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. 2018 Oct;24(10):10.18553/jmcp.2018.24.10.1002. doi: 10.18553/jmcp.2018.24.10.1002

Table 5.

Results of the Sensitivity and Scenario Analyses

Sensitivity Analysis: 3-Year Cumulative Budget
Costs, $a Current Market New Market Difference Change, %
  Base case 33,632,943 29,052,813 -4,580,130 -13.6
  Patient weight increased 20% (86.4 kg) 40,161,965 33,069,366 -7,092,599 -17.7
  Patient dose increased 20% (56.9 U/kg) 40,161,965 33,069,366 -7,092,599 -17.7
  Adherence at 100% (ERT = 26 infusion per year; SRT = MPR 100%) 36,545,241 31,542,542 -5,002,699 -13.7
Scenario Analysis: 3-Year Cumulative Results
Scenario Incremental Results, $ (%) (Reduction Compared to the Current Market) PMPM, $
  Scenario 1: Mature shift to eliglustat: 50%b -5,162,841 (-15.4) -0.029
  Scenario 1: Mature shift to eliglustat: 100%b -7,700,456 (-22.9) -0.043
  Scenario 2: Conservative markup: ERT cost markup is 20% at all sites of care. -3,228,312 (-10.7) -0.018
  Scenario 3: Site of care management: The share of patients receiving ERT infusions in a hospital setting for the current and new market is reduced to 10% from 32% in the base case. -2,490,957 (-8.9) -0.014

aAll costs are expressed in 2017 U.S. dollars.

bPercentage of patients receiving ERT infusions in the hospital outpatient setting were shifted to eliglustat in the new market.

ERT = enzyme replacement therapy; MPR = medication possession ratio; PMPM = per member per month; SRT = substrate reduction therapy; U = unit.