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Title: | Predicting Treatment Effect from Surrogate Endpoints and Historical Trials: An Extrapolation Involving Probabilities of a Binary Outcome or Survival to a Specific Time | Authors: | Baker, Stuart G. Sargent, Daniel J. BUYSE, Marc BURZYKOWSKI, Tomasz |
Issue Date: | 2012 | Publisher: | WILEY-BLACKWELL | Source: | BIOMETRICS, 68 (1), p. 248-257 | Abstract: | Using multiple historical trials with surrogate and true endpoints, we consider various models to predict the effect of treatment on a true endpoint in a target trial in which only a surrogate endpoint is observed. This predicted result is computed using (1) a prediction model (mixture, linear, or principal stratification) estimated from historical trials and the surrogate endpoint of the target trial and (2) a random extrapolation error estimated from successively leaving out each trial among the historical trials. The method applies to either binary outcomes or survival to a particular time that is computed from censored survival data. We compute a 95% confidence interval for the predicted result and validate its coverage using simulation. To summarize the additional uncertainty from using a predicted instead of true result for the estimated treatment effect, we compute its multiplier of standard error. Software is available for download. | Notes: | [Baker, Stuart G.] NCI, Bethesda, MD 20892 USA. [Sargent, Daniel J.] Mayo Clin, Rochester, MN 55905 USA. [Buyse, Marc] IDDI, B-1340 Louvain, Belgium. [Burzykowski, Tomasz] Hasselt Univ, B-3590 Diepenbeek, Belgium. sb16i@nih.gov | Keywords: | Principal stratification; Randomized trials; Reproducibility;Biology; Mathematical & Computational Biology; Statistics & Probability; Principal Stratification; Randomized trials; Reproducibility | Document URI: | http://hdl.handle.net/1942/13715 | ISSN: | 0006-341X | e-ISSN: | 1541-0420 | DOI: | 10.1111/j.1541-0420.2011.01646.x | ISI #: | 000301924400035 | Category: | A1 | Type: | Journal Contribution | Validations: | ecoom 2013 |
Appears in Collections: | Research publications |
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