Price cut allows Pfizer’s sickle cell therapy Oxbryta to rollout on NHS
03 May 2024
Drug ApprovalCell TherapyAccelerated Approval
A price cut has allowed Pfizer’s Oxbryta (voxelotor) to be recommended for use on the NHS to treat people with haemolytic anaemia caused by sickle cell disease (SCD), following an initial rejection last year. The once-daily oral drug – which the company gained through its purchase of Global Blood Therapeutics - was approved in the UK in 2022 for people aged 12 and older.
However, previous draft guidance from the National Institute for Health and Care Excellence rebuffed the treatment due to uncertainties over supporting evidence. NICE said Friday in final guidance that a cut to Oxbryta’s cost allows some of these reservations to be “mitigated,” with the drug to be funded immediately via NHS England’s Innovative Medicines Fund.
Helen Knight, director of medicines evaluation at NICE, remarked “throughout our appraisal…the independent committee has always considered that it has the potential to address the need for effective treatments for SCD.” Knight added “we are therefore delighted that the company has reduced the price of [Onbryxta] to a level that properly reflects the uncertainty in the evidence.”
Specifically, NICE said that its appraisal committee agreed that greater uncertainty in the estimates of clinical effectiveness that supported economic modelling for Onbryxta could be accepted in light of “substantial disadvantages” experienced by people with SCD, and a higher cost-effectiveness estimate than usual could be considered acceptable.
“By improving the quality of life and reducing the need for hospital care, this new treatment option also has the potential to free up doctors, nurses and other clinicians to better support other patients, so we are acting fast to get it to the front-line immediately through our Innovative Medicines Fund,” commented Amanda Pritchard, NHS chief executive.
NICE recommended Oxbryta with or without another SCD treatment hydroxycarbamide, noting that around 4000 people in England are set to benefit from Pfizer’s drug.
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