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Table 3 Percentage of EPARs where the methods are applicable

From: Applicability and added value of novel methods to improve drug development in rare diseases

METHOD Applicability in percentage of EPARs
Step Static step 1 (no adjustments) Dynamic step 2 (adjustments)
Statistic Percentage of EPARs (n/26) Percentage of clusters (n/6) Percentage of EPARs (n/26) Percentage of clusters (n/6)
Extrapolation 35% [9/26] 83% 46% [12/26] 100%
Heterogeneity estimators 4% [1/26] 17% 4% [1/26] 17%
Prior distributions for variance parameters in sparse-event meta-analysis 4% [1/26] 17% 4% [1/26] 17%
Delayed-start randomisation 13% [3/26] 50% 12% [3/26] 50%
Sample size reassessment and hypothesis testing in adaptive survival trials 35% [9/26] 83% 58% [15/26] 100%
Multi-arm group sequential designs with a simultaneous stopping rule 23% [6/26] 67% 58% [15/26] 100%
Sequential designs for small samples 31% [8/26] 67% 66% [17/26] 100%
Bayesian sample size re-estimation using power priors 12% [3/26] 33% 50% [13/26] 100%
Dynamic borrowing through empirical power priors that control type I error 15% [4/26] 33% 50% [13/26] 100%
Fallback tests for co-primary endpoints 15% [4/26] 50% 50% [13/26] 100%
Optimal exact tests for multiple binary endpoints 4% [1/26] 17% 31% [8/26] 83%
Simultaneous inference for multiple marginal GEE models 19% [5/26] 50% 23% [6/26] 67%
Goal Attainment Scaling 31% [8/26] 67% 31% [8/26] 67%
  1. EPAR European Public Assessment Report