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

351 Pages·2016·6.52 MB·English

by  Jianchang Lin

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
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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