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Specifically, the therapy was being evaluated for use in relapsed or refractory adult patients who have tried two or more other therapies. 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). However, the SMC has previously approved the use of Kymriah for patients with acute lymphoblastic leukaemia (ALL).
CAR-T therapies are currently only considered in relapsed patients who have tried other therapies as the way it works can also cause some severe side effects. CAR-T therapy, also called adoptive T-cell 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 awful (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.
The SMC indicated that the company, Novartis, did not present enough evidence to show that the benefits gained by patients were enough to offset this high cost to the NHS, and that this was the basis for their decision. The company now have the opportunity to re-submit with further evidence, although this causes a delay in access while this process is completed.
This decision creates a situation where access to CAR-T therapy varies 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 creates 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.
At the time of approval in England, the head of NHS England described Kymriah as a “true game changer”. Our Patient Advocacy Director, Zack Pemberton-Whiteley, said of today’s SMC decision: “CAR-T therapy is promising for patients who have exhausted most alternative treatments. We are disappointed that the SMC has chosen to not fund Kymriah, despite having chosen to fund it for patients with other conditions and funding being available in England, so all patients are able to access this important new treatment as soon as possible and wherever they live in the UK.”
Video: Zack Pemberton-Whiteley discusses CAR-T at EHA 2018