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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

  1. LY life-year, QALY quality-adjusted life year
  2. *Costs and cost-effectiveness ratios are rounded to the nearest $1000