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Yesterday, the Scottish Medicines Consortium (SMC) approved the use of tisagenlecleucel -T (brand name Kymriah), on the NHS in Scotland, for patients with diffuse large B-cell lymphoma (DLBCL). Kymriah is a brand of chimeric antigen receptor (CAR) T-cell therapy and is manufactured by Novartis. This is an update to a previous decision from the SMC, which was not to recommend; for our response to the initial decision by the SMC, please see here.
In response to the decision to recommend tisagenlecleucel-T, our Advocacy Officer, Charlotte, said, “We are glad that the SMC have decided to approve the use of this innovative treatment in Scotland, as it creates parity of access with patients in England. It is disappointing that this was delayed initially by the need for further economic analysis. CAR-T therapy is promising for patients who have exhausted most alternative treatments, and so these are patients in need of effective treatments as quickly as possible.”
This is the second type of CAR-T therapy that has failed to be approved by the SMC for DLBCL. The first was axicabtagene ciloleucel (brand name Yescarta, manufactured by Kite). Specifically, the therapy was being evaluated for use in relapsed or refractory adult patients who have tried two or more other therapies.
However, the SMC have previously approved the use of tisagenleceucel-T for patients with acute lymphoblastic leukaemia (ALL), a different type of blood cancer that affects a similar type of cell to DLBCL.
How does tisagenlecleucel-T work?
Tisagenlecleucel-T is one brand of CAR-T therapy. Also called adoptive T-cell therapy, CAR-T therapy is a type of immunotherapy, meaning parts of the immune system are used as a treatment. Immunotherapy usually uses parts of the immune system made in other animals or in a laboratory, such as antibodies. CAR-T cell therapy is brand new and innovative, involving the harvesting of a patient’s own immune cells from their body to be used in the treatment. The cells are taken from the body, then edited to be more effective at fighting the cancerous cells, then returned to the patient’s blood to start fighting the cells. The severe side effects can arise when the T-cells are returned to the patient and they kill so effectively that they can also harm the rest of the body. This is similar to the reason why flu feels so awful; when your immune system is trying to fight something, sometimes it fights so hard that it makes you feel worse (in the case of the flu, it is not the virus itself that causes many of the symptoms but this very strong reaction by the immune system). The process of CAR-T therapy tailors the treatment to each patient and, therefore, the costs can be high.
CAR-T therapies are currently only considered in relapsed patients who have tried other therapies because the way it works can also cause some severe side effects.
Why was tisagenlecleucel-T initially not recommended?
Initially, the SMC indicated that the company, Novartis, did not present enough evidence to show that the benefits gained by patients was enough to offset this high cost to the NHS, and that this was the basis for their decision. The company had the opportunity to re-submit with further evidence. The SMC has recommended the drug following the presentation of further evidence.
The initial decision created a situation where access to CAR-T therapy varied across the UK for DLBCL patients, as NICE has approved both Kymriah and Yescarta for DLBCL patients in England, whereas Scottish patients have no access to either brand of CAR-T therapy currently. It also created inequity of access to the same type of treatment for patients with different blood cancers, due to SMC approving Kymriah for use in patients with ALL, as mentioned above. Whilst this how now been resolved, it is disappointing that there has been a delay in patients getting treatment in the meantime.
You can find out more about this decision here: https://www.scottishmedicines.org.uk/media/4705/decision-explained-tisagenlecleucel-kymriah.pdf or contact our Advocacy team by emailing advocacy@leukaemiacare.org.uk or calling our helpline 08088 010 444.