Price cut unlocks NHS use of sickle cell drug Oxbryta


It has been nearly two years since Pfizer’s sickle cell disease (SCD) therapy Oxbryta was approved in the UK, but patients will now be able to access the treatment after an agreement was reached on a price cut.

Cost-effectiveness agency NICE has issued final draft guidance on Oxbryta (voxelotor) that clears the way for the use of the drug – with or without hydroxycarbamide – as an option for treating anaemia caused by SCD in patients aged 12 and over.

The once-daily oral therapy can only be used if hydroxycarbamide on its own is ineffective or cannot be taken by a patient, according to the guidance.

Oxbryta will be funded immediately via the Innovative Medicines Fund (IMF), set up to cover the cost of new therapies on an interim basis while additional evidence of their benefits is generated, but will switch to routine NHS funding once the NICE guidance is finalised, likely later this month.

That means that around 4,000 people with SCD in England could receive treatment with the new drug straight away, according to NICE. The health technology assessment (HTA) agency had initially rejected the drug in January 2023, saying it was too expensive. 

The agreement has been hailed as a “significant milestone “ in SCD care by John James OBE, chief executive of the Sickle Cell Society, who said it comes after decades of limited access to disease-modifying treatments for the disease.

Oxbryta was the first medicine approved in Europe that directly prevents sickle haemoglobin (HbS) polymerisation, the molecular basis of the sickling and destruction of red blood cells in SCD that leads to anaemia. It was shown in clinical trials to improve anaemia compared to standard care and was also likely to reduce the need for repeated blood transfusions.

“We applaud this decision and the work of everyone involved in making this day happen," added James. “We look forward to this being the first in a series of new treatment options for people living with sickle cell disorder."

In England, there are around 17,000 people living with SCD, an inherited blood disorder, with 250 new cases a year. It is generally more common in people of Black African, Caribbean, Middle Eastern and South Asian heritage. A decision on the drug by the Scottish Medicines Consortium (SMC) is due in June.

NICE’s director of medicines evaluation, Helen Knight, said the agency was able to reach an agreement with Pfizer after the company reduced the price of Oxbryta – which at list is just over £5,900 for a pack of 90 tablets – “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. “

Pfizer acquired Oxbryta as part of its $5.4 billion takeover of Global Blood Therapeutics (GBT), which closed in October 2022.

Worldwide sales of the drug were predicted to reach around $2 billion a year at its launch, and while it still has a long way to go, Pfizer reported that they rose 73% to reach $328 million last year – with all but $5 million of that total coming from the US market.