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Another CAR-T treatment provisionally turned down for use on NHS

The Bloodwise logo. Bloodwise appears in black text against a white background
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19 Sep 2018

The NHS medicines watchdog has provisionally recommended that a treatment called Kymriah, which uses the immune system to fight cancer, should not be made available to treat people with diffuse large B-cell lymphoma (DLBCL) who do not respond to standard treatments.

Kymriah was accepted onto the Cancer Drugs Fund (CDF) at the beginning of September for the treatment of children with leukaemia.

The National Institute for Health and Care Excellence (NICE) has asked for more evidence and feedback from the pharmaceutical company and patient groups before making a final decision on whether to approve Kymriah for the treatment of DLBCL.

CAR-T therapy is a ground breaking form of treatment that modifies patients’ own immune cells in a laboratory to recognise, seek out and kill cancer cells. CAR-T therapy is designed to be a one-off treatment and is thought to have the potential to give patients a life-long ‘immunity’ to their cancer.

NICE also turned down another very similar CAR-T therapy, called Yescarta, for use on the NHS last month – also for patients with DLBCL. People with DLBCL who do not respond to, or relapse after, two or more types of treatment have a very poor chance of survival when treated with further intensive chemotherapy.

The NICE committee, in draft guidance issued today, stated that Kymriah is a very promising treatment and has the potential to cure a significant proportion of patients.  They concluded, however, that there was not enough clinical trial evidence available yet on the long-term survival benefits of the treatment to justify the high price of the treatment. 

Trial results seen by the NICE committee showed that 49 per cent of people with DLBCL were alive after 12 months of treatment with Kymriah, but the trial is still ongoing. The NICE committee have asked for more robust data directly comparing the difference in survival rates for patients treated with Kymriah and patients treated with further intensive chemotherapy - the current standard treatment in the UK.

CAR-T therapy is very expensive. Kymriah has a list price of £282,000 per patient but the NHS would receive a confidential discount on this price if it were to be approved. 

Bloodwise is one of the patient organisations that has been consulted in NICE’s appraisal process for both Kymriah and Yescarta.

Dr Alasdair Rankin, Director of Research and Patient Experience at Bloodwise, said: “CAR-T therapies are expensive and while there have been hugely promising early results in clinical trials, long-term survival rates are not known for certain because it is such a new technology.  

“By approving CAR-T therapy to treat childhood leukaemia on the Cancer Drugs Fund earlier this month and investing in the infrastructure needed to make it available, the NHS has made it clear that it is willing to take a chance on these life-saving cancer treatments if enough evidence is available and a price can be agreed.

“People with diffuse large B-cell lymphoma who relapse after multiple treatments have a very poor outlook if they are treated again with chemotherapy. We hope the concerns and questions around CAR-T can be addressed so that these patients can be given access to the chance of a cure.”

DLBCL is diagnosed in around 5,000 people each year in the UK. It is estimated that around 150 patients with DLBCL could benefit from Kymriah each year if it were to be approved.

DLBCL is a cancer of white blood cells called B cell lymphocytes. Sometimes these lymphocytes do not fully develop, divide in an abnormal way or don’t die when they should. These abnormal lymphocytes build up in the lymph nodes, causing them to swell and form a lumps. DLBCL affects how the immune system works and persistent infections are a common symptom.

Find out more: What is CAR-T therapy?