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Last updated: 10th July 2023
Today, Leukaemia Care welcomes the decision by the Scottish Medicines Consortium (SMC) to approve oral azacitidine for use on the NHS in Scotland. This is a treatment for acute myeloid leukaemia (AML) in adults. Oral azacitidine will be used specifically as a maintenance treatment after induction therapy.
What’s the news and how was Leukaemia Care involved?
Following the approval of oral azacitidine in England in 2022, we are pleased to announce that the Scottish Medicines Consortium (SMC) have also now decided to approve this treatment for use in Scotland. The brand name for oral azacitidine is Onureg and it is produced by Celgene.
Similar to our involvement in the NICE (National Institute for Health and Care Excellence) process, Leukaemia Care also submitted a written response to the SMC representing the views of AML patients. A submission is created by drawing on experiences and data, and advocating for equity of access for Scottish patients.
We are very pleased that patients in Scotland now have equal access to oral azacitidine as those in England, Wales, and Northern Ireland.
What is maintenance treatment?
Oral azacitidine has now been approved for use on the NHS in Scotland as maintenance treatment after induction therapy for adult AML patients. Induction therapy includes treatments such as chemotherapy or radiotherapy and aims to put a patient’s AML into remission. This is done by using high doses of treatment over a short period of time.
Maintenance treatment is a long-term and usually less intensive treatment. It is given after the leukaemia is in remission, to prolong the remission and hopefully prevent a relapse from happening. This is what oral azacitidine will now be given to patients for. A transplant might be an option if a clinician is worried about relapse, but transplants are not suitable for everyone.
This is important as before this there was no maintenance treatment for the vast majority of AML patients in Scotland. A maintenance treatment called midostaurin is only available for a minority of patients whose condition was caused by a rare genetic mutation.
Which patients can access this treatment?
Oral azacitidine is recommended by the SMC as an option for maintenance treatment for adults with AML who:
– Are in complete remission, or complete remission with incomplete blood count recovery, after induction therapy with or without consolidation treatment, AND
– Who cannot have or do not want a haematopoietic stem cell transplant
We argued that those who cannot have a transplant may not be eligible for reasons out of their control and for non-clinical reasons, such as financial circumstances or challenges with getting a transplant donor. Therefore, there needs to be an alternative for people, whatever their reason for not having a transplant.
Why is the approval of this treatment important for patients?
Relapse rates in AML are common, at around 50%. Relapse is a scary and daunting prospect for many as it leads to lower chances of overall survival. It also has a negative impact on patients’ quality of life, e.g., their mental health.
Therefore, a treatment which could prevent relapse after initial remission would be in patients’ best interests. The approval of oral azacitidine fills this gap and provides a much needed treatment option, especially for those who do not want or can’t have a stem cell transplant. These are people who would be left with few other options if they were to relapse, so preventing relapse if possible is preferred.
We are very pleased that patients in Scotland now have equal access to oral azacitidine as those in England, Wales, and Northern Ireland.
What are the other benefits of the treatment to patients?
A patient with personal experience of having AML and a different, subcutaneous injection, version of azacitidine stated that:
“I personally benefited from azacitidine in sub cutaneous form and thought it was important for the committee to hear from a patient’s perspective the numerous advantages of having it available in an oral form.”
The SMC has approved azacitidine in oral form, which means it is taken as a tablet. Survey data has shown us that oral treatments are favoured by many patients for their convenience, as they reduce travel time to and from hospital, reduce financial burden of travel and spending time in hospital and allow patients to spend more time with friends and family. Oral tablets are also often preferred as they are less invasive.
If you have any further questions about the drug announcement, our Advocacy and Policy and Public Affairs team is here to help. Email them at advocacy@leukaemiacare.org.uk.