Skip to main content

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