Table 7 Scenario Analyses for nusinersen versus BSC
From: Cost effectiveness of nusinersen for patients with infantile-onset spinal muscular atrophy in US
| Cost per QALY* | Cost per LY* | |
|---|---|---|
| Base-case results | $1,112,000 | $590,000 |
| Scenario #1: Assuming no utility benefits for interim milestones | $1,303,000 | $590,000 |
| Scenario #2: Assuming lower monthly health state costs of $10,434 and $13,135 for “not sitting” and “permanent ventilation” health states, respectively | $990,000 | $525,000 |
| Scenario #3: Assuming lower utility of 0.5 for “sitting” health state | $1,265,000 | $590,000 |
| Scenario #4: Assuming lower survival (mean survival of 15.6 years) for “sitting” health state | $1,253,000 | $624,000 |
| Scenario #5: Assuming lower utility of 0.5 and lower survival (mean 15.6 years) for “sitting” health state | $1,407,000 | $624,000 |
| Scenario #6a: Assuming 10% in “sitting” health state lose milestone at end of short-term model | $1,143,000 | $593,000 |
| Scenario #6b: Assuming 20% in “Sitting” Health State Lose Milestone at End of Short-Term Model | $1,178,000 | $597,000 |
| Scenario #6c: Assuming 30% in “sitting” health state lose milestone at end of short-term model | $1,218,000 | $601,000 |
| Scenario 7: Scenario assuming 30% in “sitting” health state lose milestone at end of short-term model, lower utilities and survival for “sitting” health state | $1,509,000 | $630,000 |
| Scenario #8: Using a 10 year time horizon | $1,460,000 | $700,000 |
| Scenario #9: Using 1.5% discount rate for both costs and QALYs | $1,052,000 | $566,000 |