Quantification of prior impact in terms of effective current sample size

Bayesian methods allow borrowing of historical information through prior distributions. The concept of prior effective sample size (prior ESS) facilitates quantification and communication of such prior information by equating it to a sample size. Prior information can arise from historical observations, thus the traditional approach identifies the ESS with such historical sample size. However, this measure is independent from newly observed data, and thus would not capture an actual "loss of information" induced by the prior in case of prior-data conflict. We build on recent work to relate prior impact to a number of (virtual) samples from the current data model and introduce the effective current sample size (ECSS) of a prior, tailored to the application in Bayesian clinical trial designs. Special emphasis is put on robust mixture, power and commensurate priors. We apply the approach to an adaptive design in which the number of recruited patients is adjusted depending on the effective sample size at an interim analysis. We argue that the ECSS is the appropriate measure in this case, as the aim is to save current (as opposed to historical) patients from recruitment. Furthermore, the ECSS can help overcoming lack of consensus in the ESS assessment of mixture priors and can, more broadly, provide further insights into the impact of priors. An R package accompanies the paper. This article is protected by copyright. All rights reserved.

[1]  S J Pocock,et al.  The combination of randomized and historical controls in clinical trials. , 1976, Journal of chronic diseases.

[2]  Bradley P Carlin,et al.  Commensurate Priors for Incorporating Historical Information in Clinical Trials Using General and Generalized Linear Models. , 2012, Bayesian analysis.

[3]  Andrew Gelman,et al.  The Prior Can Often Only Be Understood in the Context of the Likelihood , 2017, Entropy.

[4]  Satoshi Morita,et al.  Prior Effective Sample Size in Conditionally Independent Hierarchical Models. , 2012, Bayesian analysis.

[5]  P. Müller,et al.  Determining the Effective Sample Size of a Parametric Prior , 2008, Biometrics.

[6]  Nicolas Bousquet,et al.  Diagnostics of prior-data agreement in applied Bayesian analysis , 2008 .

[7]  P. Horby,et al.  Performance of different clinical trial designs to evaluate treatments during an epidemic , 2018, PloS one.

[8]  L. M. Berliner,et al.  Robust Bayes and Empirical Bayes Analysis with $_\epsilon$-Contaminated Priors , 1986 .

[9]  Nan Chen,et al.  Web-based statistical tools for the analysis and design of clinical trials that incorporate historical controls , 2018, Comput. Stat. Data Anal..

[10]  George E. P. Box,et al.  Sampling and Bayes' inference in scientific modelling and robustness , 1980 .

[11]  Bradley P Carlin,et al.  Statistical modeling for Bayesian extrapolation of adult clinical trial information in pediatric drug evaluation , 2017, Pharmaceutical statistics.

[12]  Fang Chen,et al.  Use of historical control data for assessing treatment effects in clinical trials , 2014, Pharmaceutical statistics.

[13]  Brian P Hobbs,et al.  Adaptive adjustment of the randomization ratio using historical control data , 2013, Clinical trials.

[14]  Annette Kopp-Schneider,et al.  Power gains by using external information in clinical trials are typically not possible when requiring strict type I error control , 2019, Biometrical journal. Biometrische Zeitschrift.

[15]  Anthony O'Hagan,et al.  Robust meta‐analytic‐predictive priors in clinical trials with historical control information , 2014, Biometrics.

[16]  Michael Evans,et al.  Checking for prior-data conflict , 2006 .

[17]  Joseph G Ibrahim,et al.  The power prior: theory and applications , 2015, Statistics in medicine.

[18]  Michael Branson,et al.  A note on the power prior , 2009, Statistics in medicine.

[19]  Fulvio De Santis,et al.  Using historical data for Bayesian sample size determination , 2007 .

[20]  Leonhard Held,et al.  Power priors based on multiple historical studies for binary outcomes , 2017, Biometrical journal. Biometrische Zeitschrift.

[21]  L. Wasserman,et al.  The Selection of Prior Distributions by Formal Rules , 1996 .

[22]  Leonhard Held,et al.  Adaptive power priors with empirical Bayes for clinical trials , 2017, Pharmaceutical statistics.

[23]  J. Bernardo,et al.  THE FORMAL DEFINITION OF REFERENCE PRIORS , 2009, 0904.0156.

[24]  D. Spiegelhalter,et al.  Summarizing historical information on controls in clinical trials , 2010, Clinical trials.