In draft guidance published 15 April 2021, NICE has recommended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The new draft guidance means that patients will now be eligible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.”
Read the draft guidance here.
