Thousands of people in England and Wales with an incurable disease will be denied access to a treatment that could extend their lives by at least five years, just three months after it was approved in Scotland.
In a major blow to patients, the National Institute for Health and Care Excellence (NICE) has rejected the first treatment for AL amyloidosis for profitability reasons.
The drug, called Dara-Cybord, has gone through three rounds of evaluation but will be rejected on Friday when NICE announces its final decision. The officials concluded that there was insufficient data to show how much longer patients lived after treatment, Yo understands
The treatment was made available to newly diagnosed patients in Scotland. at the end of august and is considered a huge step forward for patients. Until then, they had to rely on treatments designed for other conditions, such as the incurable blood cancer myeloma.
The Northern Ireland Department of Health decides which treatments to fund based on decisions from NICE or the Scottish Medicines Consortium (SMC). As the SMC made its decision first and was based on a similar number of patients, people in Northern Ireland should be able to access treatment in due time.
AL amyloidosis is a rare and incurable condition that affects multiple parts of the body and can severely damage the kidney and heart. It is a relapsing-remitting condition, which means that patients have periods of remission after treatment, when AL amyloidosis is not active or causing symptoms, but will flare up again after a period of time. Life expectancy varies greatly depending on the level of organ damage, how the condition is treated, and how well patients respond to treatment.
Around 3,500 people have AL amyloidosis in England and Wales and around 1,000 people are diagnosed in the UK each year. As it stands, around a quarter of patients die within a few months of being diagnosed.
What is AL amyloidosis?
AL amyloidosis is caused by an abnormality in certain cells found in the bone marrow, called plasma cells. The abnormal plasma cells produce abnormal forms of light chain proteins, which enter the bloodstream and can form amyloid deposits.
Healthy people have normal light chain proteins in their blood that are part of their natural antibody proteins. These help protect the body from infection. The abnormal light chains in patients with AL amyloidosis clump together into threads (amyloid fibrils) that cannot be easily removed by the body.
Over time, amyloid fibrils accumulate as AL amyloid deposits in tissues and organs. This causes them to gradually stop working properly, causing the many symptoms of AL amyloidosis. Unlike other types of amyloidosis, AL amyloidosis is not inherited, so a person with the condition cannot pass it on to their children.
Dara-Cybord, a pioneering four-drug combination, has been shown to be effective in clinical trials with patients five times more likely to have a complete response to treatment, meaning they go into remission. A 2015 study found that 55 percent of patients with newly diagnosed AL amyloidosis treated with three of the drugs (Cybord) it is estimated that they survive five years. Fifty-nine percent of patients who received Dara-Cybord also showed no signs of AL amyloidosis after treatment and longer remission times, a 2020 study found.
The new quad treatment would have seen the use of daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone. All four drugs are now available individually on the NHS.
A source told of the decision said patients would be devastated: “How can one part of the UK approve the drug on the NHS and another reject it, based on almost the same presentation?
“Anyone recently diagnosed with AL amyloidosis in Scotland and given the combination treatment is likely to live at least several more years, while someone a few miles away goes without or is forced to spend thousands of pounds trying to get it from private way. More people are diagnosed every year in the UK and it needs to be available to everyone.”
Activists believe that Dara-Cybord has a chance to break into the Cancer Drugs Funda means by which NHS patients in England could have NICE reject cancer drugs because they were not cost-effective, or for a review to take place when the overall data on survivors is clearer.
A NICE spokeswoman said it expected to publish the decision on Friday and that “it was not possible to share any information before the publication date.”