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NICE issues ‘draft no’ on first treatment for AL amyloidosis

Patient advocacy news // 20th January 2022

The National Institute for Health and Care Excellence (NICE) has issued a ‘draft no’ for the very first treatment for AL amyloidosis.

The drugs appraisal committee today submitted a draft ‘no’ recommendation for the use of daratumumab (Darzalex®) in combination with cyclophosphamide, bortezomib (Velcade®) and dexamethasone (Dara-CyBord) for people with newly diagnosed AL amyloidosis in England and Wales.

Although all four drugs are already individually available on the NHS, NICE only assesses the combination treatment as a whole.

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.

Jo Nove, Acting Chief Executive of Myeloma UK, a charity advocating for patients with both myeloma and AL amyloidosis, said:

“We’re extremely disappointed by NICE’s decision. There is currently no approved treatment specifically for AL amyloidosis, so this drug combination is a game-changer and, as a recent study has shown, highly effective for newly diagnosed patients. NICE will be reviewing its decision pending further data at a second appraisal committee in March, but the reality of this delay means hundreds of newly diagnosed patients will have missed out on this world-first treatment. We will keep fighting to ensure every patient gets access to the latest, most effective treatment and remain hopeful that an agreement will soon be reached and the drug combination finally be made available to patients.”

Although NICE acknowledged that the new treatment was innovative and helped to halt the progression of the condition, 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 in March.

Myeloma UK Senior Patient Advocacy and Policy Officer, Daniel Cairns, said:

“NICE’s decision to knock back this new drug combination is a setback for the AL amyloidosis community. Patients have told us time and time again how hopeless they feel knowing that they’ve been diagnosed with a condition which has no dedicated treatment option. It’s important to acknowledge the impact this has not only on patients’ physical health but on their mental health too. We know this new drug combination works and helps to improve people’s quality of life. We’ll be making submissions ahead of the next appraisal committee. We are determined to get it approved and finally offer AL amyloidosis patients access to this long-awaited treatment.”

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

“Today’s announcement is devastating news for the amyloid community. 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 was diagnosed with both AL amyloidosis and myeloma, added:

“To us patients this knockback is literally a matter of life and death. 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”.

The drug combination is yet to be reviewed for patients in Scotland and Northern Ireland.

 

What is AL amyloidosis?

‘Amyloidosis’ is a general term used for a group of conditions where an abnormal protein, called amyloid, accumulates in the tissues . AL amyloidosis is the most common type of amyloidosis. It is caused by abnormal plasma cells in the bone marrow. Amyloid can build up in the kidneys, heart, liver, spleen, nerves, or digestive system  . AL amyloidosis is a relatively rare condition, with approximately 1,000 people diagnosed in the UK each year . It can be treated but it 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 . 15% of myeloma patients also have AL amyloidosis.

The NICE appraisal process

What is a ‘draft no’?

A ‘draft no’ does not mean the treatment has been rejected. It means that the treatment has benefits but there are underlying issues which need to be addressed before NICE can approve it.  NICE has now released an Appraisal Consultation Document (ACD) setting out the reasons behind the ‘draft no’ and suggesting what can be done to turn this ‘no’ into a ‘yes’. Learn more about what happens after a ‘draft no’.

Why did NICE say no?

The committee said:

“Clinical evidence suggests that daratumumab in combination increases the time until systemic AL amyloidosis gets worse compared with bortezomib plus cyclophosphamide and dexamethasone. However, the treatment has not been shown to increase how long people live. All the cost-effectiveness estimates for daratumumab are in a range higher than what NICE considers an effective use of NHS resources. Therefore, daratumumab in combination is not recommended.”

NICE felt that other factors should be included in the economic modelling including a truer reflection of treatment administration costs and the cost of patients receiving a stem cell transplant.

The committee found that, as things stood, daratumumab in combination for newly diagnosed AL amyloidosis did not meet end of life criteria but the committee concluded it is an innovative treatment.

The price of drugs is a factor in NICE decisions, but it is important to bear in mind that the committee does not look at whether a drug is expensive. Rather, it looks at whether it is cost-effective. All NICE decisions are based both on the clinical effectiveness and cost-effectiveness of the treatment being appraised compared to the current standard of treatment.

What happens next?

Myeloma UK has until February 10, 2022 to submit a consultation response.  During this consultation period Myeloma UK along with Janssen, the pharmaceutical company manufacturing the drugs, and clinicians will be able to submit further evidence and new information to address NICE’s concerns.  A second committee meeting will be held in March to review the additional evidence provided.

Is it common practice for NICE to reject a new drug/drug combination the first time around?

Most myeloma-related drug approvals result in an initial draft “no” so unfortunately this step in the process is not unusual.  It should be noted that all recent myeloma drugs appraisals (including the quadruplet combination DVTD and lenalidomide maintenance) received at least one draft no recommendation before being approved by NICE.

What about patients in Scotland and Northern Ireland?

The treatment is not available for patients in Scotland but we expect this to be brought for appraisal with the Scottish Medicines Consortium (SMC) in 2022.   The Department for Health in Northern Ireland decides which treatments to fund based on decisions from either NICE or the SMC.