NHS agrees to pay for a ‘trailblazing’ drug that prevents deadly bleeds in patients with severe haemophilia
- Severe haemophilia A is thought to affect around 1,800 people in the UK
- Patients were forced to have clotting drugs administered into vein in chest
- Drug emicizumab can be injected under the skin as little as every two weeks
The newly-available drug emicizumab, brand name Hemlibra, mimics a clotting protein that is missing in haemophilia A sufferers
A ‘trailblazing’ new treatment that prevents life-threatening bleeds in severe haemophiliacs will be available on NHS England.
Haemophilia A is one of the most well-known clotting disorders, with around 1,800 people in the UK having a severe form of the disease.
It occurs when a genetic mutation ‘deletes’ a specific protein that causes blood to clot. This can leave sufferers at risk of bleeding to death every time they have a small cut or graze.
Up until now, many patients had to have clotting drugs administered directly into a vein in their chest via a central venous access device every few days.
But the newly-available drug emicizumab, which mimics the missing protein, can be injected under the skin as little as every two weeks.
Hameophilia A occurs when the blood-clotting protein factor VIII is present at insufficient amounts in the blood.
It is defined as mild, moderate or severe depending on the amount of factor VIII.
Emicizumab, brand name Hemlibra, will be available to those with the severe form of the condition.
NHS England claims the drug will particularly benefit young children whose parents struggle to administer the existing infusion regimen.
For some, ‘man-made’ factor VIII can be given into a central venous access device at home. This is often surgically placed under the skin.
However, others have to administer the drug directly into a vein.
In contrast, Hemlibra can be given weekly or once a fortnight.
People with less extreme hameophilia A who do not respond to other treatments have been able to access the drug in England, Scotland and Wales since last year.
NHS chief executive Simon Stevens said: ‘Giving patients access to world-class, trailblazing drugs and therapies is a key part of the NHS Long Term Plan, which aims to save thousands more lives.
‘As a parent, I know cuts and scrapes happen to kids all the time but for many families these routine accidents can be distressing and life-threatening.
‘So this new treatment will change lives and lift a weight from thousands of parents.’
The Haemophilia Society chief executive Liz Carroll added: ‘This decision is fantastic news for our community.
‘Current treatments can require intravenous infusions multiple times a week which can place a significant burden on people with haemophilia and their carers.
‘This decision will mean people will have the opportunity to have treatment less frequently without intravenous access, which will enable many to live their lives more freely.’