NHS regulator approves ‘world’s most expensive drug’ costing £2.6m per dose… and it will SAVE the health service money, experts say

A gene therapy once billed as ‘the world’s most expensive drug’ is now being offered free on the NHS, but experts are confident it will save the health service money.

The drug, called Hemgenix, costs £2.6 million per patient, even though only one dose is needed.

It is the only treatment of its kind for hemophilia B, a bleeding disorder in which the body does not make enough or any of the protein crucial for blood clotting.

Clotting is an important biological mechanism that ensures that wounds stop bleeding. People with this condition can suffer serious and even life-threatening blood loss if they are injured.

In addition, they are also at risk of so-called ‘spontaneous bleeding’, which occurs without immediate injury and can be fatal if it occurs in a vital organ.

The defective gene in Hemgenix, which is unable to cause clotting, is replaced with a gene that can, eliminating the need for frequent injections

Hemophilia B patients must take regular injections of an artificial clotting agent called Factor IX for life to minimize the risk of catastrophic injury.

But with Hemgenix, the patient’s defective gene, which is unable to cause the clot, is replaced with a gene that can, eliminating the need for these injections.

Studies of the gene therapy, administered through an IV, have shown that the effects last for at least three years, but the hope is that it can work even longer.

Although there are around 2,000 people with haemophilia B in the UK, it is estimated that only 260 of them are eligible for Hemgenix through the NHS.

This brings the potential total bill to the taxpayer to around £676 million.

However, MailOnline understands that the NHS is purchasing the drug at an unknown discount, meaning the actual cost is likely to be lower.

The British National Institute for Health and Care Excellence (NICE), which decides which medicines should be available on the NHS and to whom, recommended that the medicine be made available to the healthcare system on a ‘managed access’ basis.

This means it is only offered to patients with ‘moderate or severe haemophilia B’ where the doctor thinks it could help them.

It will also be subject to ongoing evaluation for cost-effectiveness, which could mean it is rolled out to more patients one day.

One patient who has already benefited is Elliott Collins from Essex.

The 34-year-old man has severe hemophilia B and participated in gene therapy trials five years ago.

On the Today programme, Mr Collins said that thanks to the treatment he was effectively no longer suffering from haemophilia.

“Initially my clotting levels were below zero and now they’re hovering around 60, and anything over 50 technically doesn’t make you a hemophiliac,” he said.

‘Not long after I had the treatment, I remember walking by and hitting my knee on a cupboard… and initially thinking, ‘Ooh, I hit that so hard that it’s swollen up quite a bit, and I’m going to need treatment. ‘

‘And then 20 minutes went by and, nothing, maybe a tiny spot, whereas before that would definitely have swollen like a peach and I would have had to treat myself.

“This is real, this works, and I can relax a little.”

Professor Amit Nathwani, a haemophilia expert at University College London who helped develop the gene therapy, said the cost per patient was alarmingly high on paper but that using the drug could actually save the NHS money.

“It’s a lot of money, but if you look at the lifetime cost of gene therapy with factor concentrate replacement, it’s over £8 million per patient,” he said.

‘So a single administration of gene therapy leading to long-term, potentially lifelong protection, could be a bargain and represent a significant saving for the NHS.’

He added that this did not take into account other potential savings for the healthcare system, such as a reduction in the need for life-saving surgery for patients with haemophilia B.

‘Even with regular injections of Factor IX, patients continue to bleed and have life-threatening bleeding. “With gene therapy, the data shows that these spontaneous bleeding events are almost completely eliminated,” he said.

Clive Smith, chairman of the charity Haemophilia Society, welcomed the news of the approval, calling it a “major step forward” for people with haemophilia B.

“It has the potential to significantly improve the quality of life for those eligible for such treatment,” he said.

‘Gene therapy offers people the opportunity to effectively stop painful bleeding, improving joint health and allowing people to live full lives without the limitations of frequent IVs and hospital visits.’

NHS England’s most senior doctor, Professor Sir Stephen Powis, also welcomed the news.

‘This promising drug is the latest in a series of breakthrough gene therapies available to NHS patients at an affordable price. It is the first medicine to be made available through our Innovative Medicines Fund, so patients can access it early while more data is collected on its long-term benefits,” he said.

NHS England added that haemophilia B patients interested in the drug can discuss its suitability for Hemgenix with the healthcare professional responsible for their care.

Hemgenix is ​​made by Philadelphia-based pharmaceutical company CSL Behring, with Eduardo Cabas, managing director of its UK and Irish operations, welcoming NICE’s decision.

“CSL Behring is proud to be a global leader in biotechnology innovation and we remain clearly committed to improving the lives of people with rare genetic bleeding disorders, as demonstrated by this milestone for eligible patients with haemophilia B in the UK and the NHS,” he said.

Hemgenix was initially described as the ‘world’s most expensive drug’ when it first appeared on the scene in 2022.

However, other drugs have since eclipsed it in terms of total cost.