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How Rob Burrow will transform MND diagnosis and care 

Rob Burrow has already had a “transformational” affect on motor neurone disease patients and their families, the MND Association has said.

Richard Evans, director of engagement at the charity, said that children and young people especially are benefiting from the increase in support services funded by Burrow and his former Leeds Rhinos teammate Kevin Sinfield. The pair raised more than £8m before Burrow died on Sunday at the age of 41, following a lengthy battle with motor neurone disease.

Mr Evans said the money, which has been spread across several charities, including the MND Association, has made many practical day-to-day differences to people’s lives.

“Some of that money has gone to support services for children and young people in particular, helping them to cope with a diagnosis of MND within the family. Also in care centres and networks to improve multidisciplinary care, so care is co-ordinated around the individual,” he said.

“We’ve also invested in our support line so people affected by MND can call. We’ve invested some money in a research nurse network which, when we get it all up and running, will help people with MND to access clinical trials and understand what trials might be open to them. We’ve also funded more research as well to try and understand better the biology of MND.”

Burrow lived in the public eye with the disease after choosing to announce his diagnosis shortly before Christmas 2019. He used every opportunity to raise awareness of the disease, speaking publicly, undertaking media interviews and opening his life up in two moving BBC documentaries My Year With MND and Rob Burrow: Living With MND.

Rob Burrow (centre) with his wife Lindsey Burrow and Kevin Sinfield (right) during a media call for the Rob Burrow Leeds Marathon. Picture date: Friday May 10, 2024. PA Photo. See PA story ATHLETICS Leeds. Photo credit should read: Danny Lawson/PA Wire. RESTRICTIONS: Use subject to restrictions. Editorial use only, no commercial use without prior consent from rights holder.
Rob Burrow (centre) with his wife Lindsey Burrow and Kevin Sinfield (right), with whom he raised more than £8m for MND support (Photo: Danny Lawson/PA)

Sinfield said he was “pretty heartbroken” as he paid tribute to his friend and former teammate, calling him a “superhuman superman”. He said the loss was “pretty raw still” as he attended a groundbreaking ceremony for the Rob Burrow Centre for MND at Seacroft Hospital in Leeds on Monday.

Fighting back tears, Sinfield said: “I wish he could have seen the outpouring of love. I think we all lose special people but it’s very, very rare you lose someone who’s so special to so many different people. As you can see, it’s pretty raw still.

“He’ll leave a massive hole and I know there will be a lot of people out there who are heartbroken this morning at the news from yesterday.

“What’s really important is Rob Burrow continues to live for ever. I’m sure the MND community and everyone who’s supported us previously will make sure that Rob’s name is at the forefront of everything we do going forward.”

The MND Association said Burrow was a “passionate advocate” for people with the “brutal” disease, while the Prince of Wales said that the former England international had a “huge heart”.

Mr Evans said: “I can’t put too much stress on what an inspiration Rob was for the whole MND community, for the awareness he raised, it really makes such a difference to people. His death has been felt hugely in our community and he’ll be missed hugely by us at the MND Association, the rugby community and, more broadly, the country at large.

“When I speak to people [affected by MND] one of the things that comes up again and again is the courage with which Rob and his family lived with the condition. Talking openly about it really made life easier for everyone else with MND, because there was a sense that the awareness he raised really helped the public at large understand what they were going through. That’s a legacy he will leave for people in the future. His courage and determination with which he faced his challenge inspired everyone in that community too as well as the whole country.”

Mr Evans said research into MND over the past 20 years has not led to the same number of breakthroughs as seen in many other conditions, which has been “deeply frustrating”.

He said: “Only one new treatment has come through, called Riluzole, which has only extended life by a modest amount. What we have developed is a much better understanding of the biology of MND. We’ve put ourselves in a position where we understand what is happening more and, hopefully, we’re now on the precipice of making that big discovery.”

The MND Association is locked in a battle with the drugs watchdog over approval for a “fantastic” treatment that would help up to 100 people with the devastating condition, Mr Evans said.

Tofersen, developed by Biogen, is targeted to treat people living with motor neurone disease who have alterations in the SOD1 gene. It has already been approved for use in the United States after trials showed it slows the disease’s progression. Last week it became the first approved treatment in the European Union for a therapy targeting a genetic cause of MND, but the charity believes it will be denied to patients on the NHS.

The MND Association wants the National Institute for Health and Care Excellence (NICE) to assess the drug through its Highly Specialised Technologies (HST) route, which it believes will make it more likely to be approved on the NHS. However, NICE is refusing to do so saying it will use its single technology appraisal (STA) route with the cost of the drug expected to be a barrier under that criteria.

Tofersen, developed by Biogen, is targeted to treat people living with MND who have alterations in the SOD1 gene – approximately 2 per cent of the MND population, estimated at 60-100 people in the UK. Ordinarily, products targeted to rare diseases that affect less than 1 in 50,000 people in England are appraised through the HST route, but NICE has confirmed it will conduct a standard appraisal on the basis that it does not view SOD1 MND as a clinically distinct disease.

NICE applies a “cost-effectiveness” threshold of up to £30,000 for a drug that gives a patient a year of good-quality life, known as a Qaly, but the figure has not changed since it was set in 2004. The threshold would today be closer to £50,000 if it had increased in line with inflation.

Tofersen is expensive because it is a genetic therapy administered by monthly lumbar puncture and requires short spells in hospital. If it was assessed as a treatment for an extremely rare condition for which there is a £100,000 threshold.

Richard Evans, director of engagement at The MND Association, told i: “Fundamentally, it comes down to a question of money – whether Nice would be prepared to grant approval based on cost. We are arguing that because Toferson is only going to help around 60 people, that they should be routing it via the HST route, where there is a higher threshold to how much they will pay.

“Nice are, at the moment, being quite firm that it should be done via the standard route. We think that is the wrong decision and are calling on them to change that. We’ll continue to make that case over the months to come, but that’s not an argument we’ve won yet, so we’re behind Europe as a result that.”

Mr Evans said he hoped Tofersen would be the first of several new treatments for MND, which affects up to 5,000 adults in the UK, if approved for use on the NHS.

“[But] it’s already clear it’s going to be an uphill struggle to make Tofersen a standard treatment on the NHS, because it’s expensive. So we’ll be working with Nice and the pharmaceutical industry to ensure those treatments are available for people with MND, as there’s no point developing fantastic treatments if they’re not available to the people who need them.”

Tofersen does not currently have a marketing authorisation in the UK for treating MND. This means it will need to gain a licence for use from the Medicines and Healthcare products Regulatory Agency (MHRA) for Nice to appraise its clinical and cost effectiveness for use in the NHS in England and Wales.

It is understood the argument centres on evidence of MND prevalence. To meet the criteria to be considered for Nice’s HST programme the disease must very rare, defined as 1 in 50,000 in England. And prevalence estimates of MND vary depending on how the condition is defined with a range of 3-13 in 100,000. Officials said there are geographical variations but prevalence is likely to be larger than 1 in 50,000.

A Nice spokesperson said: “Our topic selection oversight panel carefully considered whether to route the technology to the Highly Specialised Technologies rather than the Single Technology Appraisal programme. Our initial decision has been reconsidered following a challenge by the company, drawing on additional clinical expertise, but this therapy does not meet the criteria for the HST programme. Nice will not therefore be reviewing this decision.”

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