NHS watchdog approves ‘world’s most expensive drug’ that costs £2.6million a dose… and it will SAVE the health service money, say experts

A gene therapy once billed as the ‘world’s most expensive drug’ will be offered for free on the NHS, but experts insist it will save the health service money. 

Called Hemgenix the drug costs £2.6million per patient, although only one dose is needed.

It is the only treatment of its kind for haemophilia B, a bleeding disorder where the body doesn’t make enough, or any, of a protein critical to clotting.

Clotting is a vital biological mechanism that stops wounds from bleeding meaning people with the disorder can suffer severe and even life-threatening blood loss if they suffer an injury.

Additionally, they also run the risk of what are called ‘spontaneous bleeds’ that occur without a direct injury and are potentially deadly if they occur in a vital organ. 

Haemophilia B patients have to take frequent injections of an artificial clotting agent called Factor IX for life in order to keep their risk of catastrophic injury to a minimum. 

But with Hemgenix the patient’s defective gene, which is incapable of producing the clotting, is replaced with one that can, eliminating the need for these injections.

Studies on the gene therapy, given via an IV drip, have shown the effects last for at least three years but the hope is it could work for even longer.

While there are approximately 2,000 people with haemophilia B in the UK, only 260 are estimated to be eligible for Hemgenix on the NHS.

This puts the potential total bill to the taxpayer at about £676million.

However, MailOnline understands the NHS is acquiring the drug at an undisclosed discount, so the actual cost is likely to be less. 

The UK’s National Institute for Health and Care Excellence (NICE), which decides what drugs should be available on the NHS and to whom, recommended the drug be made available on the health service on a ‘managed access’ basis. 

This means it will only be offered to patients with ‘moderately severe or severe haemophilia B’ whose doctor thinks it would benefit them. 

It will also be subject to an ongoing review regarding its cost effectiveness. This could mean it will one day be rolled out to more patients. 

One patient who is already benefitting is Elliott Collins from Essex.

The 34-year-old who has severe haemophilia B and took part in trials for the gene therapy five years ago.

Speaking on the Today programme, Mr Collins said he was effectively no longer a haemophiliac thanks to the treatment. 

‘Initially my clotting levels were below zero and now they are teetering around 60, and anything 50 and above you’re technically not a haemophiliac,’ he said.

‘Not long after I had the treatment, I remember walking past and whacking my knee on a cupboard…and initially thinking ‘Ooh, I’ve whacked that hard enough for that to swell up quite badly, and I’m going to need treatment’.

‘And then 20 minutes passed and, nothing, maybe a little mark, whereas beforehand that would have definitely swelled up like a peach and I would have had to have treated myself.

‘This is real, this is working, and I can relax a little bit.’

Professor Amit Nathwani, an expert in haemophilia at University College London who helped develop the gene therapy, said while on paper the cost per patient is eyewatering, using the drug could in fact save the NHS money. 

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

‘So, a single administration of gene therapy that leads to long term, potentially life-long, protection could be bargain, could be a substantial saving to the NHS.’

He added that this didn’t account for other potential savings for the health service such as a reduction in need for life-saving operations for haemophilia B patients. 

‘Even with regular injections with Factor IX patients continue to bleed and have life-threatening bleeds, with gene therapy the data shows these spontaneous bleeds are almost completely eliminated,’ he said. 

Clive Smith, chair of the charity the Haemophilia Society, welcomed the news of the approval calling it a ‘major step forward’ for people living with haemophilia B. 

‘It has the potential to significantly improve the quality of life of those who are eligible for such treatment,’ he said. 

‘Gene therapy provides an opportunity for people to effectively eliminate painful bleeds, thereby improving joint health and allowing people to lead a full life, unrestricted by frequent infusions and trips to hospital.’ 

NHS England’s most senior medic Professor Sir Stephen Powis also hailed the news.

‘This promising drug is the latest in a series of pioneering gene therapies secured for NHS patients at an affordable price and becomes the first drug to be made available in our Innovative Medicines Fund to provide early access for patients while further data is collected on its long-term benefits,’ he said. 

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

Hemgenix is made by Philadelphia-based pharmaceutical company CSL Behring, with Eduardo Cabas, general manager of its UK and Ireland operations, welcoming NICE’s decision. 

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

Hemgenix was initially described as the ‘most expensive drug in the world’ when it originally came onto the scene in 2022.

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