a different method to calculate vial use.
It accounted for the distribution of body
surface area in the population, and also
calculated a mean body surface area of
1.83m 2 by applying the gender weighting
from Study 301 to the data from the Sacco
study. The ERG considered that hospital
length of stay was overestimated in the
model compared with that seen in Study
301. Therefore in its preferred analysis,
it reduced the number of hospital days in
the consolidation period. The ERG used
a lower cost of stem cell transplant than
the company, based on using the costs of
transplants from sibling donors instead
of from unrelated adult donors. It also
increased the follow-up cost to reflect
a 2 year follow-up, instead of 6 months.
The clinical experts stated that, although
sibling donors had been more common, it
was now more likely that unrelated adult
stem cells would be used for transplants.
The committee noted that these changes
to costs and resource use in the model
had little effect on the cost-effectiveness
results. It concluded that it was reasonable
to use the ERG’s method of calculating
vial use, for the length of hospital stay in
the model to match that in the trial and
to include transplant follow-up costs for
two years. However, it agreed that stem
cells for transplant would likely come from
unrelated matched donors.
Cost-effectiveness results
The most plausible incremental cost-
effectiveness ratio compared with standard
cytarabine and daunorubicin is lower than
£50,000 per quality-adjusted life year
(QALY) gained.
The company updated its economic model
after consultation to include the committee’s
preferred assumptions, specifically:
• correcting some errors identified by
the ERG
• basing outcomes after transplant only
on overall survival
• adjusting mortality rates after
transplant
• using some alternative utility values
• using a different method to calculate
vial use
• reducing the number of hospital days
in the consolidation period.
The company used cure models after
stem cell transplant and also increased the
discount in the commercial arrangement.
This resulted in an incremental cost-
effectiveness ratio (ICER) for liposomal
cytarabine–daunorubicin of £45,055 per
QALY gained. When the company used
a 25% cure fraction for the standard
cytarabine and daunorubicin group, the
ICER increased to £48,127 per QALY gained.
When the ERG reproduced the analyses
to include the confidential commercial
arrangement discount for azacitidine
(included in the model as a subsequent
treatment), both ICERs were below £50,000
per QALY gained. The committee concluded
that the most plausible ICER was lower
than £50,000 per QALY gained.
Innovation
The benefits of liposomal cytarabine–
daunorubicin are captured in the cost-
effectiveness analysis.
The company considered that liposomal
cytarabine–daunorubicin was an innovative
treatment because of its formulation. The
drug accumulates in the bone marrow and
is released inside the cells. The company
also highlighted that infusion time is
reduced and that people can have it as
outpatients. It also noted that liposomal
cytarabine–daunorubicin is the only
new treatment in recent years to show
a survival benefit for people with high-
risk acute myeloid leukaemia. Patient
and professional groups highlighted
that liposomal cytarabine–daunorubicin
is the first example of this type of
technology in acute myeloid leukaemia,
and that it is more targeted than standard
chemotherapy. The committee concluded
that liposomal cytarabine–daunorubicin
would be beneficial for patients but that
it had not been presented with evidence
of any additional benefits that were not
captured in the measurement of QALYs.
hospitalpharmacyeurope.com | 2019 | 9