Encyclopedia of biopharmaceutical statistics PDF
Preview Encyclopedia of biopharmaceutical statistics
[raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] Encyclopedia of Biopharmaceutical Statistics [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] Encyclopedia of Biopharmaceutical Statistics Third Edition Revised and Expanded Volume One A–L Edited by Shein-Chung Chow Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina, U.S.A. [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] Firstpublishedin2000byInformaHealthcare,TelephoneHouse,69-77PaulStreet,LondonEC2A4LQ,UK. Thiseditionpublishedin2010byInformaHealthcare,TelephoneHouse,69-77PaulStreet,LondonEC2A4LQ,UK. SimultaneouslypublishedintheUSAbyInformaHealthcare,52VanderbiltAvenue,7thFloor,NewYork,NY10017,USA. InformaHealthcareisatradingdivisionofInformaUKLtd.RegisteredOffice:37–41MortimerStreet,LondonW1T3JH,UK. RegisteredinEnglandandWalesnumber1072954. #2010InformaHealthcare,exceptasotherwiseindicated NoclaimtooriginalU.S.Governmentworks Reprintedmaterialisquotedwithpermission.Althougheveryefforthasbeenmadetoensurethatallownersofcopyrightmaterial have been acknowledged in this publication, we would be glad to acknowledge in subsequent reprints or editions any omissions broughttoourattention. Allrightsreserved.Nopartofthispublicationmaybereproduced,storedinaretrievalsystem,ortransmitted,inanyformorbyany means,electronic,mechanical,photocopying,recording,orotherwise,unlesswiththepriorwrittenpermissionofthepublisherorin accordancewiththeprovisionsoftheCopyright,DesignsandPatentsAct1988orunderthetermsofanylicencepermittinglimited copying issued by the Copyright Licensing Agency, 90 Tottenham Court Road, London W1P 0LP, UK, or the Copyright Clearance Center,Inc.,222RosewoodDrive,Danvers,MA01923,USA(http://www.copyright.com/ortelephone978-750-8400). Productorcorporatenamesmaybetrademarksorregisteredtrademarks,andareusedonlyforidentificationandexplanationwithout intenttoinfringe. This book contains information from reputable sources and although reasonable efforts have been made to publish accurate information, the publisher makes no warranties (either express or implied) as to the accuracy or fitness for a particular purpose of theinformationoradvicecontainedherein.Thepublisherwishestomakeitclearthatanyviewsoropinionsexpressedinthisbookby individual authors or contributors are their personal views and opinions and do not necessarily reflect the views/opinions of the publisher. Any information or guidance contained in this book is intended for use solely by medical professionals strictly as a supplement to the medical professional’s own judgement, knowledge of the patient’s medical history, relevant manufacturer’s instructionsandtheappropriatebestpracticeguidelines.Becauseoftherapidadvancesinmedicalscience,anyinformationoradvice ondosages,procedures,ordiagnosesshouldbeindependentlyverified.Thisbookdoesnotindicatewhetheraparticulartreatmentis appropriateorsuitableforaparticularindividual.Ultimatelyitisthesoleresponsibilityofthemedicalprofessionaltomakehisorher own professional judgements, so as appropriately to advise and treat patients. Save for death or personal injury caused by the publisher’snegligenceandtothefullestextentotherwisepermittedbylaw,neitherthepublishernoranypersonengagedoremployed bythepublishershallberesponsibleorliableforanyloss,injuryordamagecausedtoanypersonorpropertyarisinginanywayfrom theuseofthisbook. ACIPrecordforthisbookisavailablefromtheBritishLibrary. ISBN-13:9781439822456 Ordersmaybesentto:InformaHealthcare,SheepenPlace,Colchester,EssexCO33LP,UK Telephone:+44(0)2070175540 Email:[email protected] Website:http://informahealthcarebooks.com/ Forcorporatesalespleasecontact:[email protected] Forforeignrightspleasecontact:[email protected] Forreprintpermissionspleasecontact:[email protected] TypesetbyMPSLimited,AMacmillanCompany PrintedandboundintheUK [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] Contents Contributors.... xi BayesianMethodsin Meta-Analysis 116 Preface.... xvii ElizabethStojanovski and Kerrie L.Mengersen VOLUME1 BayesianStatistics 122 JuliaA.Varshavsky and Stacy R.Lindborg Acceptance Sampling 1 William A.Brenneman and WilliamR. Myers BayesianDesignsfor Phase II Oncology ClinicalTrials 128 Active ControlTrials 8 SzeHuey Tan,David Machin, and Say BengTan George Y. H.Chi, GangChen,Mark Rothmann, andNing Li Binary232 Crossover Trials 136 AdaptiveDesign Methodsin ClinicalTrials 16 LiZhu,Juan Li,and EricChi Shein-Chung Chowand AnnpeyPong Bioassay 143 AdaptiveSurvivalTrials 28 AnantM.Kshirsagar William F.Rosenberger and Lanju Zhang BioavailabilityandBioequivalence 146 Adjustment for Covariates 30 BrianSmith Thomas Permutt Bioinformatics 151 Adverse EventReporting 33 SeanD.Mooney and JeffreyT. Chang Cynthia Penniseand Cheng-Tao Chang Biologics 158 AlphaSpending Function 38 P.A.Lachenbruch, A.D.Horne, C. J.Lynch,J.Tiwari, Jihao Zhouand GlenAndrews andSusan S.Ellenberg AmesTest 45 BiomarkerinClinical Trials 167 WherlyP.Hoffman and Michael L.Garriott Mark Chang Analysis of23K Tables 52 Biopharmaceutics 170 ShivaGautam IainJ.McGilveray Analysis ofClustered BinaryData 58 BiosimilarityofFollow-On Biologics 186 Valerie Durkalski Shein-ChungChow and Jen-pei Liu Analysis ofClustered Categorical Data 63 Blinding 196 Kalyan Das Shein-ChungChow and JunShao Analysis ofHeritability 71 Bootstrap,The 202 Brent D. Burchand IanR. Harris JunShao Analysis ofRepeatedMeasures Data withMissing Bracketing Design 207 Values: AnOverview ofMethods 76 JohnR. Murphy Keumhee Carriere Chough,Yuanyuan Liang,and TaesungPark BridgingStudies 208 Jen-peiLiu Analysis ofVariance 82 Maria Overbeck-Larisch and WernerSanns Calibration 212 Shein-ChungChow ANCOVAApproach for Premarketing Shelf Life Determination withMultipleFactors 93 CanadianHealth Productsand FoodBranch (HPFB) Yi Tsong,Chi-wan Chen,Wen Jen Chen,RoswithaE. Kelly, andTherapeutic Products Directorate(TPD) 219 DaphneT. Lin,and KarlK. Lin DongshengTu AssayDevelopment 97 CancerTrials 223 Timothy L.Schofield CletNiyikiza and Douglas E.Faries AssayValidation 104 Carcinogenicity StudiesofPharmaceuticals 230 Timothy L.Schofield KarlK. Lin BayesianApproach to Stability Analysis 111 Carry-ForwardAnalysis 245 Jie Chen,JinglinZhong, and LeiNie NaiteeTing v [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] vi Contents Case-ControlStudies, Inferencein 250 Cutoff Designs 386 GaryKingand LangcheZeng JosephC. Cappelleriand William M.K. Trochim CenterWeighting in MulticenterTrials 260 Data Miningand BiopharmaceuticalResearch 392 PaulGallo Patricia B.Cerrito ClinicalDataManagement 267 Data MonitoringCommittees(DMC) 397 TerriMadison and Marianne Plaunt Walter W.Offen ClinicalEndpoint 273 Design andAnalysis for Demonstrating SofiaPaul Disease Modification Effects 405 Djokouri A.Kouassiand AnnpeyPong ClinicalPharmacology 276 BrianSmith Diagnostic Imaging 409 Suming W.Changand AnnpeyPong ClinicalTrialProcess 279 PaulF. Kramer Dose Proportionality 415 C. GordonLaw ClinicalTrialSimulation 282 Hung-IrLiand Pan-Yu Lai Dose Response Analysis inClinicalTrials 418 James MacDougall ClinicalTrialSimulations for EarlierDevelopment Phases 285 Dose Response StudyDesign 423 Mark Chang NaiteeTing ClinicalTrialSimulations for LaterDevelopment Dropout 428 Phases 290 CindyRodenberg,Dan Schnell,and CameronS. Liu Mark Chang Drug Development 433 ClinicalTrials 297 NaiteeTing KennethB.Schechtman Ecologic Inference 439 ClusterTrials 304 SanderGreenland JohnS. Preisser and BingLu ED /ED 449 50 90 Clustered StudyDesigns: PowerAnalysis 312 YangxinHuang J.Zhang,C. Feng,W.Tang, X.M.Tu,and J.Kowalski Enrichment Design 456 CombinationDrug Clinical Trial 324 Jen-pei Liu H.M. JamesHung Equivalence Trials 459 Comparing Variabilitiesin ClinicalResearch 327 Jen-pei Liu YongheeLee,Hansheng Wang,and Shein-Chung Chow Ethnic Factors 464 ConfidenceInterval andHypothesis Testing 340 Melody H. Lin and Helen McGough IvanS. F.Chan and DevanV.Mehrotra On Evaluation ofLinearity inAssayValidation 467 Confoundingand Interaction 343 Jen-pei Liu and EricHsieh Catharine B.Stack Expiration DatingPeriod 475 ContentUniformity 348 AnnpeyPong JohnR. Murphy Exploratory FactorAnalysis 480 Contract ResearchOrganization (CRO) 351 JosephC. Cappelleriand Robert A.Gerber T.Y. Lee Extra Variation Models 486 CorrelatedProbit Model 355 JorgeG.Morel and Nagaraj K. Neerchal Ralitza V.Gueorguieva Factor Analysis 500 Cost-Effectiveness Analysis 363 Mary D.Sammel, Sarah J.Ratcliffe,and ShahramHeshmat Benjamin E.Leiby Covariate-AdjustedAdaptive Dose-Finding Factorial Designs 505 inEarly Phase ClinicalTrials 369 Junfang Li PeterF. Thalland HoangQ. Nguyen Failure-Time Model 516 CrossoverDesign 380 Chin-ShangLi StephenSenn [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] Contents vii False DiscoveryRate (FDR) 522 InstrumentDevelopment andValidation 635 Avner Bar-Hen,Philippe Broet, CyrilDalmasso, CindyRodenberg,JamesT. Kuznicki, and Jean-Jacques Daudin,and Stephane Robin GloriaH. Yiu Food andDrug Administration 529 IntegratedSummary Report 646 Stacy R.Lindborg and SusanS.Ellenberg LauraJ.Meyerson Generalizability Probabilityin ClinicalResearch 534 Intention-to-Treat Analyses(ITT) 650 Shein-Chung Chow RonaldK. Knickerbocker GeneralizedEstimating Equation 537 InterimAnalysis 654 MyungheeChoPaik and Hye-Seung Lee PaulGallo GeneralizedEstimating Equations (GEE)Method: InternationalConference onHarmonization (ICH) 659 Sample Size Estimation 543 Jen-peiLiu and Shein-Chung Chow Sin-Ho Jung InvestigatingQuality-of-Life in ClinicalTrials 663 GeneralizedInference 547 PatriciaB.Cerrito Chen-TuoLiao andChi-Rong Li IVRS/IWRS forRandomization 668 GeneticLinkageandLinkageDisequilibriumAnalysis 550 Mon-Gy Chen Kongming Wang,Bernice Porjesz,Henri Begleiter, and Kaplan–Meier Estimator 674 Kevin Jones HongyuJiangand Jason Fine GlobalDatabase andSystem 560 KappaCoefficients in MedicalResearch 679 AliceT. M.Hsuan and PatrickGenyn HelenaChmura Kraemer GoodClinicalPractice 565 Kullback–LeiblerDivergence for Evaluating Mamoru Narukawa and Masahiro Takeuchi Equivalence 686 GoodProgramming Practice 571 Vladimir Dragalin AileenL. Yam LaboratoryAnalyses 694 GoodStatistics Practice 577 MichaelJ.Klepper Shein-Chung Chow LatentClass Analysis 700 GroupSequential Methods 582 ElizabethS. Garrett-Mayer andJeannie-Marie S. Leoutsakos Weichung JoeShih LillyReferenceRanges 708 GroupSequential TestsandVariance Heterogeneity MichaelG.Wilson in ClinicalTrials 590 LocalInfluence Analysis 722 Keumhee Carriere Chough,Abdulkadir Hussein,and Nicola Sartori TaesungPark Hypotheses andFalse PositiveRatein Active Control LogisticRegression 725 Non-InferiorityTrials 595 RickChappell and JasonFine GangChen,George Y.H. Chi,and Yong-Cheng Wang LogisticRegressionin Three-Point Designs 730 Hypothesis Testing 601 Ma˚rten Va˚gero¨and Rolf Sundberg DevanV.Mehrotra and Ivan S.F. Chan VOLUME2 Imputation inClinical Research 604 MaximumTolerable Dosefor Cancer Chemotherapy 735 Hansheng Wangand Shein-Chung Chow Jen-peiLiu Imputation withItemNonrespondents 609 McNemar’sTest 740 Hansheng Wangand Shein-Chung Chow Robert G.Lehr InVitro BioequivalenceTesting 614 Measuring Agreement 745 Hansheng Wang,YingZhang, JunShao,and Shein-Chung Chow LawrenceI-Kuei Lin InVitro Dissolution ProfileComparison 620 MedDRAandItsImpacton Pharmaceutical Yi Tsong,Pradeep M.Sathe,and Vinod P.Shah Development 751 InVitro Micronucleus Test 625 KeyaT. Pitts WherlyP.Hoffman, MichaelL. Garriott, and CindyLee MedicalDevices 756 Individual Bioequivalence 629 GregCampbell,Lilly Q. Yue,Gene Pennello, and Shein-Chung Chowand Jen-pei Liu MaryK. Barrick [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] viii Contents Meta-AnalysisofTherapeutic Trials 768 Noninferiority Analysis inActive Controlled JosephC. Cappelleri,John P.A.Ioannidis, andJoseph Lau ClinicalTrials 904 Sue-Jane Wang,H. M.James Hung,and Yi Tsong Microarray GeneExpression 780 JamesJ.Chen and Chun-HouhChen Non-parametric Regression 908 Kongming Wang and TheoGasser MinimizationProcedure 795 DongshengTu Odds Ratio 917 Dongsheng Tu MinimumEffective Dose 799 Jen-peiLiu OnsetofAction 922 Thomas R. Stigerand ChristyChuang-Stein MinistryofHealth, LabourandWelfare and Pharmaceutical Administrationin Japan 801 Ordered MultipleClass ReceiverOperating Characteristic KiyohitoNakai (ROC)Analysis 929 Christos T. Nakasand Constantin T.Yiannoutsos MissingValuesin RepeatedMeasurement Designs 808 KeumheeCarriere Chough,Yuanyuan Liang,RongHuang,and Outlier Detection in ClinicalResearch 933 XiaomingSheng Siu-Keung Tse andLiming Xiang MixedEffects Models 813 P-Values 940 DonghuiZhang, ChunpengFan, and A.Lawrence Gould StephenSenn Mixed-OutcomeData 817 P-Values,Evidence, andMultiplicity Considerations A.R.de Leonand Keumhee Carriere Chough for Controlled ClinicalTrials 949 Mohammad F. Huque, Mohamed Alosh, MMRMwithMissing Data 823 Satya D.Dubeyand Rafia Bhore AnnpeyPong,Allen Lee,JunZhao, Phillip Phiri,and LevSverdlov ParallelDesign 962 ManiY. Lakshminarayanan Modified LargeSample Method 827 YongheeLee Patient Compliance 967 Kenneth B.Schechtman Multicenter Trials 833 WilliamJ.Huster Percentile Charts on CorrelatedMeasures 972 XumingHeand Kai W.Ng Multicollinearity 836 K.Van Steen and G.Molenberghs Pharmacodynamic Issues 976 Cheng-Tao Chang and Robert L.Wong Multidimensional DataAnalysis:An Overview ofPenalized RegressionMethods 844 Pharmacodynamics withCovariates 983 KeumheeCarriere Choughand Xiaoming Wang Cheng-Tao Chang and Robert L.Wong Multinational ClinicalTrial 849 Pharmacodynamics withNoCovariates 993 Thomas R.Weihrauch Cheng-Tao Chang and Robert L.Wong MultipleComparisons 855 Pharmacoeconomics 999 ManiY.Lakshminarayanan JosephHeyse, John R.Cook, andMichael F. Drummond Multiple-DoseBioequivalence Studies 860 Phase I CancerClinical Trials 1011 VernonM.Chinchilli Seung-Ho Kangand ChulAhn MultipleEndpoints 864 Placebo Effect 1016 MarioComelli Thomas R. Stigerand EdwardF. C. Pun Multiple-stageDesigns for Phase IICancer Trials 872 PopulationBioequivalence 1020 MashaKocherginsky and ShangP.Lin NaiteeTing, GregC. G.Wei, and William W.B.Wang MultiplicativeIntensityModels 882 PopulationPK/PD Analysis 1026 KeumheeCarriere Choughand Abdulkadir Hussein D. Concordet, C.Ane´,and F. Le´ger Multiplicity in ClinicalTrials 889 Postmarketing Adverse Drug EventSignaling 1034 PeterWestfall andFrank Bretz Yi Tsong Multivariate Meta-Analysis 897 Postmarketing Surveillance 1045 ElizabethStojanovski andKerrie L.Mengersen Patricia B.Cerrito [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] Contents ix Power 1052 ROCCurve 1185 KennethB. Schechtman Robert G.Lehrand AnnpeyPong PredictionTrees 1054 SampleSize Calculation BasedonNonparametric Antonio Ciampi Statistics 1192 HanshengWang and Shein-Chung Chow Principal ComponentAnalysis 1061 AiyiLiu and EnriqueF. Schisterman SampleSize Calculation for SurvivalData 1200 QiJiang, StevenSnapinn, and Boris Iglewicz Prior EffectiveSample Size ofa BayesianModel 1066 Satoshi Moritaand Peter F.Thall SampleSize Determination 1206 LillyQ. Yue, DavidLi,and ShanBai Process Validation 1070 James S.Bergum and MerlinL. Utter SampleSize Re-estimation Based onObserved Treatment Difference 1219 Profile Analysis 1083 LuCui Bin Chengand Shein-Chung Chow Screening Design 1223 Propensity Score Analysis andItsApplication JohnR. Murphy in RegulatorySettings 1088 LillyQ. Yue SelectionofControlin ClinicalTrials 1224 IrvingK.Hwang ProportionofTreatment Effect 1090 CongChen and HongweiWang Semi-ParametricTime-Varying Regression Models 1229 Thomas H.Scheike Proportional Hazards RegressionModel 1093 Bee LengLee and JasonFine SequentialEstimation for the AdditiveHazards RateModelwithStaggeredEntry 1233 Protocol Development 1099 Laurent Bordesand Christelle Breuils Robert D. Chew SlopeApproach forAssessment ofDose Proportionality/ QT Analysis 1102 LinearityUnder aCrossover Design 1237 LangLi,StephenHall, and MehulDesai BinCheng and Shein-ChungChow Randomization 1110 Spatio-Temporal Modeling 1242 Shein-Chung Chowand JunShao YingC. MacNab and Charmaine B.Dean Rank-Based Robust Analysisfor Crossover Design 1114 Specifications 1251 M.Mushfiqur Rashid JohnR. Murphy Rank Regressionin Stability Analysis 1125 SROCCurve 1253 AnnpeyPong, YingQingChen,and XiudongLei S.D. Walterand Petra Macaskill ReleaseTargets 1129 Stability Analysisfor Frozen Drug Products 1262 GregC. G.Wei Shein-ChungChow and JunShao Reliability 1133 Stability MatrixDesigns 1266 ValentinRousson andTheo Gasser EarlNordbrock Reproducibility Probability inClinical Research 1141 StatisticalGenetics 1271 Shein-Chung Chow RonglingWu, GuifangFu,and HongyingLi Reproductive/Developmental Studies 1151 StatisticalPrinciples forClinical Trials 1278 James J.Chen AlanPhillips Response-Adaptive Designs 1157 StatisticalProcess Control 1282 FeifangHu andAnastasia Ivanova WilliamR. Myers andWilliam A.Brenneman Response-Adaptive Repeated Measurement StatisticalSignificance 1290 Designsfor ClinicalTrials 1162 DieterHauschke, Robert Schall,and Herman G.Luus Keumhee Carriere Choughand Yuanyuan Liang StatisticalTests for BiomarkerDevelopment with Response Surface Methodology 1171 Applicationsto Genetics Data 1294 William R.Myers GangZheng andJungnam Joo Risk RatioAnalysis 1180 Structural EquationModel 1300 Kung-JongLui JosepL.Carrasco [raghwendra][D:/informa_Publishing/CHOW_H100102_2400036/z_production/z_3B2_3D_files/978-1-4398-2245- 6_CH0000_O-Vol-I.3d][21/4/010/23:3:0][1–18] x Contents Stuart–MaxwellTest 1306 Translational Medicine: Concepts, Jiang-MingJohnnyWu Statistical Methods, andRelatedIssues 1381 Siu-Keung Tse andShein-Chung Chow Subgroup Analysis 1310 PatriciaB.Cerrito Trend Estimation 1392 JianQingShi and Man LaiTang Subject-Treatment Interaction 1316 GaryL.Gadbury Two-StageDesign: Phase IICancer ClinicalTrials 1397 Sin-Ho Jung SurrogateEndpoint 1322 ShuZhangand Edward F.C. Pun USP Tests 1401 John R.Murphy SurvivalAnalysis 1326 DirkF. Moore Vaccine ClinicalTrials 1402 Ivan S.F. Chan, William W.B.Wang,and Joseph Heyse TargetedClinicalTrials 1331 Jen-peiLiu Validation ofQuantitative andQualitative Assays 1416 BobZhong, Chunfu Qiu,and Dejun Tang Testfor Ordered Categorical Data 1338 Shein-ChungChow,Siu-Keung Tse,and Chunyan Yang Validity ofLOCF 1427 Bin Chengand Shein-Chung Chow Testing forQualitative Interaction 1343 XinYanand Xiaogang Su Z-Score 1433 Jiang-Ming JohnnyWu Therapeutic Equivalence 1349 Jen-peiLiu Zero-Inflated Poisson Distribution 1436 Siu Keung Tse,Shein Chung Chow,and QingshuLu TitrationDesign 1354 MarilynA.Aginand Edward F.C. Pun Index....1443 Toxicological Studies 1358 GuentherBaus and Thomas Hofmann TraditionalChinese Medicine—General Consideration 1370 Shein-ChungChow,Annpey Pong,and Yu-WeiChang
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