a scenario analysis that included a 25%
cure fraction in the standard cytarabine
and daunorubicin group. This reduced
the cost effectiveness of liposomal
cytarabine–daunorubicin compared with
standard cytarabine and daunorubicin.
The committee concluded that the model
was appropriate for decision making.
However, it agreed that there was still
uncertainty in the difference in overall
survival between the two treatment
groups after stem cell transplant and in
the cure fraction assumed for the standard
cytarabine and daunorubicin group.
Event-free survival analysis for patients
who had a complete response is unreliable
because of small patient numbers.
The company and ERG agreed that
the analysis used to model event-free
survival after transplant for patients who
had a complete response in the model
was uncertain because of small patient
numbers. The ERG also suggested that
it lacked face validity. This was because
there was little difference between the
two treatment groups, unlike for overall
survival after transplant. Therefore the ERG
excluded these data from the model and
used the overall survival analysis to inform
a 2-state model. In this model, patients
were either in remission or dead. This
change increased the cost effectiveness of
liposomal cytarabine–daunorubicin.
In response to consultation, the company
adopted the ERG’s approach to modelling
event-free survival. The committee would
have preferred the whole population to
be modelled together (whether or not
they had a stem cell transplant) but
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