Publication: Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts

We have now published a journal article summarising our work as the Evidence Review Group (ERG) for the NICE Single Technology Appraisal TA399: Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts.

The journal article has been published in the journal PharmacoEconomics, and is currently available as an “online first” article, meaning it is accepted and published, but has not yet been assigned to a particular issue of the journal.

The recommended citation for the article is currently:

Tikhonova IA, Hoyle MW, Snowsill TM, Cooper C, Varley-Campbell JL, Rudin CE, Mujica Mota RE. Azacitidine for Treating Acute Myeloid Leukaemia with More Than 30 % Bone Marrow Blasts: An Evidence Review Group Perspective of a National Institute for Health and Care Excellence Single Technology Appraisal. PharmacoEconomics 2016. DOI: 10.1007/s40273-016-0453-5

What is acute myeloid leukaemia?

Acute myeloid leukaemia (AML) is a cancer of the bone marrow, which leads to the excessive production of immature white blood cells, called myeloblasts. These myeloblasts can crowd out other types of cell, leading to anaemia, poor blood clotting, and reduced ability to fight infections.

Although younger patients stand a chance of overcoming AML, older patients (aged 65 and over) have a poor prognosis, with less than 1 in 18 surviving longer than five years after diagnosis. The treatment options available for such patients are often limited, as they frequently have frailty or other conditions that make treatments for younger patients (such as intensive chemotherapy and stem cell transplantation) unsuitable.

Further reading: Wikipedia article on acute myeloid leukaemia

What is azacitidine?

Azacitidine, which is marketed by Celgene as Vidaza®, is a hypomethylating agent developed over 40 years ago, which has been granted European marketing authorisation for the treatment of adult patients aged 65 years or older with acute myeloid leukaemia with more than 30% bone marrow blasts.

A course of treatment with azacitidine (without the confidentially disclosed discount available to the NHS) is estimated to cost around £4,500, and on average patients received 8.8 cycles of treatment.

What does the NICE guidance say?

The NICE guidance says that azacitidine is not recommended as an option for adults aged 65 and over with acute myeloid leukaemia with >30% bone marrow blasts. This means that these patients will not normally be able to access azacitidine as a treatment option.

The Committee concluded that the evidence for the effectiveness of azacitidine was not compelling, and left a lot of uncertainty. The Committee also concluded that at their best estimate, azacitidine would not be a cost-effective use of limited NHS resources.

What happens next?

The date for an appeal to be registered has passed. The guidance is next due to be reviewed in July 2019. Celgene has European marketing exclusivity for azacitidine until December 2018, but lost US exclusivity in 2011 so generic development has already started.