Health
NHS Takes Bold Step With £2.6 Million Drug For First Patient Amid Hopes And Concerns
After becoming the first NHS patient to get a dose of what has been branded the “world’s most expensive” medication, a man has been granted a new lease on life.
Your health cannot be valued, but the same cannot be true about medications, as some miracle drugs can cost millions of dollars.
Yesterday, June 18, the 44-year-old unnamed patient received the one-time procedure at Guy’s and St. Thomas’ NHS Foundation Trust in the UK capital.
He claims that after taking a dose of the medication Hemgenix, he will now be able to live the life he has “always dreamed of.”
It is not inexpensive, with an estimated cost of £2.6 million per patient, but it is expected to be a game-changer for people with haemophilia B.
It is administered as a one-time infusion that takes one to two hours, and it is the only treatment of its kind for the clotting condition.
Previously, individuals with haemophilia B required several weekly infusions to manage the potentially fatal symptoms of the illness.
Hemgenix has the potential to significantly alter the game, it goes without saying.
What is haemophilia B?
A certain protein called Factor IX is either absent or insufficient in a person’s blood, resulting in haemophilia B, a form of clotting condition.
Given that clotting is necessary to prevent cuts, wounds, and traumas from bleeding, and that people with this condition might lose a potentially fatal amount of blood from even a small wound, this is rather regrettable.
According to the Great Ormond Street Hospital, people with haemophilia B are unable to mend their damaged blood vessels properly.
It explains, “Factor IX in the blood is too low to complete the clotting process. Bleeding may occur inside the body as well as from the skin – this can include bleeds inside the joints.”
“It can follow an injury or sometimes no trigger occurs – this is called a ‘spontaneous bleed’. Over time, each bleed can damage the joint making it swollen and harder to bend.”
“Bruising is also common in people with Haemophilia B. Other areas of the body may develop bleeds, such as the digestive or urinary systems, where blood may be visible in the faeces or urine.”
“Nose bleeds can also happen spontaneously.”
How does Hemgenix work?
Although Hemgenix was authorised for use on the NHS in 2024, the NHS gave the medication for the first time on Wednesday.
The medication, sometimes referred to as etranacogene dezaparvovec, helps people with haemophilia B clot by substituting the faulty gene they lack.
Patients no longer need to have frequent injections thanks to the one-time Hemgenix infusion.
Experts are optimistic that the effect may continue longer than the three years that studies have indicated it can.
Only 260 of the approximately 2,000 individuals with “moderately severe or severe” variants of haemophilia B in the UK are now eligible to get Hemgenix through the NHS.
According to those calculations, giving the medication to all of those individuals would cost £676 million; but, since monthly clotting injections cost about £8 million per patient over their lifetime, the health care will ultimately save money.
Why is Hemgenix so expensive?
In an explainer medically reviewed by chemist Leigh Ann Anderson, Drugs.com claims that gene therapies, such as Hemgenix, are more difficult to design than conventional pills, requiring years of expensive research and development expenses to be recovered.
The one-time expenses will need to be greater since fewer people will take the treatment because severe haemophilia B is quite uncommon.
Furthermore, maker CSL Behring contends that the expense is justified because a one-time therapy is effective for at least three years, saving patients from having to pay for continuous, long-term care.
CSL Behring claimed that Hemgenix will save the US healthcare system up to $5.8 million per patient when the FDA approved its use in the US.
What does Hemgenix mean for the patient?
Professor Sir Stephen Powis, the National Medical Director of the NHS, has called the gene therapy Hemgenix a “transformative” treatment for individuals with haemophilia B.
He stated the infusion “has the potential to significantly improve the lives of hundreds of people by helping to reduce symptoms such as painful bleeds” when he described how it works in June of last year.
Professor Powis continued, “It is a one-time therapy that could be truly life-changing for some, as it could help people avoid the need for regular hospital visits.”
The 44-year-old man who was given the first dose of Hemgenix by the NHS earlier this week said that he was diagnosed with haemophilia B at the age of 18 months.
“I’ve always had to be more cautious and to plan ahead,” he told the Daily Mail. “There is a level of anxiety in that and being overly cautious has often led to missed opportunities and things I can’t do, like contact sports.”
“To experience a life free of the worry and to do things that I wouldn’t normally do will also be amazing.”
“Not needing to plan ahead for treatment deliveries or looking up hospitals in foreign destinations when going on holiday, or having to tell people, ‘Sorry, I can’t do that I’ve got haemophilia’, will be something I’ve always dreamed of.”
The London hospital’s consultant haematologist, Dr. Pu-Lin Luo, who administered the man’s dose of Hemgenix, described the action as a “exciting” step in improving the lives of those who have haemophilia B.
“This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients,” she stated. “It is also a testament to the advancement of cell and gene therapies in the UK.”
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