BINARYDATAANALYSISOFRANDOMIZEDCLINICALTRIALSWITHNONCOMPLIANCE

Itisquitecommoninarandomizedclinicaltrial(RCT)toencounterpatientswhodonotcomplywiththeirassignedtreatment.Sincenoncomplianceoftenoccursnon-randomly,thecommonly-usedapproaches,includingboththeas-treated(AT)andas-protocol(AP)analysis,andtheintent-to-treat(ITT)(oras-randomized)analysis,areallwellknowntopossiblyproduceabiasedinferenceofthetreatmentefficacy.ThisbookprovidesasystematicandorganizedapproachtoanalyzingdataforRCTswithnoncomplianceunderthemostfrequently-encounteredsituations.Theseincludeparallelsampling,stratifiedsampling,clustersampling,parallelsamplingwithsubsequentmissingoutcomes,andaseriesofdependentBernoullisamplingforrepeatedmeasurements.Theauthorprovidesacomprehensiveapproachbyusingcontingencytablestoillustratethelatentprobabilitystructureofobserveddata.Usingreal-lifeexamples,computer-simulateddataandexercisesineachchapter,thebookillustratestheunderlyingtheoryinanaccessible,andeasytounderstandway.Keyfeatures:Consort-flowdiagramsandnumericalexamplesareusedtoillustratethebiasofcommonlyusedapproaches,suchas,ATanalysis,APanalysisandITTanalysisforaRCTwithnoncompliance.Real-lifeexamplesareusedthroughoutthebooktoexplainthepracticalusefulnessoftestproceduresandestimators.Eachchapterisself-contained,allowingthebooktobeusedasareferencesource.IncludesSASprogramswhichcanbeeasilymodifiedincalculatingtherequiredsamplesize.Biostatisticians,clinicians,researchersanddataanalystsworkinginpharmaceuticalindustrieswillbenefitfromthisbook.Thistextcanalsobeusedassupplementalmaterialforacoursefocusingonclinicalstatisticsorexperimentaltrialsinepidemiology,psychologyandsociology.