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. 2019 Apr;25(4):10.18553/jmcp.2019.25.4.490. doi: 10.18553/jmcp.2019.25.4.490
Input Value Probabilistic Deterministic
Distribution Normal/Log-Normal Beta/Dirichlet % of Mean Variation (±)
SE as Proportion of Mean, % Sample Size
Population size in each EDSS state Varies by EDSS (not shown) Log-normal 10 20
Discontinuation probabilities 0.1 Beta 1,000 20
Natural history EDSS transitions RRMS-RRMS Transition matrix (not shown) Dirichlet 900
SPMS-SPMS Transition matrix (not shown) Dirichlet 925
Relative mortality due to MS By EDSS state Varies by EDSS (not shown) Normal 10 20
Base utility EDSS 0 0.8752 Gamma 10 20
Utility adjustment (RRMS) By EDSS state Varies by EDSS (not shown) 1-Gamma 10 20
Utility adjustment (SPMS) -0.0437 Gamma 10 20
LOE price reduction Fingolimod Varies by year (Table 1) Beta 1,000 20
IM IFNβ-1a Beta 1,000 20
LOE proportion of population switching Fingolimod Varies by year (Table 1) Beta 1,000 20
IM IFNβ-1a Beta 1,000 20
Input Probabilistic Deterministic
Distribution LCL UCL
Age of cohort 29 Log-normal 23 35
Relative risk of disability progression versus placebo Fingolimod 0.68 Log-normal 0.51 0.90 Lower and upper estimate based on LCL and UCL, respectively
IM IFNβ-1a 0.79 Log-normal 0.63 1.00
Relative rate of relapse versus placebo Fingolimod 0.46 Log-normal 0.39 0.55
IM IFNβ-1a 0.83 Log-normal 0.74 0.94
Natural history of relapse By EDSS state (RRMS)
0 0.71 Normal 0.57 0.85
1 0.73 Normal 0.58 0.88
2 0.68 Normal 0.54 0.82
3 0.72 Normal 0.58 0.86
4 0.71 Normal 0.57 0.85
5 0.59 Normal 0.47 0.71
6 0.49 Normal 0.39 0.59
7 0.51 Normal 0.41 0.61
8 0.51 Normal 0.41 0.61
9 0.51 Normal 0.41 0.61
By EDSS state (SPMS)
1 0 Normal 0 0.1
2 0.47 Normal 0.38 0.56
3 0.88 Normal 0.7 1.06
4 0.55 Normal 0.44 0.66
5 0.52 Normal 0.42 0.62
6 0.45 Normal 0.36 0.54
7 0.34 Normal 0.27 0.41
8 0.34 Normal 0.27 0.41
9 0.34 Normal 0.27 0.41
Natural history EDSS transitions (RRMS-SPMS) 0 0 Beta 0 0.003
1 0.003 Beta 0.002 0.004
2 0.032 Beta 0.026 0.038
3 0.117 Beta 0.094 0.14
4 0.21 Beta 0.168 0.252
5 0.299 Beta 0.239 0.359
6 0.237 Beta 0.19 0.284
7 0.254 Beta 0.203 0.305
8 0.153 Beta 0.122 0.184
9 1 Beta 0.9 1

Source: Institute for Clinical and Economic Review. Confidence limits based on those reported in the ICER disease-modifying therapies for relapsing and primary-progressive multiple sclerosis: effectiveness and value.7

EDSS = Expanded Disability Status Scale; ICER = Institute for Clinical and Economic Review; IM IFNβ-1a = intramuscular interferon beta-1a; LCL = lower confidence limit; LOE = loss of exclusivity; MS = multiple sclerosis; RRMS = relapsing-remitting multiple sclerosis; SE = standard error; SPMS = secondary progressive multiple sclerosis; UCL = upper confidence limit.