Victory as Myeloma UK and the UKMS win IsaPD appeal

Published: Wednesday, 13 Nov 2024

Myeloma UK and the UK Myeloma Society have won their appeal which challenged NICE’s decision to not recommend IsaPD.

The appeal panel ruled that the review of the treatment was unfair due to the impact a recent system change had on the appraisal.

In June, the National Institute for Health and Care Excellence (NICE) withdrew IsaPD, an effective and well tolerated treatment which is seen as standard care for patients at fourth line.

Around 1,500 patients have benefited from IsaPD since it was rolled out through the Cancer Drugs Fund back in 2020. 

The treatment is a combination of isatuximab (Sarclisa®), pomalidomide (Imnovid®) and dexamethasone and is aimed at patients whose cancer has returned three times. But a recent change in the way NICE assesses treatments meant that IsaPD no longer met the cost-effectiveness threshold to be approved permanently on the NHS. 

Until recently, NICE had used what is known as an ‘end-of-life modifier’ to weigh up whether a treatment was cost-effective. But, in 2022, the ‘end-of-life modifier’ was replaced by a ‘severity modifier’.   

IsaPD was initially approved under the ‘end-of-life modifier’ and rolled out in November 2020 through the Cancer Drugs Fund – a pot of money which provides cancer patients in England with faster access to the most promising new drugs pending full approval from NICE.   

However, when it was reviewed under the new ‘severity modifier’ back in June, IsaPD failed to meet NICE’s affordability threshold for use on the NHS in England and Wales.  

This was the first time a myeloma treatment approved through the Cancer Drugs Fund had been rejected by NICE for permanent use on the NHS. 

Myeloma UK and the UKMS immediately appealed the decision and, five months on, an independent panel has now upheld the appeal, agreeing that NICE’s review was unfair.

The panel ruled that NICE had acted unfairly by neglecting to consider the significant impact that a recent change in NICE methods and process had on the appraisal. They believed that NICE should have considered applying flexibility outside its usual processes because of the exceptional circumstances of this evaluation. 

NICE’s appraisal committee will now have to reconvene to address the appeal’s findings before making a new decision. 

The decision by Myeloma UK and the UKMS to appeal means that the treatment has remained available and can be offered to patients not currently on IsaPD whilst the appeal is in progress. People who are currently on IsaPD are not affected by this decision.

Shelagh McKinlay, Director of Research and Advocacy at blood cancer charity Myeloma UK, said:

“We have been fighting this decision for months and we are delighted to have won our appeal. The appeal panel felt, as do we, that the review of IsaPD was unfair and that NICE should have considered showing more flexibility in light of what were unique and unprecedented circumstances.
“The ball is now in NICE’s court. We’re urging them to do the right thing, reconsider their decision, show some flexibility, and make this life-changing treatment permanently available on the NHS as soon as possible.
“In practice, the new system has made it much harder to get treatments to patients who are nearing the end of their life – despite the fact that the right treatments, like IsaPD, could keep their cancer at bay for longer.
“We also believe that it has an inbuilt bias against certain conditions and groups of patients – especially older people. This is doubly unfair since the majority of people with myeloma are diagnosed later in life.
“Myeloma UK will continue to work with the UKMS on this issue to make the case for IsaPD to be made available to those who need it. Every day counts when you’re living with myeloma because only one thing is certain: myeloma will always come back. This means patients need the best, most effective treatments now.”

Meanwhile, IsaPD was approved by the Scottish Medicines Consortium (SMC) for use on the NHS in Scotland in 2021.  

Myeloma patient Jon Missin, from Brixworth, Northamptonshire, was told he would likely get another 11 to 12 months on IsaPD back in 2021. But three years on, the father-of-one continues to defy the odds.

This has been his longest remission from cancer to date.

He has now hailed the appeal panel’s ruling as a return to common sense.

“This is truly fantastic news,” said Jon, who was diagnosed with myeloma in 2015 at the age of 41. His son was two at the time.

“Thank goodness common sense has prevailed. This treatment has given so many patients a lifeline since its inception. For me and many others who have gained many more months and years from this treatment than we ever expected, I am truly thankful.
“IsaPD has enabled me to enjoy life, to enjoy holidays and time with my son and my family. My son is 11 now and I never thought when I first heard about myeloma that I would see him start primary school. After I did, my target was to see him finish primary school and settle into secondary school, and thanks to the extra time IsaPD has given me, it’s another target ticked off which is immense.”

He added:

“Once again, the team at Myeloma UK have shown their steadfast commitment by not accepting an outcome that has gone against the myeloma community, and have built such a commendable case it could not be ignored. Hats off to all involved and a huge thank you.”

Jon didn’t think too much of it when he started experiencing back pain back in 2015.

A keen runner, he was no stranger to aches and niggles, so he booked an appointment with an osteopath. But after a couple of sessions, his osteopath began to suspect something was seriously wrong and told Jon to call his GP and ask for an MRI scan.

The scan showed a tumour, known as a plasmacytoma, was wrapped around his spinal cord. Jon had to get surgery to have it removed. The tumour was so large, Jon was told the operation could leave him paralysed.

Thankfully, he narrowly avoided paralysis.

He said:

“It was a very scary time. The plasmacytoma was wrapped around my spinal cord and they had to remove part of three of my vertebrae to remove it. They said there might be a chance I could come out paralysed if not permanently, at least for a period of time. I was very lucky.”

The tumour, it turned out, was caused by myeloma.

Jon also had holes, known as lesions, in his bones, including a very large one in one of his ribs. As a result, he had to undergo a second operation to remove part of his rib. He also had radiotherapy.

He went on to receive chemotherapy to treat his myeloma, followed by a stem cell transplant in 2016. He was in remission for three years before his cancer came back.

Eventually, he was put on IsaPD. He was told he’d likely get another 11 to 12 months on the treatment, but three years on, Jon is doing well.

When Jon found out IsaPD had been rejected by NICE back in July, he was “gobsmacked”.

He said:

“I was absolutely gobsmacked. It seemed bizarre. I was dumbfounded. Talking to other people, I know I’m not alone. Other people have gone beyond the 11 to 12 months we were initially told we’d have. Surely that must count for a lot. You can’t help thinking it’s a cost thing. But what cost do you put on an extra two years of life?
“It’s taking away a huge opportunity for a lot of people. I can’t believe that could be justified.
“To have been given the opportunity to make many more memories with my family and friends, you just cannot put a price on it. I am delighted the appeal has been upheld and that many more patients in future could have the same opportunity that I had.”

What is IsaPD?

IsaPD is a combination of three drugs: isatuximab, pomalidomide and dexamethasone.

It is used to treat patients who have relapsed multiple times. In the UK it is typically used to treat patients who have had three previous lines of treatment, including lenalidomide and a proteasome inhibitor, and whose disease has progressed on their last treatment.

Isatuximab belongs to a group of drugs known as monoclonal antibodies. It works by harnessing the body’s immune system.

It is a synthetic antibody designed to target myeloma cells by detecting a protein commonly found on the surface of myeloma cells called CD38. When it finds and attaches to CD38, it makes the myeloma cell more visible to the immune system. It helps the patient’s immune system locate and kill myeloma cells.

What does the decision mean for patients and clinicians?

It means that NICE has to review its decision not to recommend IsaPD as a treatment for myeloma patients in England. NICE needs to consider showing additional flexibility when assessing the cost-effectiveness of IsaPD due to the recent systems change.

The decision does not affect patients currently on IsaPD, they will continue to get their treatment as long as it is working for them.

The treatment will remain available and can be offered to patients not currently on IsaPD whilst an appeal is in progress.

What about patients in Scotland, Wales and Northern Ireland?

In Scotland, the Scottish Medicines Consortium makes decisions about which treatments to fund through the NHS. The SMC approved IsaPD for use through NHS Scotland in April 2021.

In Wales, the All-Wales Medicines Strategy Group (AWMSG) makes decisions about which treatments to fund through the NHS. It usually follows NICE decisions.

In Northern Ireland, the Department for Health makes decisions on which treatments to fund through the NHS. It can follow guidance issued by either NICE or the SMC but usually follows the guidance issued by NICE.

What is the Cancer Drugs Fund?

The Cancer Drugs Fund (CDF) is a pot of money which helps cancer patients in England get faster access to promising new drugs.

NICE can approve a treatment through the CDF when a treatment shows promising results in trials but there isn’t enough evidence to permanently recommend it.

After a period of two years, NICE reconsiders the medicine and makes a final decision whether to recommend it or not.

What is a modifier?

Modifiers are tools used by NICE to evaluate new treatments. They are used to give certain types of treatment, or treatment benefits, a higher value when assessing cost effectiveness. Since 2022, NICE has been using the severity modifier to review treatments. It lets NICE increase the value of treatments for severe diseases and means NICE is willing to pay more for these treatments. The severity modifier looks at the impact the health condition and the new treatment has on life expectancy and quality of life.

Between 2009-2022, NICE used a different modifier, called the end-of-life modifier. It let NICE increase the value of treatments for people whose life expectancy without the treatment was less than two years, if the treatment extended lives by more than six months.