A new treatment is to be approved for use on the NHS for two blood disorders, currently treated with one of the world’s most expensive drugs.
Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) are currently treated with eculizumab, which costs hundreds of thousands of pounds per year according to the National Institute for Health and Care Excellence (NICE). Eculizumab is currently administered intravenously every two weeks.
However, ravulizumab has now been recommended for use in final guidance for treating PNH, and draft guidance for aHUS. Ravulizumab is less costly, and needs to be administered intravenously only every eight weeks. This increase in time between treatments may improve wellbeing and sense of independence for people living with the conditions, patient groups say.
NICE said clinical trials showed ravulizumab causes fewer episodes of breakthrough haemolysis in PNH patients than the existing drug and is at least as clinically effective. This in turn reduces hospital admissions and the need for blood transfusions.
The proposals will benefit about 430 people a year (270 with PNH, and 160 with aHUS), and will represent a cost-saving and better use of resources for the NHS, NICE say.
NHS England commercial medicines director Blake Dark said: “The NHS is here for everyone, whether it’s providing COVID-19 vaccines for millions or securing access to innovative new medicines, and adopting new treatments such as ravulizumab demonstrates how the NHS can improve the lives of people suffering with rare chronic conditions and also secure the best value for the taxpayer.”
NICE deputy chief executive Meindert Boysen said: “Living with a rare blood condition can be both physically and mentally challenging, especially when frequent treatment is required.
“We are pleased to be able to recommend ravulizumab for people who have paroxysmal nocturnal haemoglobinuria or atypical haemolytic uraemic syndrome. We are hopeful that the increased time between doses with this new treatment will lead to a better quality of life for these individuals and their loved ones.”
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