Unit information prior for adaptive information borrowing from multiple historical datasets

In clinical trials, there often exist multiple historical studies for the same or related treatment investigated in the current trial. Incorporating historical data in the analysis of the current study is of great importance, as it can help to gain more information, improve efficiency, and provide a more comprehensive evaluation of treatment. Enlightened by the unit information prior (UIP) concept in the reference Bayesian test, we propose a new informative prior called UIP from an information perspective that can adaptively borrow information from multiple historical datasets. We consider both binary and continuous data and also extend the new UIP to linear regression settings. Extensive simulation studies demonstrate that our method is comparable to other commonly used informative priors, while the interpretation of UIP is intuitive and its implementation is relatively easy. One distinctive feature of UIP is that its construction only requires summary statistics commonly reported in the literature rather than the patient-level data. By applying our UIP to phase III clinical trials for investigating the efficacy of memantine in Alzheimer's disease, we illustrate its ability to adaptively borrow information from multiple historical datasets. The Python codes for simulation studies and the real data application are available at https://github.com/JINhuaqing/UIP.

[1]  Eran Segal,et al.  Overlapping codes within protein-coding sequences. , 2010, Genome research.

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

[3]  S. Gauthier,et al.  Synthesis and Comparison of the Meta-Analyses Evaluating the Efficacy of Memantine in Moderate to Severe Stages of Alzheimer’s Disease , 2013, CNS Drugs.

[4]  Steven G Potkin,et al.  Memantine treatment in mild to moderate Alzheimer disease: a 24-week randomized, controlled trial. , 2006, The American journal of geriatric psychiatry : official journal of the American Association for Geriatric Psychiatry.

[5]  L. Wasserman,et al.  A Reference Bayesian Test for Nested Hypotheses and its Relationship to the Schwarz Criterion , 1995 .

[6]  C. Rudin,et al.  Nivolumab versus Docetaxel in Advanced Nonsquamous Non-Small-Cell Lung Cancer. , 2015, The New England journal of medicine.

[7]  J. Olin,et al.  Memantine treatment in patients with mild to moderate Alzheimer's disease already receiving a cholinesterase inhibitor: a randomized, double-blind, placebo-controlled trial. , 2008, Current Alzheimer research.

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

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

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

[11]  Emmanuel Lesaffre,et al.  Modified power prior with multiple historical trials for binary endpoints , 2018, Statistics in medicine.

[12]  Tim Friede,et al.  Dynamically borrowing strength from another study through shrinkage estimation , 2018, Statistical methods in medical research.

[13]  Se-Ran Jun,et al.  Alignment-free genome comparison with feature frequency profiles (FFP) and optimal resolutions , 2009, Proceedings of the National Academy of Sciences.

[14]  L. Crinò,et al.  Nivolumab versus Docetaxel in Advanced Squamous-Cell Non-Small-Cell Lung Cancer. , 2015, The New England journal of medicine.

[15]  Ying Cheng,et al.  Nivolumab Versus Docetaxel in a Predominantly Chinese Patient Population With Previously Treated Advanced NSCLC: CheckMate 078 Randomized Phase III Clinical Trial , 2019, Journal of thoracic oncology : official publication of the International Association for the Study of Lung Cancer.

[16]  Rachelle Doody,et al.  Memantine in moderate-to-severe Alzheimer's disease. , 2003, The New England journal of medicine.

[17]  Keying Ye,et al.  Evaluating water quality using power priors to incorporate historical information , 2006 .

[18]  J. Kornhuber,et al.  Memantine in moderate-to-severe Alzheimer's disease. , 2003, The New England journal of medicine.

[19]  Yoshua Bengio,et al.  Generative Adversarial Nets , 2014, NIPS.

[20]  M. Mega,et al.  The Neuropsychiatric Inventory , 1994, Neurology.

[21]  M. Mega,et al.  The spectrum of behavioral responses to cholinesterase inhibitor therapy in Alzheimer disease. , 1999, Archives of neurology.

[22]  S. Bakchine,et al.  Memantine treatment in patients with mild to moderate Alzheimer's disease: results of a randomised, double-blind, placebo-controlled 6-month study. , 2007, Journal of Alzheimer's disease : JAD.

[23]  Bradley P Carlin,et al.  Hierarchical Commensurate and Power Prior Models for Adaptive Incorporation of Historical Information in Clinical Trials , 2011, Biometrics.

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

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

[26]  Tomoyuki Kakizume,et al.  Applying Meta-Analytic-Predictive Priors with the R Bayesian Evidence Synthesis Tools , 2019, J. Stat. Softw..

[27]  C. Lai,et al.  The merits and problems of Neuropsychiatric Inventory as an assessment tool in people with dementia and other neurological disorders , 2014, Clinical interventions in aging.

[28]  Roy W Jones,et al.  Memantine in Moderate to Severe Alzheimer’s Disease: a Meta-Analysis of Randomised Clinical Trials , 2007, Dementia and Geriatric Cognitive Disorders.

[29]  Tim Friede,et al.  Bounds for the weight of external data in shrinkage estimation , 2020, Biometrical journal. Biometrische Zeitschrift.

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

[31]  Pierre N Tariot,et al.  Memantine treatment in patients with moderate to severe Alzheimer disease already receiving donepezil: a randomized controlled trial. , 2004, JAMA.

[32]  J. Ibrahim,et al.  Power prior distributions for regression models , 2000 .

[33]  P. Tariot,et al.  A 24-week Randomized, Controlled Trial of Memantine in Patients With Moderate-to-severe Alzheimer Disease , 2007, Alzheimer disease and associated disorders.

[34]  Carlos Barrios,et al.  Atezolizumab versus docetaxel in patients with previously treated non-small-cell lung cancer (OAK): a phase 3, open-label, multicentre randomised controlled trial , 2017, The Lancet.