ICSA Book Series in Statistics Series Editors: Jiahua Chen · Ding-Geng (Din) Chen Jianchang Lin Bushi Wang Xiaowen Hu Kun Chen Ray Liu Editors Statistical Applications from Clinical Trials and Personalized Medicine to Finance and Business Analytics Selected Papers from the 2015 ICSA/ Graybill Applied Statistics Symposium, Colorado State University, Fort Collins ICSA Book Series in Statistics SeriesEditors JiahuaChen DepartmentofStatistics UniversityofBritishColumbia Vancouver Canada Ding-Geng(Din)Chen UniversityofNorthCarolina ChapelHill,NC,USA Moreinformationaboutthisseriesathttp://www.springer.com/series/13402 Jianchang Lin • Bushi Wang (cid:129) Xiaowen Hu Kun Chen (cid:129) Ray Liu Editors Statistical Applications from Clinical Trials and Personalized Medicine to Finance and Business Analytics Selected Papers from the 2015 ICSA/Graybill Applied Statistics Symposium, Colorado State University, Fort Collins 123 Editors JianchangLin BushiWang GlobalStatistics Biostatistics TakedaPharmaceuticals BoehringerIngelheimPharmaceuticals Cambridge,MA,USA Ridgefield,CT,USA XiaowenHu KunChen ColoradoStateUniversity UniversityofConnecticut FortCollins,CO,USA Storrs,CT,USA RayLiu GlobalStatistics TakedaPharmaceuticals Cambridge,MA,USA ISSN2199-0980 ISSN2199-0999 (electronic) ICSABookSeriesinStatistics ISBN978-3-319-42567-2 ISBN978-3-319-42568-9 (eBook) DOI10.1007/978-3-319-42568-9 LibraryofCongressControlNumber:2016954077 ©SpringerInternationalPublishingSwitzerland2016 Thisworkissubjecttocopyright.AllrightsarereservedbythePublisher,whetherthewholeorpartof thematerialisconcerned,specificallytherightsoftranslation,reprinting,reuseofillustrations,recitation, broadcasting,reproductiononmicrofilmsorinanyotherphysicalway,andtransmissionorinformation storageandretrieval,electronicadaptation,computersoftware,orbysimilarordissimilarmethodology nowknownorhereafterdeveloped. Theuseofgeneraldescriptivenames,registerednames,trademarks,servicemarks,etc.inthispublication doesnotimply,evenintheabsenceofaspecificstatement,thatsuchnamesareexemptfromtherelevant protectivelawsandregulationsandthereforefreeforgeneraluse. Thepublisher,theauthorsandtheeditorsaresafetoassumethattheadviceandinformationinthisbook arebelievedtobetrueandaccurateatthedateofpublication.Neitherthepublishernortheauthorsor theeditorsgiveawarranty,expressorimplied,withrespecttothematerialcontainedhereinorforany errorsoromissionsthatmayhavebeenmade. Printedonacid-freepaper ThisSpringerimprintispublishedbySpringerNature TheregisteredcompanyisSpringerInternationalPublishingAG Theregisteredcompanyaddressis:Gewerbestrasse11,6330Cham,Switzerland Preface The 2015Joint 24th InternationalChinese Statistical Association (ICSA) Applied Statistics Symposium and 13th Graybill Conference was successfully held from June 14 to June 17,2015,in FortCollins, Colorado,USA. The conferencecovers a variety of exciting and state-of-the-art statistical application topics (over 400 presentations) from the bio-pharmaceutical applications, e.g., clinical trials and personalized medicine, to non-bio-pharmaceutical applications, e.g., finance and businessanalytics,withattendeesfromindustry,government,andacademia. The 24paperswere selectedfromthe presentationsin the annualmeetingwith abroadrangeoftopicssothatreadersofthisbookcouldnotonlyenjoythetopics closetotheirownresearchareasbutalsofromotherdifferentareas.Allpapershave gonethroughthepeerreviewprocessandpartscoveredinthebookinclude: (cid:129) biomarkerandpersonalizedmedicine (cid:129) Bayesianmethodsandapplications (cid:129) doserangingstudiesinclinicaltrials (cid:129) innovativeclinicaltrialdesignsandanalysis (cid:129) clinicalandsafetymonitoringinclinicaltrials (cid:129) statisticalapplicationsinnonclinicalandpreclinicaldrugdevelopment (cid:129) statisticallearningmethodsandapplicationswithlarge-scaledata (cid:129) statisticalapplicationsinbusinessandfinance We are very grateful to the authors who contributed their papers to these proceedings and carefully prepared their manuscripts within a tight timeline. These proceedingswould also notbe possible without the successful symposium, which gave us the opportunity to share, learn, and choose so many high-quality papers.OurdeepgratitudegoestotheleadershipofNaiteeTingandtheexecutive organizing committee, the program committee, and many other volunteers of the v vi Preface 24th ICSA Applied Statistics Symposium and 13th Graybill Conference. We also thank Michael Penn of Springer for the assistance through the entire process of completingthebook. Cambridge,MA,USA JianchangLin Ridgefield,CT,USA BushiWang FortCollins,CO,USA XiaowenHu Storrs,CT,USA KunChen Cambridge,MA,USA RayLiu Contents PartI BiomarkerandPersonalizedMedicine OptimalBiomarker-GuidedDesignforTargetedTherapy withImperfectlyMeasuredBiomarkers....................................... 3 YongZangandYingYuan Statistical Considerations for Evaluating Prognostic Biomarkers:ChoosingOptimalThreshold ................................... 15 ZhengZhang Accuracy of Meta-Analysis Using Different Levels ofDiagnosticAccuracyMeasures.............................................. 21 YanyanSong,YingLu,andLuTian PartII BayesianMethodsandApplications BayesianFrailtyModelsforMulti-StateSurvivalData ..................... 37 MáriodeCastro,Ming-HuiChen,andYuanyeZhang BayesianIntegrationofInVitroBiomarkertoAnalysisofIn VivoSafetyAssessment.......................................................... 49 Ming-DauhWangandAlanY.Chiang A Phase II Trial Design with Bayesian Adaptive Covariate-AdjustedRandomization ........................................... 61 JianchangLin,Li-AnLin,andSerapSankoh PartIII DoseRangingStudiesinClinicalTrials SampleSizeAllocationinaDose-RangingTrialCombinedwithPoC..... 77 QiqiDengandNaiteeTing PersonalizedEffectiveDoseSelectioninDoseRangingStudies ............ 91 XiwenMa,WeiZheng,andYuefengLu vii viii Contents PartIV InnovativeClinicalTrialDesignsandAnalysis EvaluationofConsistencyRequirementsinMulti-Regional ClinicalTrialswithDifferentEndpoints....................................... 107 ZhaoyangTeng,JianchangLin,andBinZhang A Statistical Decision Framework Applicable toMultipopulationTailoringTrials............................................ 121 BrianA.Millen AssessingBenefitandConsistencyofTreatmentEffectUnder aDiscreteRandomEffectsModelinMultiregionalClinicalTrials ........ 127 Jung-TzuLiu,Chi-TianChen,K.K.GordonLan, Chyng-ShyanTzeng,Chin-FuHsiao,andHsiao-HuiTsou Design and Analysis of Multiregional Clinical Trials inEvaluationofMedicalDevices:ATwo-ComponentBayesian ApproachforTargetedDecisionMaking...................................... 137 YunlingXu,NelsonLu,andYingYang SemiparametricAnalysisofInterval-CensoredSurvivalData withMedianRegressionModel................................................. 149 JianchangLin,DebajyotiSinha,StuartLipsitz,andAdrianoPolpo ExplainedVariationforCorrelatedSurvivalDataUnderthe ProportionalHazardsMixed-EffectsModel.................................. 165 GordonHonerkamp-SmithandRonghuiXu SomeMisconceptionsontheUseofCompositeEndpoints.................. 179 Jianjun(David)LiandJinXu PartV ClinicalandSafetyMonitoringinClinicalTrials AStatisticalModelforRisk-BasedMonitoringofClinicalTrials.......... 191 GregoryJ.Hather BlindedSafetySignalMonitoringfortheFDAINDReporting FinalRule......................................................................... 201 GregBallandPatrickM.Schnell PartVI StatisticalApplicationsinNonclinicalandPreclinical DrugDevelopment DesignandStatisticalAnalysisofMultidrugCombinations inPreclinicalStudiesandPhaseIClinicalTrials............................. 215 MingT.Tan,Hong-BinFang,HengzhenHuang,andYangYang StatisticalMethodsforAnalyticalComparability............................ 235 LeslieSidor Contents ix StatisticalApplicationsforBiosimilarProductDevelopment............... 259 RichardMontes,BryanBernat,andCatherineSrebalus-Barnes PartVII StatisticalLearningMethodsandApplicationswith Large-ScaleData AStatisticalMethodforChange-SetAnalysis................................ 281 Pei-ShengLin,JunZhu,Shu-FuKuo,andKatherineCurtis AnAlarmSystemforFluOutbreaksUsingGoogleFluTrendData....... 293 GregoryVaughan,RobertAseltine,SyHanChiou,andJunYan Identifying Gene-EnvironmentInteractionswitha Least RelativeErrorApproach........................................................ 305 YangguangZang,Yinjun Zhao,QingzhaoZhang,Hao Chai, SanguoZhang,andShuanggeMa PartiallySupervisedSparseFactorRegressionForMulti-Class Classification...................................................................... 323 ChongliangLuo,DipakDey,andKunChen PartVIII StatisticalApplicationsinBusinessandFinance ABivariateRandom-EffectsCopulaModelforLength ofStayandCost.................................................................. 339 XiaoqinTang,ZhehuiLuo,andJosephC.Gardiner Index............................................................................... 353 Contributors RobertAseltine DivisionofBehavioralScienceandCommunityHealth,Univer- sityofConnecticutHealthCenter,Farmington,CT,USA Center for Public Health and Health Policy, University of Connecticut Health Center,Farmington,CT,USA GregBall BiostatisticsandResearchDecisionSciences,MerckResearchLabora- tories,Rahway,NJ,USA BryanBernat Hospira,aPfizercompany,LakeForest,IL,USA Mário de Castro Instituto de Ciências Matemáticas e de Computação,Universi- dadedeSãoPaulo,SãoCarlos,Brazil HaoChai DepartmentofBiostatistics,YaleUniversity,NewHaven,CT,USA Ming-Hui Chen Departmentof Statistics, University of Connecticut, Storrs, CT, USA KunChen DepartmentofStatistics,UniversityofConnecticut,Storrs,CT,USA Center for Public Health and Health Policy, University of Connecticut Health Center,Farmington,CT,USA Chi-TianChen InstituteofPopulationHealthSciences,NationalHealthResearch Institutes,Zhunan,MiaoliCounty,Taiwan Alan Y.Chiang GlobalStatisticalSciences,LillyCorporateCenter,EliLillyand Company,Indianapolis,IN,USA Sy HanChiou DepartmentofBiostatistics, HarvardT.H.ChanSchoolofPublic Health,Boston,MA,USA KatherineCurtis DepartmentofCommunityandEnvironmentalSociology,Uni- versityofWisconsin-Madison,Madison,WI,USA xi