Blow for patients at fourth line as treatment knocked back by NICE

A treatment shown to give patients at fourth line remissions of over a year has been knocked back by NICE.

The National Institute for Health and Care Excellence has issued a draft ‘no’ for the use of isatuximab (Sarclisa®), pomalidomide (Imnovid®) and dexamethasone (IsaPD) for myeloma patients who have already received three rounds of treatment in England and Wales.

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.

The treatment was up for review to decide whether it should be made permanently available to patients on the NHS in England.

Although NICE agreed that IsaPD works, its appraisal committee felt it could not accurately assess whether it would be cost-effective for the NHS at fourth line.

The committee is due to reconsider its decision in March pending further evidence.

The drug combination will remain available to patients through the CDF until a final decision is reached.

Over the years, IsaPD has been shown to increase remissions for patients at fourth line by more than 12 months on average.

The triplet treatment has been available on the NHS in Scotland since 2021.

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

“We know this will be really disappointing news for myeloma patients, especially since IsaPD has already been available on the NHS in Scotland for more than two years.

“This is a very challenging appraisal but it’s important to remember that this is not a final no and we’ll redouble our efforts to make sure an agreement is reached, and that IsaPD is approved permanently on the NHS in England and Wales.

“We are committed to making sure people with myeloma have access to the latest, most effective treatments no matter where they live in the UK.”

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.

Around 1,500 patients have benefited from the treatment since it was rolled out through the CDF.

Among them is 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.

The retired information professional from North London said:

“I am very disappointed by the committee’s recommendation. This is the first treatment which has brought my light chains down to normal levels and 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 may be denied the chance to experience the benefits that I have enjoyed.”

The father-of-one added:

“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.

“I recognise that this was an initial recommendation and remain hopeful that the review process may see a change in the decision.”

Dr Neil Rabin, Consultant Haematologist at University College London Hospitals and North Middlesex University Hospital, was one of clinical experts advocating for IsaPD to be approved at the NICE appraisal committee. He said:

“I have always been impressed at how well patients respond to isatuximab with pomalidomide. It’s well tolerated and a great addition to our armamentarium. I really hope this will continue to be made available for our patients.”

What does this decision mean for patients?

The recommendation from NICE is a draft decision based on the evidence the committee has received and heard during the appraisal so far.

Draft decisions don’t affect patient’s treatment of care. Patients receiving IsaPD will continue to get their treatment and the treatment will remain available to eligible patients.

Draft decisions are published to give the people and organisations involved in the appraisal the chance to see and respond to the committee’s decision.

Myeloma UK will respond to the consultation and continue to work with clinicians, the pharmaceutical company and NICE to make the case for permanent approval of IsaPD.

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.

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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