Today, Rare Disease Day, NICE has
published final draft guidance recommending efanesoctocog alfa as
an option for treating and preventing bleeding episodes in people
aged 2 and over with severe
haemophilia A.
Efanesoctocog alfa (also called
Altuvoct and made by Swedish Orphan
Biovitrum) can be given as a
once-weekly injection instead of some current treatments that are
given several times a week.
Affecting mostly males, haemophilia A
is a rare, inherited and incurable condition that inhibits the
blood's ability to clot. People with haemophilia A do not have
enough of a clotting factor called factor VIII in their blood, or
it isn't working properly. This means they cannot form strong
clots and so they bleed for longer than usual. Bleeds can be the
result of an injury and, when the condition is severe, bleeding
into joints and muscles can happen without any
injury.
Bleeds can be prevented by injections
of factor VIII given every 2 to 3 days. A non-factor VIII
treatment, emicizumab, is also available for people of all
ages.
Efanesoctocog alfa's improved
convenience for some people, together with its effective bleeding
control, has the potential to have a positive impact on quality
of life.
Helen Knight, director of
medicines evaluation at NICE, said:
“Severe haemophilia A is a lifelong
inherited condition that can be life-threatening. It can impact a
person's ability to carry out normal daily activities because of the risk of suffering a
bleed. Current factor VIII
replacements can be difficult to manage due to the need for
frequent dosing to prevent potentially life-threatening and
debilitating bleeding episodes.
“Efanesoctocog alfa only has to be
taken once-a-week. Combined with its effective bleeding control,
it has the potential to have a significant positive impact for
some people with severe
haemophilia A.
“Today's decision, which comes just a
week after efanesoctocog alfa received its UK licence, shows how,
by collaborating with system partners NICE can quickly recommend
treatments that deliver tangible benefits for patients and the
NHS alongside value-for-money for
taxpayers.”
A Department of Health and
Social Care spokesperson said:
“We know that those living with rare
diseases and their families face immense everyday challenges, and
through our Rare Diseases Action Plan we aim to centre their
voices to address the difficulties they
face.
“We have committed to 3 new actions to
improve coordination of care, access to specialised treatments
and bolster research into innovative therapies for these rare
conditions.
“More widely, our Plan for Change will
transform the NHS by driving down waiting lists and investing in
quality facilities to ensure all patients – including those with
rare diseases - receive the care and treatment they
deserve.”
Professor , NHS England's Medical
Director for Specialised Services said:
"This is an important step forward in
treatment for patients with severe haemophilia A and ensures they
continue to have access to the latest advances in care on the NHS
to help prevent bleeding episodes.
“This condition can have really
debilitating and painful impacts and it's fantastic news that
this time-saving therapy will now be available to help improve
patients' lives, reinforcing the NHS' commitment to providing
innovative treatments at value for the
taxpayer.”
Efanesoctocog alfa is given as a
weekly injection. The dose is based on the person's weight and
for an adult weighing 81.8 kg the annual cost of the treatment
would be around £510,000 at its list price. The company has
agreed a confidential discount to the price of the
treatment.
Around 7,700 people in England have
severe haemophilia A with an estimated 1,900 who have severe
haemophilia A.
-
The final draft guidance on
efanesoctocog alfa for treating
severe haemophilia A is available on the NICE website
at: https://www.nice.org.uk/guidance/gid-ta11106/documents/html-content-7
