Skip to main content
. 2021 Feb 28;9(1):1889841. doi: 10.1080/20016689.2021.1889841

Table 2.

Inputs used in model

Variable Source
Health state transitions: proportion of patients sitting or walking. onasemnogene abeparvovec:
AVXS-101-CL-101 [NCT02122952][8,28]
nusinersen:
ENDEAR [NCT02193074][17] and SHINE [NCT02594124][16]
BSC:
No sitting/walking patients.
Health state transitions: Probability of death or permanent ventilation (D state only) Permanent Ventilation (E state) –
onasemnogene abeparvovec/nusinersen/BSC:
Based on a retrospective chart review of SMA patients with tracheotomy or non-invasive ventilation, projected using parametric estimation[13]
Not sitting (D state) –
onasemnogene abeparvovec:
Short term model: AVXS-101-CL-101 [NCT02122952][8]
Long term: Projected overall and event-free survival using curve fitted to ENDEAR/SHINE (sham control arm) [NCT02193074, NCT02594124][16,17]
nusinersen:
Short term model: ENDEAR [NCT02193074][17] and SHINE [NCT02594124][16]
Long term: Projected overall and event-free survival using curve fitted to ENDEAR/SHINE (sham control arm) [NCT02193074, NCT02594124][16,17]
BSC:
ENDEAR (sham control arm)[17]
Sitting (C state) –
onasemnogene abeparvovec/nusinersen:
Parametric curve fitted to overall survival curve for SMA type 2[11]
Walking (B state) –
onasemnogene abeparvovec/nusinersen:
Assume general population mortality in US[29]
Utility Scores Equivalent to weightings used in the ICER analysis[14], derived from multiple sources
Permanent Ventilation: 0.19
Not sitting: 0.19 (BSC) 0.29 (Treatment arms)
Sitting: 0.60 (BSC) 0.65 (Treatment arms)
Walking: US general population utility
Cost inputs Price of onasemnogene abeparvovec: $2.125 M one-time dose
All other costs were equal to costs used in the ICER analysis[14]
Caregiver disutility (Modified societal perspective) Equivalent to values used in the ICER analysis[14], derived from The Lewin Group report[30]:
Permanent Ventilation: −0.394
Not sitting: −0.2955
Sitting: −0.197
Walking: 0
Lost Household income (Modified societal perspective) Equivalent to values used in the ICER analysis (estimate 1)[14], derived from The Lewin Group report[30]:
SMA early onset: predicted loss $19,833
SMA other: predicted loss $14,800
Non-Medical costs
(Modified societal perspective)
Equivalent to values used in the ICER analysis (estimate 1)[14], derived from The Lewin Group report[30]:
Permanent ventilation, Not sitting, Sitting: $964 per month
Patient productivity gains Equivalent to values used in the ICER analysis (estimate 1)[14], derived from The Lewin Group report[30]:
Sitting, Walking: potential monthly income $4,540, estimated from age 30–65 years.