NHS England is to fund a life changing treatment for thousands of people with severe Haemophilia, which will dramatically cut their risk of life-threatening bleeds and reduce treatment time.
The new treatment is part of a package of measures set out in the NHS Long Term Plan which will save lives through access to the most advanced medical interventions.
People with Haemophilia A are at risk of spontaneous or uncontrolled bleeding because they do not have enough of a blood clotting protein or it does not work properly.
While a cut or graze is easily dealt with for most people, anyone with this condition does not have enough or effective parts of the blood which allow this to happen, leaving them at risk of uncontrolled bleeding that can cause serious harm and even death.
A new drug – emicizumab (also referred to as Hemlibra®) – will bolster the blood by mimicking the action of the blood protein factor VIII to avoid uncontrolled bleeding, while cutting treatment times from multiple time-consuming infusions every week to a single injection given once-a-week or fortnight.
The NHS in England has agreed to fund the drug for around 2,000 people in the country who live with the condition.
They include many young children whose parents sometimes struggle to administer the current infusion several times a month.
Simon Stevens, NHS chief executive, 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 that 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.
“This treatment has the potential to significantly improve the lives of people with haemophilia, especially children – reducing treatment time and even ending the dangerous bleeds which can lead to life-threatening cuts and life-changing damage.”
Liz Carroll, chief executive of The Haemophilia Society, said: “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 that people will have the opportunity to have treatment less frequently without intravenous access which will enable many to live their lives more freely.’’