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Table 3 Overview of the characteristics of the six economic evaluations by population, intervention, and results

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

References Population Intervention/comparator/(including drug prices) Results (Drugs costs are never included in the analysis)
CADTH
[34]
SMA1, 2, 3 Nusinersen versus standard of care
Nusinersen:
First year:
578,916 US $
Per year after 289,458 US $
For SMA1:
Nusinersen led to greater QALYs (gain of 4.80), life years (gain of 4.79), and cost (increase of $3.1 million) for an incremental cost per QALY gained of $665,570
For SMA2:
Nusinersen led to greater QALYs (gain of 3.67), life years (gain of 2.18), and cost (increase of $7.6 million) for an incremental cost per QALY gained of $2.1 million
For SMA3:
Nusinersen led to greater QALYs (gain of 1.56), no difference in life years (gain of 2.18), and an increase in cost ($4.5 million) for an incremental cost per QALY gained of $2.9 million
For all three SMA types:
The probability that nusinersen was cost effective assuming that the threshold value for a QALY was $300,000 was 0%
ICER
[36]
SMA1, 2, 3 and pre-symptomatic Nusinersen versus standard of care and Onasemnogene abeparvovec versus standard of care
Nusinersen:
Per year after the first:
396,443 US $
Onasemnogene abeparvovec: 2 million US$
ICER of nusinersen is $709,000 per QALY gained from a healthcare-sector perspective and $687,000 from a modified societal perspective, far exceeding usual cost-effectiveness thresholds
For Onasemnogene abeparvovec (at a placeholder price of $2 million) the ICER from a healthcare-sector perspective in patients with symptomatic SMA1 is $243,000 per QALY gained
Jalali [32] SMA1 and pre-symptomatic Standard of care compared to Nusinersen with and without newborn screening
Nusinersen:
First year:
776,000 US $
Per year after: 388,000 US $
Compared with no screening and no treatment, the ICER for nusinersen with screening was $330 558 per event-free life year saved
The ICER for nusinersen treatment without screening was $508,481 per event-free life year saved
For nusinersen with screening to be cost-effective at a willingness-to-pay (WTP) threshold of $50,000 per event-free LY saved, the price would need to be $23,361 per dose, less than one-fifth its current price of $125,000
Preliminary data from the NURTURE trial indicated an 85.7% improvement in expected LYs saved compared with our base results
-In probabilistic sensitivity analysis, nusinersen and screening was a preferred strategy 93% of the time at a $500,000 WTP threshold
Malone et al. [33] SMA1 patients with 2 copies of SMN2 Onasemnogene abeparvovec was compared to nusinersen. Nusinersen:
First year:
776,000 US $
Per year after: 388,000 US $
Onasemnogene abeparvovec: between 2,5 and 5 million of US$
Expected survival (undiscounted) over a lifetime predicted by the model was 37.20 life years for Onasemnogene abeparvovec and 9.68 life years for nusinersen (discounted QALYs, 15.65 and 5.29, respectively)
Using a potential Onasemnogene abeparvovec price range ($2.5–5.0 M/treatment), the average lifetime cost/patient was $4.2–6.6 M for Onasemnogene abeparvovec and $6.3 M for nusinersen
The ICER range was (-$203,072) to $31,379 per QALY gained for Onasemnogene abeparvovec versus nusinersen, indicating that Onasemnogene abeparvovec was cost-effective when priced at ≤ $5 M per treatment
National Center for Pharmaco-economics [37] SMA1, 2, 3, 4 Nusinersen versus standard of care
First year:
681,421 US$
Per year after:
341,105 US$
Nusinersen cannot be considered cost-effective at current price
A tenfold reduction in the price of nusinersen for the treatment of infantile SMA is required to produce an ICER approaching the € 45,000/QALY threshold
For later-onset SMA, nusinersen is less cost-effective and a 20-fold price reduction results in an ICER just under € 100,000/QALY
The 5-year net budget impact for Ireland is estimated at €37.88 million
Zuluaga-Sanchez et al
[35]
SMA1, 2 Nusinersen versus standard of care
First year:
516,896 US$
Per year after:
258,448 US$
For SMA1:
Nusinersen resulted in 3.86 patient incremental QALYs
Nusinersen resulted in 0.02 caregiver incremental QALYs
Nusinersen incremental cost was $280,000 over standard of care
ICER for nusinersen (including caregiver QALYs) of $544,000 per QALY gained
For SMA2:
Nusinersen resulted in 9.54 patient incremental QALYs
Nusinersen resulted in 2.39 caregiver incremental QALYs
Nusinersen incremental cost of $3.6 million over standard of care
ICER for nusinersen (including caregiver QALYs) of $308,000 per QALY gained