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 ($2.5ā5.0Ā M/treatment), the average lifetime cost/patient was $4.2ā6.6Ā M for Onasemnogene abeparvovec and $6.3Ā 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 atāā¤ā$5Ā M per treatment |
National Center for Pharmaco-economics [37] | SMA1, 2, 3, 4 | Nusinersen versus standard of care First year: 681,421 US$ 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 ā¬ 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 ā¬ 100,000/QALY The 5-year net budget impact for Ireland is estimated at ā¬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 |