Estimating optimal treatment regimes via subgroup identification in randomized control trials and observational studies

Statistics in Medicine
Haoda FuDouglas E Faries

Abstract

With new treatments and novel technology available, personalized medicine has become an important piece in the new era of medical product development. Traditional statistics methods for personalized medicine and subgroup identification primarily focus on single treatment or two arm randomized control trials. Motivated by the recent development of outcome weighted learning framework, we propose an alternative algorithm to search treatment assignments which has a connection with subgroup identification problems. Our method focuses on applications from clinical trials to generate easy to interpret results. This framework is able to handle two or more than two treatments from both randomized control trials and observational studies. We implement our algorithm in C++ and connect it with R. Its performance is evaluated by simulations, and we apply our method to a dataset from a diabetes study. Copyright © 2016 John Wiley & Sons, Ltd.

References

Dec 14, 2001·AAPS PharmSci·L MancinelliW Sadée
Mar 25, 2005·Diabetic Medicine : a Journal of the British Diabetic Association·B H CharbonnelUNKNOWN QUARTET Study Group
Apr 21, 2006·The New England Journal of Medicine·Stephen W Lagakos
Aug 10, 2007·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Melinda L TelliAlice E Guardino
Nov 23, 2007·The New England Journal of Medicine·Rui WangJeffrey M Drazen
Aug 27, 2009·JAMA : the Journal of the American Medical Association·Alan R ShuldinerPaul A Gurbel
Jul 30, 2010·Clinical Trials : Journal of the Society for Clinical Trials·Stephen J RubergYanping Wang
Nov 3, 2010·Current Genomics·Rachel E EllsworthDarrell L Ellsworth
Jun 15, 2011·Annals of Statistics·Min Qian, Susan A Murphy
Aug 5, 2011·Statistics in Medicine·Jared C FosterStephen J Ruberg
May 4, 2012·Biometrics·Baqun ZhangMarie Davidian
Feb 5, 2013·Frontiers of Medicine·Yingqi Zhao, Donglin Zeng
May 1, 2013·Journal of the American Statistical Association·Yingqi ZhaoMichael R Kosorok
Aug 21, 2013·International Journal of Methods in Psychiatric Research·Douglas E FariesRobert L Obenchain
Sep 24, 2013·Journal of the American Statistical Association·Lihui ZhaoL J Wei
Dec 21, 2013·Diabetes Care·UNKNOWN American Diabetes Association
Oct 28, 2014·Statistics in Medicine·Gong ChenViswanath Devanarayan

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Citations

Jun 21, 2017·Statistical Methods in Medical Research·Demissie AlemayehuMarianthi Markatou
Dec 20, 2017·Statistical Methods in Medical Research·Brent R LoganPurushottam W Laud
Nov 18, 2017·Statistical Methods in Medical Research·Chathura SiriwardhanaK B Kulasekera
Feb 23, 2019·Journal of Biopharmaceutical Statistics·Aniek SiesIven Van Mechelen
Jun 28, 2019·Statistics in Medicine·Oleg SysoevKatarina Ekholm Selling
Nov 20, 2019·Biometrics·Crystal T NguyenMichael R Kosorok
May 20, 2017·The International Journal of Biostatistics·Aniek Sies, Iven Van Mechelen
Nov 28, 2017·Journal of Biopharmaceutical Statistics·Ilya LipkovichBohdana Ratitch
Mar 20, 2019·Statistics in Medicine·Jingli WangWeng Kee Wong
Oct 23, 2019·Statistics in Medicine·Chathura SiriwardhanaSomnath Datta
Mar 14, 2018·Biometrics·Jingxiang ChenYufeng Liu
Nov 7, 2019·Journal of Biopharmaceutical Statistics·Chathura SiriwardhanaK B Kulasekera
Jan 1, 2018·Journal of Computational and Graphical Statistics : a Joint Publication of American Statistical Association, Institute of Mathematical Statistics, Interface Foundation of North America·Kevin DoubledayJin Zhou
Oct 13, 2020·Journal of the American Statistical Association·Peng WuYuanjia Wang
Jul 31, 2021·Therapeutic Innovation & Regulatory Science·Pierre BunoufIlya Lipkovich

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