PharmacoEconomics

Azacitidine for Treating Acute Myeloid Leukaemia with More Than 30% Bone Marrow Blasts: a NICE Single Technology Appraisal

Irina Tikhonova,1,3 Martin Hoyle,1 Tristan Snowsill,1 Chris Cooper,1 Jo Varley-Campbell,1 Claudius Rudin,2 Ruben Mujica Mota1

1 Peninsula Technology Assessment Group (PenTAG), Exeter, UK

2 Royal Devon and Exeter NHS Foundation Trust, Exeter, UK

3

Table 7 Assessment of End-of-Life criteria

Criterion / Data available (Celgene) / The ERG’s comment
The treatment is indicated for patients with a short life expectancy, normally less than 24 months / Median OS reported in the literature ranges between 1.5 months (aged >65 years) and 2 months (aged >55 years) / Median OS in AZA-AML-001 trial is 6.5 months without azacitidine treatment. Restricted mean survival at 30 months is estimated to be 10.55 months (Appendix 4, the ERG’s report [25]).
Azacitidine is also indicated for intermediate-2 and high-risk myelodysplastic syndromes, CMML with 10–29% marrow blasts without myeloproliferative disorder and AML with 20–30% blasts and multi-lineage dysplasia. The results of the AZA-001 trial in this population suggest median OS of 15.0 months without azacitidine treatment [14].
There is sufficient evidence to indicate that the treatment offers an extension to life, normally of at least an additional 3 months, compared with current NHS treatment / Median OS based on the primary endpoint was 10.4 months in the azacitidine group and 6.5 months in the CCR group, providing an OS benefit of 3.8 months with azacitidine. As reported in the Celgene’s submission, various pre-defined analyses demonstrated that treatment with azacitidine provided a statistically significant survival benefit versus CCR / Extension to life should be assessed considering differences in mean overall survival in addition to median OS. The ERG’s analyses based on restricted mean survival at 30 months suggest an extension to life of 1.8–2.5 months (depending on how treatment switching is handled – see Appendix 4, the ERG’s report [25]).
The estimated improvement in restricted mean OS is greatest for patients pre-selected to BSC, although comparisons to individual CCR are subject to uncertainty.
The treatment is licensed or otherwise indicated for small patient populations / The estimated total population for all licensed indications in England is 3,354, consisting of 1,026 covered by the proposed new indication and 2,328 for all existing indications. / No comment.

AML acute myeloid leukaemia, BSC best supportive care, CCR conventional care regimens, CMML chronic myelomonocytic leukaemia, ERG Evidence Review Group, OS overall survival, NHS National Health Service

Source: The ERG’s report [22]