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Webpage last updated on: 14th September 2023
What is the news?
Leukaemia Care is pleased to share the decision by the National Institute for Health and Care Excellence (NICE) to approve ruxolitinib treatment for use on the NHS in patients with polycythemia vera (PV) in England. This treatment is only approved for adults with PV who cannot tolerate hydroxycarbamide (also called hydroxyurea), or when their condition has become resistant to it.
How was Leukaemia Care involved in the decision to approve the treatment?
For this treatment appraisal, Leukaemia Care collaborated with the charity MPN Voice to provide evidence on patient experience and outline the case for this treatment to be approved. Leukaemia Care and MPN Voice submitted a written response to NICE representing the views and experiences of PV patients by drawing on sources including survey data, conversations with patients and information from patient forums.
Charlotte Crowley, Leukaemia Care’s Policy and Evidence Manager, also spoke to the NICE committee on behalf of PV patients, further emphasising the impact that the approval of this drug could have on eligible patients.
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.
Why has the NICE committee made this decision and why is it important for patients?
Standard treatment to control blood cell counts in the treatment of PV is hydroxycarbamide or interferon alfa. Ruxolitinib would be used for people who cannot tolerate hydroxycarbamide or when their condition has become resistant to it.
Studies considered by NICE suggest that ruxolitinib is more effective than standard treatment at controlling blood cell counts and reducing spleen size.
NICE commented that the data on whether ruxolitinib increases how long people live is uncertain/not conclusive. However, despite this, the treatment still met NICE’s cost effectiveness criteria, and it provides a very important new option for those who cannot tolerate or become resistant to hydroxycarbamide.
How does the treatment work?
Ruxolitinib is a targeted cancer drug, which prevents cancer cells from dividing and growing. Ruxolitinib works by blocking the JAK2 gene that is important in the production of blood cells. Ruxolitinib is an oral tablet treatment that is taken for a continuous duration, i.e. for as long as it continues to work.
If you have any questions about your own treatment plan, please speak with your doctor in the first instance.
If you have any questions about this announcement, please contact advocacy@leukaemiacare.org.uk.
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, or send your question to WhatsApp on 07500 068 065 (services available Monday to Friday 9am – 5pm).