Page last updated on: 21st April 2023
What is the news?
Leukaemia Care is delighted to share the decision by the National Institute for Health and Care Excellence (NICE) to approve the combination treatment ibrutinib with venetoclax for use on the NHS in previously untreated adults with CLL in England.
This positive decision was reached by NICE in a shorter timeframe than usual with leukaemia drugs. Leukaemia Care’s CEO, Zack Pemberton-Whiteley comments “this approval could be one the most significant advances in CLL treatment in the last decade, avoiding chemotherapy treatment for many newly diagnosed patients”. This article aims to explain why.
How was Leukaemia Care involved in the decision to approve the treatment?
Leukaemia Care submitted a written response to NICE representing the views of CLL patients by drawing on experiences and data, and advocating for untreated patients to have access to this new combination therapy.
Nick York is Leukaemia Care’s Healthcare Liaison Officer and a CLL patient. He spoke to the NICE committee to advocate for patients collectively and explain why having this treatment available would be important to patients. His views were considered by the committee, alongside Leukaemia Care’s evidence, as part of the decision-making process.
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 and information on the cost of delivering the new drug. NICE also takes into account comments from patient and clinical experts on the impact the drug would have on patients. A committee of people at NICE then makes a recommendation based on the evidence they have.
Which patients are able to access this treatment?
The treatment can be used as a first treatment for adults with chronic lymphocytic leukaemia (CLL) who have not received any treatment before. This includes being an alternative treatment for both those who are considered fit and unfit for intensive chemotherapy treatments such as FCR (fludarabine plus cyclophosphamide and rituximab), as well as both those with and without a del17p/TP53 mutation.
Why has the NICE committee made this decision?
Compared with one of the existing treatments used as a first treatment for CLL patients, obinutuzumab plus chlorambucil, there is clinical evidence that patients take longer to get worse and live longer when treated with ibrutinib with venetoclax instead. There was also an indirect comparison made between ibrutinib with venetoclax and acalabrutinib, FCR and ibrutinib alone, which also suggests that CLL takes longer to get worse when treated with ibrutinib with venetoclax.
The committee considered inputs from clinical and patient experts, i.e. Nick, who suggested there were currently limited treatment options for both the high-risk and non-high-risk groups. This created a greater need to approve a new treatment in this setting for patients. Also, ibrutinib with venetoclax’s fixed treatment duration, oral tablet form, and better toxicity profile than current treatments made it a highly valued treatment option.
The committee considered ibrutinib with venetoclax to represent a cost-effective use of NHS resources, and therefore recommended it for routine commissioning on the NHS for anyone with untreated chronic lymphocytic leukaemia.
Why is the approval of this treatment important for patients?
CLL is a chronic long-term condition most common in those over 75. Effective treatments such as this one can help ensure that this disease no longer prevents people from living a normal lifespan. Previously, such effective treatments have largely been reserved for the second or third treatment option.
Current treatments used first in CLL patients can be unsuitable, ineffective or intolerable for some people, and can have long-term side effects. Ibrutinib with venetoclax has been shown to be effective and have an improved side-effect profile. Having this treatment approved in the first line therefore marks a massive step forward in the treatment of CLL.
Both ibrutinib and venetoclax are taken orally and are a fixed duration treatment, meaning that patients would only need to take the treatment for a limited period of time rather than continuously. Both of these factors are preferable to most patients with CLL, according to our Living with Leukemia survey.
Nick York said “Today’s announcement marks a significant moment towards improving the quality of life and survival of people living with CLL. These combination therapies do not carry the risks associated with traditional chemotherapy used as first treatment.”
This treatment offers many more patients of all types, the fittest, high risk and frailer groups, a chance of achieving a remission and living life without CLL symptoms or the need for long-term daily treatments and issues these may involve.
Extending the time before the next treatment means additional options remain available for the future, as there is less chance of developing treatment resistance and more time to return to living a more normal life again.”
How does the treatment work?
Ibrutinib (Imbruvica, Janssen-Cilag) is a small-molecule inhibitor of a protein called Bruton’s tyrosine kinase (BTK), which stops B-cell (lymphocyte) proliferation and promotes cell death.
Venetoclax targets a protein called B-cell lymphoma-2 (BCL-2), which allows cancer cells to stay alive. It does so by acting as a selective blocker of this protein.
If you have any questions about this announcement, please contact email@example.com.
If you 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 our helpline 08088 010 444, whatsapp on 07500068065 (services available Monday to Friday 9am – 5pm).