Price reduction enables NHS rollout of Pfizer's sickle cell therapy Oxbryta

A significant price reduction has enabled Pfizer's Oxbryta (voxelotor) to be endorsed for use by the National Health Service (NHS) to treat individuals with haemolytic anaemia resulting from sickle cell disease (SCD). This approval follows an initial rejection last year. Oxbryta, an oral medication taken once daily, was approved in the United Kingdom in 2022 for persons aged 12 and above. Originally, the National Institute for Health and Care Excellence (NICE) had expressed reservations about the drug due to uncertainties regarding the supporting evidence.

In its final guidance released on Friday, NICE acknowledged that the reduced price of Oxbryta addresses some of these concerns, making it possible for the drug to be funded immediately through NHS England's Innovative Medicines Fund. Helen Knight, the director of medicines evaluation at NICE, stated that the independent committee has consistently recognized the potential of Oxbryta to meet the need for effective SCD treatments. Knight expressed satisfaction that Pfizer has lowered the price to a level that appropriately reflects the evidence-related uncertainties.

The NICE appraisal committee specifically noted that while there are uncertainties in the clinical effectiveness estimates used for economic modelling of Oxbryta, these uncertainties could be tolerated due to the significant disadvantages faced by SCD patients. As a result, they found a higher cost-effectiveness estimate acceptable.

Amanda Pritchard, NHS chief executive, commented that the new treatment promises to enhance patients' quality of life and reduce the necessity for hospital care. This, in turn, would allow healthcare professionals to better support other patients, thereby facilitating quicker deployment through the Innovative Medicines Fund.

NICE has recommended the use of Oxbryta, either alone or in combination with another SCD treatment, hydroxycarbamide. Approximately 4,000 individuals in England are expected to benefit from Pfizer's drug.