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.