Myeloma UK fights back after NICE pulls treatment

Myeloma UK is fighting back after a treatment shown to give people at fourth line remissions of over a year was pulled by NICE because of a systems change.

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

It was up for review by The National Institute for Health and Care Excellence (NICE) to decide whether it should be made permanently available on the NHS in England and Wales.

IsaPD has been shown to improve remissions by more than 12 months and has been an important part of myeloma treatment over the past three years. But a recent change in the way NICE assesses treatments has meant that IsaPD no longer meets the cost-effectiveness threshold to be approved on the NHS.

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

Myeloma UK will be appealing the decision.

The drug combination will remain available to patients through the Cancer Drugs Fund for the duration of the appeal process.

Shelagh McKinlay, Director of Research and Advocacy at blood cancer charity Myeloma UK, said: “This decision is a huge blow and many patients will rightly feel like the rug has been pulled from under their feet. It sets a dangerous precedent, not only for people with myeloma but also for other conditions because NICE has suddenly moved the goalposts.

“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.

“IsaPD works and has significantly improved people’s quality of life and remission times since 2020. We simply should not be here; where a vital and effective treatment which has been the standard of care for years cannot be approved. The system is not delivering for patients and we mean to challenge it.

“Myeloma UK has been involved in every committee meeting about IsaPD and we believe this decision is flawed. We will be submitting an appeal, and we won’t rest until IsaPD is available to everyone who needs it, no matter where they live.”

IsaPD has been available to patients since 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.

It is a combination of isatuximab (Sarclisa®), pomalidomide (Imnovid®) and dexamethasone and is aimed at patients whose cancer has returned three times.

Isatuximab belongs to a group of drugs known as monoclonal antibodies and works by attaching to a protein present on the surface of myeloma cells. This flags the cell to the immune system allowing it to target and kill the myeloma cell.

Used in combination with pomalidomide and dexamethasone, it has been shown to give patients average remissions of more than 12 months. That’s around six months longer than for patients on pomalidomide and dexamethasone alone.

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

Under the new system, IsaPD failed to meet NICE’s affordability threshold for use on the NHS in England and Wales.

Yet IsaPD was approved on the NHS in Scotland back in 2021.

Nigel Spencer who was diagnosed with myeloma in 2018 at the age of 59.

He has been receiving IsaPD since 2021 and credits the treatment for allowing him to enjoy “an unexpectedly rich and fulfilling chapter” of his life.

“I am very disappointed by the committee’s decision,” said the retired information professional from North London. “There are real people involved here, people who, when they reach the stage when they need it, won’t be able to access it.

“When you are diagnosed with myeloma your perception of the future changes forever which is a traumatic experience. My attempts to frame the situation in a positive way have been based on a knowledge that there are a range of treatment paths available. However, moving through the treatment lines can see hope turn to anxiety. Therefore any removal of access to an effective treatment option inevitably affects the psychological wellbeing of all myeloma patients at all stages of treatment and directly leads to a substantial reduction in their quality of life.”

Nigel, 65, added: “No previous treatment has given me this length of remission. The absence of significant side-effects has meant that the last two years have been an unexpectedly rich and fulfilling chapter of my life and that of my family. It has been a time of opportunities and options rather than one defined and limited by the constraints of my illness. It is deeply upsetting to think that other myeloma patients in England and Wales will be denied the chance to experience the benefits that I have enjoyed.”

What does this decision mean for patients?

The decision from NICE means that IsaPD will be discontinued as a treatment for myeloma patients in England.

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 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 July 2019.

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.

Why did NICE say no?

NICE agreed that IsaPD improved remission times and life expectancy compared to the treatments currently approved for use at fourth line, daratumumab, and pomalidomide plus dexamethasone. However, they felt there were limitations in the way the treatments were compared because the data for each treatment came from individual studies rather a single controlled clinical trial. This made it harder to confirm the benefit of IsaPD compared to daratumumab and pomalidomide plus dexamethasone.

NICE also felt that the cost-effectiveness estimates for isatuximab plus pomalidomide, and dexamethasone were considerably higher than what NICE considers an acceptable use of NHS resources.

Therefore, isatuximab plus pomalidomide and dexamethasone is not recommended.

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 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 is the Cancer Drugs Fund?

The Cancer Drugs Fund (CDF) is a pot of money which provides cancer patients in England with faster access to the most promising new drugs. The CDF only applies to England. However, Wales has a New Treatments Fund which also aims to speed up access to the latest drugs, but which is not exclusively aimed at cancer. In Scotland there is the New Medicines Fund for people with rare or incurable conditions, including cancer. Unfortunately, no such dedicated fund exists in Northern Ireland.

What is the CDF trying to achieve?

The CDF aims to make promising cancer drugs available to patients before they are fully approved for use on the NHS by the National institute for Health and Care Excellence (NICE). It has three key objectives:

  • Provide patients with faster access to the most promising new cancer drugs.
  • Help ensure more value for money for taxpayers.
  • Offer pharmaceutical companies who price their drugs responsibly a new fast-track route to NHS funding.

The CDF can be thought of as part of the NICE process for reviewing cancer drugs, providing NICE with a third option when appraising cancer drugs.

NICE can say:

Yes – the drug should be routinely available on the NHS.

No – the drug should not be routinely available on the NHS.

Maybe – the drug is recommended for use within the CDF for a set time period while more evidence on the drug is collected.

NICE will use the CDF route if it thinks that a drug shows promise but there isn’t enough evidence for a straight ‘yes’ decision. When taking this ‘maybe’ route through the CDF, NICE will look again at the drug, usually after two years or so, to consider the additional evidence that has been collected before making a final yes or no decision.

What has the CDF done for myeloma?

Ixazomib (Ninlaro®) in combination with lenalidomide (Revlimid®) and dexamethasone was approved by the CDF in December 2017. Following a review, it was approved for continued use on the NHS in January 2023.

In 2018, daratumumab monotherapy became the second myeloma treatment to be approved via the CDF. Following a review, it was approved for continued use on the NHS in March 2022.

Daratumumab in combination with bortezomib (Velcade®) and dexamethasone was approved for use through the CDF in 2019. Following a review, it was approved for continued use on the NHS in April 2023.

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