VICTORY FOR PATIENTS DENIED ACCESS TO FIRST EVER AL AMYLOIDOSIS TREATMENT

Charity Myeloma UK has claimed a hard-fought victory after a panel ruled a decision to block access to the first treatment for the incurable disease AL amyloidosis on the NHS was ‘unfair’. 

In December 2022, the National Institute for Health and Care Excellence (NICE) rejected DaraCyBorD, the very first treatment for AL amyloidosis for newly-diagnosed patients in England and Wales, on the grounds it was not cost-effective. 

This was a huge setback for patients who currently rely on treatments designed to target other conditions. 

The decision came just three months after DaraCyBorD was approved and rolled out in Scotland on the NHS. 

Blood cancer charity Myeloma UK, along with patients, appealed the decision and launched the #UnlockDaraCyBorD campaign. 

Five months on, an independent panel has upheld the appeal, agreeing that NICE’s decision was unfair and its appraisal committee may have incorrectly interpreted key evidence. 

NICE’s appraisal committee will now have to reconvene and review all the evidence before making a new decision. 

A pioneering four-drug combination, DaraCyBorD has been shown to be effective in clinical trials with patients five times more likely to have a complete response to treatment. 

This means that 59% of the patients who received DaraCyBorD had no sign of AL amyloidosis following treatment and longer remission times. 

The first of its kind, the treatment combines daratumumab (Darzalex®) with cyclophosphamide, bortezomib (Velcade®) and dexamethasone. 

All four drugs are already individually available on the NHS. 

Dr Sophie Castell, Chief Executive at Myeloma UK, has now thanked patients, clinicians and all those who backed the #UnlockDaraCyBorD campaign and fought with the charity to win the appeal. 

She said: “I want to say a very heartfelt thank you to everyone who has fought alongside us and helped us win this appeal. We could not have done this without your determination and support. We know this new drug combination works and improves quality of life and remission times and this hard-earned victory takes us one step closer to unlocking DaraCyBorD for people in England and Wales. Access to vital treatments should not come down to a postcode lottery. We will continue to work with NICE to make this game-changing treatment available to every last person who needs it, no matter where they live.” 

AL amyloidosis is a rare, incurable condition which affects multiple parts of the body and can severely damage the kidney and heart. 

Overall, between 500 and 600 people are diagnosed with AL amyloidosis in the UK every year. 

As it stands, around a quarter of patients die within six months of being diagnosed. 

Between 3,000 and 4,000 people have AL amyloidosis in the UK. 

During the appeal hearing, Myeloma UK argued that NICE had acted unfairly – by failing to invite an AL amyloidosis expert or a haematologist to participate in the review process – and that decisions regarding treatment benefits and cost-effectiveness were unreasonable in light of the evidence submitted.  

The independent panel found against NICE on all counts. 

AL amyloidosis is a relapsing-remitting condition. This means patients have periods of remission after treatment when the AL amyloidosis is not active or causing symptoms, but it will become active again after a period of time. 

Life expectancy is highly variable depending on the level of organ damage, how the condition is treated and how well patients respond to treatment. 

Those who respond well to treatment have a life expectancy of ten years or more. 

Patient Michael Jameson, from Walsall, was just 41 when he was diagnosed with both AL amyloidosis and the incurable blood cancer myeloma back in December 2021. He was initially given three months to live.  

Thankfully, the father-of-two was able to receive DaraCyBorD through private healthcare. 18 months on, Michael has had a complete response to treatment. 

“It’s given me another chance at life - I would not be alive without it,” said Michael, who has two daughters, Grace and Emily, aged 10 and six, respectively. 

Michael first started feeling pain in his chest and left arm in September 2021 and was admitted to hospital with a suspected heart attack. After a month in hospital, he was diagnosed with myocarditis, an inflammation of the heart muscle and put on a six-week rehabilitation programme. But his symptoms only worsened, and he began to experience nausea and vomiting. He lost four stone. 

Finally, on 23 December 2021, he was diagnosed with both the incurable blood cancer myeloma and the incurable disease AL amyloidosis.  

“I was given a nominal life expectancy of three months which, for a 41-year-old man with two young children, was devastating not only for myself but also my family.” 

“When I started the treatment I was in a really bad way,” recalled the 42-year-old, who works for an aerospace company. “I had dangerously low blood pressure. I was physically drained of all energy and practically bedbound. Due to the vomiting and lack of activity I had lost over four stone in weight since I initially became symptomatic and I was now struggling with everyday tasks. I would become breathless just sitting up.  

“Since then I’ve managed to go on two family holidays and I got married in September 2022. I started working again. I’m doing all the things that a normal person would do.  

“For those who have had this treatment, it’s a no-brainer. Just look at what it’s doing and you can see how effective this treatment is.” 

For more information about AL amyloidosis or to get in touch with Myeloma UK go to www.myeloma.org.uk. Myeloma UK runs an Infoline on 0800 980 3332.