Leukaemia Care successfully campaigns for new leukaemia treatment on behalf of AML patients

Leukaemia Care was consulted at all possible stages of the process, which includes a submission with evidence of patient experience and why a new treatment is needed. The draft decision by NICE was not to approve this treatment for use on the NHS. Leukaemia Care responded to this draft decision.

Among our reasons for pushing for this treatment to be approved is that it is an alternative to a stem cell transplant (see next section). A transplant is currently often used as maintenance treatment for AML patients, to keep them in remission.

However, for some patients a stem cell transplant is unsuitable; for example, not everyone can support themselves financially during the time needed to recover from a transplant.

Also, people from minority ethnic backgrounds or who are of mixed heritage can find it hard to get a transplant donor.

We also pushed NICE to recognise that azacitidine as maintenance treatment is needed because without maintenance treatment, people often do not live for long if they relapse. Therefore, we should be willing to fund this treatment for people who will otherwise be at the end of their life. Following an additional committee meeting we were pleased that NICE decided to approve the treatment.

For this appraisal, Leukaemia Care also enlisted the help of a patient expert, someone who has had AML and who could speak on behalf of AML patients about the benefits this treatment could have. The patient spoke at several of the committee meetings, and explains that he “was able to speak for patients who have experienced AML and explain in human terms what an extension of life or improvement in quality of life can mean for someone with a potentially fatal illness”.

Oral azacitidine has now been approved for use on the NHS in England 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. This is important as before this, there was no maintenance treatment for AML approved. A transplant might be an option if a clinician is worried about relapse, but transplants are not suitable for everyone.

Oral azacitidine is recommended by NICE 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.

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.

The patient expert on this appraisal spoke to NICE from personal experience of having AML and a different, subcutaneous injection, version of azacitidine.

He comments, “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.”

NICE 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.