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Table 3 Comparison of treatment effects of pivotal trial using surrogate markers with matched postapproval trials using surrogate markers: primary and sensitivity analyses

From: Comparison of treatment effect sizes from pivotal and postapproval trials of novel therapeutics approved by the FDA based on surrogate markers of disease: a meta-epidemiological study

  Summary difference between standardized mean differences or relative odds ratios (95% CI)
Method of analyses
 At least two matching criteria (n = 43)
  Reported non-continuous endpoints 1.50 (1.01 to 2.23)
  Reported continuous endpoints 0.01 (−0.15 to 0.16)
  All standardized as odds ratios (secondary) 1.12 (0.88 to 1.42)
 At least three matching criteria (n = 39)
  Reported non-continuous endpoints 1.45 (0.99 to 2.14)a
  Reported continuous endpoints 0.05 (−0.08 to 0.19)
  All standardized as odds ratios (secondary) 1.17 (0.95 to 1.45)
 All four matching criteria (n = 33)
  Reported non-continuous endpoints 1.21 (0.89 to 1.64)
  Reported continuous endpoints 0.06 (−0.10 to 0.21)
  All standardized as odds ratios (secondary) 1.13 (0.90 to 1.42)
  1. A positive difference between standardized mean differences or ratio of odds ratios >1.0 indicates a larger benefit of treatment compared to the comparator in pivotal trials compared to postapproval trials. Pooled using DerSimonian and Laird random-effects meta-analyses
  2. CI confidence interval
  3. a95% CI of 1.00 to 2.10 with a between-study correlation of 0.5