13 May 2024

The drug Voxelotor is to be available on the NHS, helping up to 4,000 patients in England with sickle cell disease, it has been announced.

The drug is administered once a day as a tablet and earlier in May gained approval from the National Institute for Health and Care Excellence (NICE). NHS England said it would fund the treatment through its Innovative Medicines Fund.

In clinical trials, 51% of patients receiving voxelotor experienced an increase in haemoglobin while 74% reported improvements in how they felt, according to NHS England. The drug is the first haemoglobin oxygen-affinity modulator.

NICE said the drug can be given with or without hydroxycarbamide. It approved the drug following a reduction in its price. The Sickle Cell Society had appealed against a previous decision to reject the drug.

Professor Bola Owolabi, director of the National Healthcare Inequalities Improvement Programme at NHS England, said: “We are proud to make this new treatment available on the NHS – it could help thousands of patients living with sickle cell disease across the country to have a higher quality of life and experience fewer side effects, and represents a significant step forward in addressing the healthcare inequalities experienced by some of our communities.”

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 sickle cell disease, as well as address NICE’s aim of reducing health inequalities.”

NHS chief executive Amanda Pritchard said: “The NHS has worked hard to make this life-changing treatment available at a fair price for the taxpayer, as part of our wider drive to improve the quality and experience of care for sickle cell patients, and tackle the stigma and inequalities they have told me they face.

“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 frontline immediately through our Innovative Medicines Fund.”

Source: NICE/NHS England

Link: https://www.england.nhs.uk/2024/05/nhs-rolls-out-life-changing-treatment-for-thousands-with-sickle-cell-disease/


Disclaimer: The news stories shared on this site are used as a way to inform our members and followers of updates and relevant information happening in Haematology. The BSH does not endorse the content of news items from external sources, and is not in a position to verify the findings, accuracy or the source of any studies mentioned. Any medical or drugs information is provided as an information resource only, and is not to be relied on for any diagnostic or treatment purposes.

News service provided by Englemed News.