National Institute for Health and Clinical Excellence (NICE)
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NICE recommends new treatment for rare blood disorder

Hundreds of people with a rare blood disorder have access to a new treatment regimen following draft guidance from NICE.

In draft guidance published 15 April 2021, NICE has recommended ravulizumab (Ultomiris, Alexion Pharmaceuticals) as a treatment for paroxysmal nocturnal haemoglobinuria (PHN).

Currently, people with symptomatic PHN only have one treatment option available on the NHS, eculizumab, which is delivered intravenously every 2 weeks.

The new draft guidance means that patients will now be eligible for treatment with ravulizumab, which is delivered every 8 weeks. Patient experts stated that this increase in time between treatments may improve the quality of life for people with PHN, increasing their independence and psychological wellbeing. 

PHN is a rare blood disorder characterised by an excessive breakdown in red blood cells, which can lead to anaemia, kidney problems, and blood clots in the blood vessels. It is a chronic condition which can be life-threatening when left untreated.

Clinical trial evidence showed that treatment with ravulizumab is at least as clinically effective as eculizumab, and was found to cause fewer episodes of breakthrough haemolysis, a symptom of PHN, reducing the number of hospital admissions and the need for blood transfusions. Ravulizumab is also cost saving for the NHS.

Therefore, ravulizumab is considered an effective use of NHS resources and is recommended for routine use on the NHS.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said:

“Living with a chronic 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 with paroxysmal nocturnal haemoglobinuria.

“Not only is this a cost saving treatment for the NHS, but 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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