First treatment for AL amyloidosis approved in Scotland

The very first treatment for AL amyloidosis has today been approved in Scotland. The Scottish Medicines Consortium approved the use of daratumumab (Darzalex®) in combination with cyclophosphamide, bortezomib (Velcade®) and dexamethasone (DaraCyBorD) for people with newly-diagnosed AL amyloidosis in Scotland.

This is a major step forward for Scottish patients who, up until now, have had to rely on treatments designed for other conditions, such as myeloma. The news comes six months after DaraCyBorD was rejected by The National Institute for Health and Care Excellence (NICE) for patients in England and Wales.

AL amyloidosis is a rare incurable condition which can affect multiple parts of the body, including the kidney and heart. Around 30 people are diagnosed with AL amyloidosis in Scotland each year. Overall, 1,000 people are diagnosed in the UK every year.

The new quadruplet treatment, which has been shown to be effective in clinical trials, delivers deep responses, with 53% of patients having no sign of AL amyloidosis following treatment and longer remission times. All four drugs are already individually available on the NHS.

Dr Sophie Castell, Chief Executive, said:

“We’re absolutely delighted with the SMC’s decision. This is the very first treatment available specifically for AL amyloidosis and a real game-changer for newly-diagnosed patients in Scotland. This will make a tremendous difference to people’s lives, improve remission times and give many a few more precious years with their loved ones.

“While this is a turning point for patients in Scotland, we can’t forget about the hundreds in England and Wales who are being left behind. The reality is that while Scottish people reap the benefits of this new drug combination, hundreds of newly-diagnosed patients in England and Wales will miss out on this world-first treatment. We will keep fighting to ensure every patient across the UK gets access to the latest, most effective treatment and remain hopeful that DaraCyBorD will finally be made available to those who need it the most, regardless of where they live.”

Myeloma UK Acting Director of Research and Patient Advocacy, Shelagh McKinlay, said:

“This is a momentous day for the AL amyloidosis community in Scotland. For years patients had been telling us how hopeless they felt knowing that they’d been diagnosed with a condition which had no dedicated treatment option. We are now more determined than ever to get it approved in the rest of the UK and offer newly-diagnosed AL amyloidosis patients everywhere access to this life-changing treatment.”

Brian Nicol, from Cambuslang, Glasgow was among the patients making the case for the treatment to become available in Scotland. Brian, 50, had been feeling unusually exhausted for a couple of months when his legs started swelling up. He was quickly admitted to hospital and diagnosed with AL amyloidosis in November 2019. Speaking of the decision, Brian said:

“It’s a very good result. “Anything that can help make things easier, can control the disease and benefit people should be available to patients.”

However, patients in England and Wales are currently waiting for NICE to review its decision. Although NICE acknowledged back in January that the new treatment was innovative and helped to halt the progression of the disease, its appraisal committee felt that, based on the available data, the drugs had “not been shown to increase how long people live” and therefore were not cost-effective for the NHS. NICE is due to review its decision pending further data at a follow-up appraisal committee.

Huw Stiley, 55, from London, served as a patient expert, championing the voice of the AL amyloidosis community at NICE’s initial appraisal committee. He said:

“Amyloid patients deserve to have an approved treatment that offers everyone hope for the future. As a patient I sincerely hope that NICE will be able to reconsider the additional information submitted by the various stakeholders and to progress to the next stage of the drug approval process.”

Patient Charles Montlake, 60, from Greenwich, who has both AL amyloidosis and myeloma, added:

“What about the newly-diagnosed patients who could have benefitted from the drug combination and now will have missed out? They could have had a few more years with this drug combination. These are not new drugs; this is just a new combination. We already know the drugs work”.

Learn more about Health Technology Assessment (HTA) and the role of organisations such as NICE in deciding whether drugs are funded by the NHS.

If you would like to speak to us for information, advice or support, please call the Myeloma Infoline on 0800 980 3332 or email askthenurse@myeloma.org.uk.

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