What’s the news?
As of today NICE has announced the approval of venetoclax on its own (monotherapy) for the treatment of adults with CLL on the NHS. This follows a campaign by Leukaemia Care jointly with CLL Support and Lymphoma Action. This is an important treatment for patients who cannot have or or have relapsed from existing treatment options.
This appraisal has been through what’s called the Cancer Drugs Fund (CDF). This means that it was temporarily approved at first, because NICE didn’t feel they had sufficient data at the time of the original appraisal. The Cancer Drugs Fund provides funding of treatments to allow the collection of more real-world evidence. Following the review of real-world data about venetoclax collected since 2017, the treatment has been approved for use permanently.
How was Leukaemia Care involved?
Leukaemia Care, working with two other charities CLL Support and Lymphoma Action, were asked to share why this treatment was important to patients. This was quite challenging; the number of treatments now available for patients with CLL is large and many have long remission times after their first and second treatments. However, as more patients with CLL are successfully treated and live longer, there remains a need for more treatments for people to have once they have had other treatments. There is also a need for venetoclax on its own for people who cannot have other treatments.
As well as providing evidence in a written submission, Nick York (CLL patient as well as Healthcare Liaison Officer at Leukaemia Care) and another patient attended the committee meeting at NICE. Both attendees gave extremely passionate speeches about the worry of CLL returning, since it is incurable. Many patients think about the long term and like to know what the next treatment will be before they have had their current treatment. Having an increasing number of potential options approved is reassuring for many CLL patients.
Which group of patients can access this new treatment?
Anyone who has started treatment with venetoclax on its own whilst it was available in the CDF will still have access; your treatment should not be affected by this decision.
This treatment is for patients who have already tried the other treatment options that are available to them, or who are unsuitable for the other options. This includes:
- Patients with genetic abnormalities (a 17p deletion or TP53 mutation) whose disease has progressed after treatment with a BCR inhibitor, e.g. ibrutinib
- Patients with a 17p deletion or TP53 mutation who are unsuitable for BCR inhibitors. For example, those with existing heart problems might not be suitable for ibrutinib
- Patients without a 17p deletion or TP53 mutation who have already had chemotherapy and a BCR inhibitor but whose disease has progressed.
Why has NICE made this recommendation?
The key benefit of venetoclax is to prolong the life of CLL patients once they’ve run out of other options. NICE were not satisfied that they had enough data from clinical trials to be sure of how effective venetoclax was, on its own, at the time of the original appraisal in 2017. While in the CDF, NICE were able to collect further evidence of venetoclax’s effectiveness using data from the Systemic Anti-Cancer Therapy (SACT) dataset. This was the key source of data that led to this permanent decision to approve venetoclax.
People with CLL for whom venetoclax monotherapy would be an option would usually only have limited alternatives, since it is approved for those who have tried most other options. Venetoclax therefore meets NICE’s criteria as an end of life treatment, increasing the price that the NHS is willing to pay for the treatment.
Are there any other benefits of venetoclax?
Another advantage is that the majority of patients receiving venetoclax are likely to be outpatients, even during the dose escalation stage. For patients this means that while you might have to go into hospital more regularly during dose escalation, you do not have to stay in, which enables you to spend more time at home.
How does this drug work?
Venetoclax works by attaching to a protein called BCL-2. Large amounts of this protein are present in CLL cancer cells and it helps the cells survive for longer in the body and can make them resistant to other medicines. By attaching to and blocking BCL-2, venetoclax causes the death of cancer cells thereby slowing down disease progression. Venetoclax is also effective against other types of leukaemia, including acute myeloid leukaemia.
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Published: Friday 6th May 2022