appraisal committee in its previous draft guidance and means the treatment can now be recommended as a cost-effective use of NHS resources.

NICE’s final draft guidance (published Friday 3 May 2024), recommends voxelotor (also called Oxbryta and made by Pfizer) with or without another treatment for SCD called hydroxycarbamide.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.

Helen Knight, director of medicines evaluation at NICE, said: “This is great news for people with sickle cell disease, particularly given the health inequalities experienced by people with the condition.

“Throughout our appraisal of voxelotor the independent committee has always considered that it has the potential to address the need for effective treatments for SCD, as well as address NICE’s aim of reducing health inequalities.

“We are therefore delighted that the company has reduced the price of voxelotor to a level that properly reflects the uncertainty in the evidence and enables NICE to recommend it as a good use of taxpayer’s money.”

Health Minister Andrew Stephenson said: “Sickle cell disease can be debilitating and have a severely negative impact on people’s lives.

“This is hugely welcome news that will help tackle longstanding health inequalities experienced by this community.

“Access to voxelotor will be accelerated for eligible patients through funding from the Innovative Medicines Fund, which enables treatment to be offered without delay and at a fair price to the taxpayer.”

The committee agreed that greater uncertainty in the estimates of clinical effectiveness that supported the economic modelling for voxelotor could be accepted in light of the substantial disadvantages experienced by people with sickle cell disease, and a higher cost-effectiveness estimate than usual could be considered acceptable.