Page last updated on: 26th April 2023
What is the news?
Today NICE has announced that CAR-T therapy will now be provisionally approved for use on the NHS in England as part of the Cancer Drugs Fund (CDF). It will be used to treat eligible patients with acute lymphoblastic leukaemia (ALL) who are over the age of 25. Until now, CAR-T therapy has only been available to younger ALL patients on the NHS, those aged 25 and under.
What is the Cancer Drugs Fund (CDF)?
The CDF is a pot of funding which exists so that NICE can approve a treatment on the NHS in England for a limited period of up to 2 years in order to gather more real-world evidence on the impact of the treatment. This is evidence which NICE did not have enough of at the time of the appraisal, and which they need in order to make a permanent decision about whether the treatment is cost effective enough to be approved on the NHS long-term. Access will be granted for a maximum of 2 years, within which NICE will decide to undertake another review of this treatment as soon as they have enough evidence to do so.
Who are NICE and what do they do?
The role of the National Institute for Health and Care Excellence (NICE) in England is to decide if a treatment is cost-effective and therefore suitable to be used on the NHS. They do this by comparing the treatment’s effectiveness and price to existing treatments, if there are any. The process therefore involves looking at clinical trial data, information on the cost of delivering the new drug. A committee of people at NICE then makes a recommendation based on the evidence they have.
Why has NICE made this decision?
NICE reviewed a study which suggests that people having this CAR-T treatment may live longer and have more time before their disease relapses. They also looked at evidence which suggested that this CAR-T treatment can cure B-cell ALL. However, NICE requires more evidence in order to show that approving the treatment long-term would meet the cost-effectiveness criteria for use in the NHS.
By provisionally approving CAR-T NICE hopes to be able to gather more data to enable them to make a final decision.
Which groups of patients can access this treatment?
CAR-T will be available provisionally for relapsed/refractory B-precursor acute lymphoblastic leukaemia in adults (also known as B-cell ALL). CAR-T is therefore only to be used in patients for whom other treatments have not worked. 75-80% of ALL cases are classified as B-precursor ALL. The decision to treat a patient with CAR-T will be made by the patient’s clinician.
If you are unsure whether this is a treatment which might be available to you, please consult your doctor or clinician.
What does the treatment involve and how does it work?
CAR-T refers to chimeric antigen receptor T-cell therapy. CAR-T describes the process of removing the patient’s T cells and genetically altering them to make them recognise and target specific cancer cells before they are reinfused back into the patient.
There are multiple types of CAR-T products. This is the first CAR-T treatment to be approved for adults with ALL. This particular CAR-T treatment is called brexucabtagene autoleucel, known by the brand name Tecartus. It is manufactured by the pharmaceutical company Kite, which is part of Gilead Sciences.
The CAR-T treatment process requires a number of steps that take place over several weeks. The aim of CAR-T is for the modified T cells to stay in the body for a long time, recognising and attacking the specific cancer cells to give the patient the best chance of long-term remission.
How was Leukaemia Care involved?
Leukaemia Care submitted a written response to NICE representing the views of ALL patients by drawing on experiences and data, and advocating for adults to have equal access to this potentially life-saving treatment. Sophie Wheldon is Leukaemia Care’s Advocacy Officer and also an ALL patient. Sophie spoke to the NICE committee as the patient expert, drawing on her personal experience to advocate for all ALL patients. Sophie was treated with CAR-T therapy on the NHS in 2019, as she was eligible for the treatment due to being under 25 at the time. Sophie has now been in remission for over 3.5 years and describes CAR-T as saving her life. Sophie and Leukaemia Care felt strongly that CAR-T should be a treatment available to all ALL patients regardless of their age. On the announcement of the provisional approval of this treatment by NICE, Sophie commented:
“I am so pleased to hear that CAR-T therapy is going to be available to ALL patients over the age of 25. This is something that I have felt passionately about from the moment I knew I needed CAR-T therapy, as that at the time, my sister would not have been eligible if it were her in my position as she was over the age threshold.
This decision will bridge a big unmet need for patients who will undoubtedly benefit from the treatment, and it will hopefully allow more patients to achieve remission where their other treatment options have previously been extremely limited or completely exhausted.
This is a wonderful first step in providing access for these groups, and I am hopeful that the decision to make this treatment available through the Cancer Drugs Fund will lead to long-term access for other patients in the future.”
Leukaemia Care are pleased with NICE’s decision to provisionally approve CAR-T for eligible adults (over 25), and acknowledge that having it available through the CDF gives patients access to this treatment which they might not otherwise have had. We will continue to be involved in further stages of the NICE process to advocate for patients to have long-term access to what would be a significant advancement in the treatment of ALL.
To find out more about CAR-T, including what is involved at each stage of the treatment, you can read our more in-depth booklet on CAR-T therapy here.
If you have any questions about this announcement, want to know more about what treatments are available for you, how treatment decisions are made or have any other question related to treatment, please contact us via firstname.lastname@example.org, or WhatsApp 07500068065 (services available Monday to Friday 9am – 5pm).