A British man has just become the first-ever patient to receive a therapy dubbed the ‘world’s most expensive drug’ outside of a clinical trial, and it was on the NHS.
The anonymous 44-year-old from the North East of England was given the life-changing drug at Guy’s and St Thomas’ hospital to treat a rare blood clotting disorder he has called haemophilia B.
People with haemophilia B experience random painful bleeding because their blood doesn’t clot properly, and they usually have to have blood infusions once or twice every week.
The new therapy called Hemgenix is given via a one to two-hour infusion and helps the liver make the gene that is needed for blood to clot in the body. However, there’s a catch. It costs a staggering £2.6 million per dose!
The 44-year-old, who was diagnosed with the disorder as a child, told the NHS: “I’ve always had to be more cautious and to plan ahead. 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. Being free from the burden of knowing I have the condition will be fantastic.”
Credit: NHS
“To experience 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 doctor who gave him the therapy at Guy’s and St Thomas’, Dr Pu-Lin Luo, said: “This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients. It is also a testament to the advancement of cell and gene therapies in the UK and these are exciting times.”
“Administering the first dose of Hemgenix outside of a clinical study was made possible by exceptional cross-centre collaboration between Guy’s and St Thomas’, where the patient was treated, and Newcastle Hospitals, their local haemophilia centre.”
Around 2,500 people are currently living with the condition in the UK, and 250 of them are expected to be eligible for the new treatment in England on the NHS.
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