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Table 3 Parameter estimation of the final model of ALSFRS-R

From: Accelerating drug development for amyotrophic lateral sclerosis: construction and application of a disease course model using historical placebo group data

 

Estimate

RSE (%)

Bootstrap (999)*

Median (95% CI)

Emax (Score)

26.2

7.0

27.3(23.4–46.4)

ET50 (month)

17.5

4.4

17.7(16.6–38.8)

γ

1.2

5.8

1.18(1.01–1.29)

η (Emax), %

25.8

18.0

25.7(12.8–31.9)

η (γ), %

12.2

20.0

11.9(7.7–17.3)

εadd

4.00

14.9

3.88 (2.61–5.10)

  1. Emax the maximum effect value, ET50 time to reach half of the maximum effect value, γ the shape parameter, η is the inter-study variability of model parameter, εadd additive residual error, RSE relative standard error, CI confidience interval
  2. *999 means that the model succeeded 999 times out of 1000 bootstrap attempt
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172