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Table 2 Overview of economic evaluation studies of SMA

From: Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

References Country Perspective Time horizon Method Outcomes used Industry funding
CADTH
[34]
Canada
2018
Healthcare payer SMA1:
25 years
SMA2:
50 years
SMA3:
80 years
Three Markov models:
for SMA1,
for SMA2,
for SMA3
Life years
QALY
No, commissioned by health authorities
ICER
[36]
England
2018
Healthcare payer and societal perspective Two scenarios:
5 years
10 years
Three Markov models:
for SMA1,
for SMA2 and SMA3,
for pre-symptomatic SMA
QALY No, commissioned by health authorities
Jalali [32] USA
2020
Societal perspective 30 months Four Markov models:
for untreated patients SMA1,
for treated SMA1 identified by symptoms,
for untreated patients identified by newborn screening,
for nusinersen-treated patients identified by newborn screening
Life Years
QALY
No
Malone et al. [33] USA
2019
Healthcare payer Lifetime
horizon
Markov model comparing nusinersen and Onasemnogene abeparvovec for SMA1 QALY Avexis
National Center for Pharmaco-economics [37] Ireland
2017
Societal perspective Lifetime
horizon
Two separate Markov models:
for early-onset SMA,
for later-onset SMA
QALY No, commissioned by health authorities
Zuluaga-Sanchez et al. [35] Sweden
2018
Societal
and payer perspective
SMA1:
40 years
SMA2:
80 years
Markov model: incremental cost QALY gained and overall survival. Two models:
for early-onset SMA,
for later-onset SMA
QALY Biogen