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Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease.
[fabry disease]
To
evaluate
the
cost-effectiveness
of
enzyme
replacement
therapy
(
ERT
)
compared
to
standard
medical
care
without
ERT
in
the
Dutch
cohort
of
patients
with
type
1
Gaucher
disease
(
GD
I
)
.
Cost-effectiveness
analysis
was
performed
using
a
life-time
state
-transition
model
of
the
disease
's
natural
course
.
Transition
probabilities
,
effectiveness
data
and
costs
were
derived
from
retrospective
data
and
prospective
follow-up
of
the
Dutch
study
cohort
.
The
tertiary
referral
center
for
Gaucher
disease
in
the
Netherlands
.
T
he
Dutch
cohort
of
patients
with
GD
I
.
ERT
versus
standard
medical
care
without
ERT
in
symptomatic
patients
.
Years
free
of
end
organ
damage
(
YFEOD
)
(
splenectomy
,
bone
complication
,
malignancy
,
multiple
complications
)
,
quality
adjusted
life
years
(
QALY
)
,
and
costs
.
Over
an
85
year
lifetime
,
an
untreated
GD
I
patient
will
generate
48
.
9
YFEOD
and
55
.
86
QALYs
.
Starting
ERT
in
a
symptomatic
patient
increases
the
YFEOD
by
12
.
8
years
,
while
the
number
of
QALYs
gained
increases
by
6
.
27
.
The
average
yearly
ERT
medication
costs
range
between
€
124
,
000
and
€
258
,
000
per
patient
.
The
lifetime
costs
of
ERT
starting
in
the
symptomatic
stage
are
€
5
,
716
,
473
against
€
171
,
780
without
ERT
,
a
difference
of
€
5
,
544
,
693
.
Consequently
,
the
extra
costs
per
additional
YFEOD
or
per
additional
QALY
are
€
434
,
416
and
€
884
,
994
respectively
.
After
discounting
effects
by
1
.
5
%
and
costs
by
4
%
and
under
a
reasonable
scenario
of
ERT
unit
cost
reduction
by
25
%
,
these
incremental
cost-effectiveness
ratios
could
decrease
to
€
149
,
857
and
€
324
,
812
respectively
.
ERT
is
a
highly
potential
drug
for
GD
I
with
substantial
health
gains
.
The
conservatively
estimated
incremental
cost-effectiveness
ratios
are
substantially
lower
than
for
Pompe
and
Fabry
disease
.
We
suggest
that
the
high
effectiveness
has
contributed
importantly
to
acceptance
of
reimbursement
of
ERT
for
GD
I
.
The
present
study
may
further
support
discussions
on
acceptable
price
limits
for
ultra-orphan
products
.
Diseases
Validation
Diseases presenting
"conservatively estimated incremental cost-effectiveness ratios"
symptom
fabry disease
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