Clinical Epidemiology: Practice and Methods

648 Pages·2021·12.269 MB·English

by  Patrick S. Parfrey, Brendan J. Barrett

Methods in
Molecular Biology 2249
Patrick S. Parfrey
Brendan J. Barrett Editors
Clinical
Epidemiology
Practice and Methods
Third Edition
M M B
ETHODS IN OLECULAR IO LO GY
SeriesEditor
JohnM.Walker
School of Lifeand MedicalSciences
University ofHertfordshire
Hatfield, Hertfordshire, UK
Forfurther volumes:
http://www.springer.com/series/7651
For over 35 years, biological scientists have come to rely on the research protocols and
methodologiesinthecriticallyacclaimedMethodsinMolecularBiologyseries.Theserieswas
thefirsttointroducethestep-by-stepprotocolsapproachthathasbecomethestandardinall
biomedicalprotocolpublishing.Eachprotocolisprovidedinreadily-reproduciblestep-by-
step fashion, opening with an introductory overview, a list of the materials and reagents
neededtocompletetheexperiment,andfollowedbyadetailedprocedurethatissupported
with a helpful notes section offering tips and tricks of the trade as well as troubleshooting
advice. These hallmark features were introduced by series editor Dr. John Walker and
constitutethekeyingredientineachandeveryvolumeoftheMethodsinMolecularBiology
series. Tested and trusted, comprehensive and reliable, all protocols from the series are
indexedinPubMed.
Clinical Epidemiology
Practice and Methods
Third Edition
Edited by
Patrick S. Parfrey and Brendan J. Barrett
Clinical Epidemiology Unit, Faculty of Medicine, Memorial University of Newfoundland, St. John’s, NL,
Canada
Editors
PatrickS.Parfrey BrendanJ.Barrett
ClinicalEpidemiologyUnit ClinicalEpidemiologyUnit
FacultyofMedicine FacultyofMedicine
MemorialUniversityofNewfoundland MemorialUniversityofNewfoundland
St.John’s,NL,Canada St.John’s,NL,Canada
ISSN1064-3745 ISSN1940-6029 (electronic)
MethodsinMolecularBiology
ISBN978-1-0716-1137-1 ISBN978-1-0716-1138-8 (eBook)
https://doi.org/10.1007/978-1-0716-1138-8
©SpringerScience+BusinessMedia,LLC,partofSpringerNature2021
Thisworkissubjecttocopyright.AllrightsarereservedbythePublisher,whetherthewholeorpartofthematerialis
concerned,specificallytherightsoftranslation,reprinting,reuseofillustrations,recitation,broadcasting,reproduction
onmicrofilmsorinanyotherphysicalway,andtransmissionorinformationstorageandretrieval,electronicadaptation,
computersoftware,orbysimilarordissimilarmethodologynowknownorhereafterdeveloped.
Theuseofgeneraldescriptivenames,registerednames,trademarks,servicemarks,etc.inthispublicationdoesnotimply,
evenintheabsenceofaspecificstatement,thatsuchnamesareexemptfromtherelevantprotectivelawsandregulations
andthereforefreeforgeneraluse.
Thepublisher,theauthors,andtheeditorsaresafetoassumethattheadviceandinformationinthisbookarebelievedto
betrueandaccurateatthedateofpublication.Neitherthepublishernortheauthorsortheeditorsgiveawarranty,
expressedorimplied,withrespecttothematerialcontainedhereinorforanyerrorsoromissionsthatmayhavebeen
made.Thepublisherremainsneutralwithregardtojurisdictionalclaimsinpublishedmapsandinstitutionalaffiliations.
ThisHumanaimprintispublishedbytheregisteredcompanySpringerScience+BusinessMedia,LLC,partofSpringer
Nature.
Theregisteredcompanyaddressis:1NewYorkPlaza,NewYork,NY10004,U.S.A.
Preface
Clinical epidemiology provides the scientific basis for the practice of medicine, because it
focuses on the diagnosis, prognosis, and management of human disease. It is a relatively
recent science that initially focused on longitudinal studies, randomized controlled trials,
and evaluation of diagnostic tests, and progressed to evidence-based decision-making
revolving around critical appraisal, systematic reviews, meta-analysis, health technology
assessment,clinicalpracticeguidelines,andhealtheconomics.
Morerecently,clinicalepidemiologyhasextendedtoknowledgetranslationandpatient
engagementtogether withendeavorstoimprovehealth-relatedbehaviorsusingimplemen-
tationscience,andavailingofadministrativedatabasesandbigdatatechnology.
Issues related to research design, measurement, and evaluation are critical to clinical
epidemiology.ThisthirdeditionofClinicalEpidemiology:PracticeandMethodshasupdated
chaptersonlongitudinalstudies,randomizedtrials,other researchmethods,andevidence-
baseddecision-making,andincludesanewsectiononchanginghealth-relatedbehaviors.Its
intendedreachisconsequentlywidenotonlytoincludetraineesinclinicalepidemiologyand
researcherswhowanttoundertakeclinicalresearch,whethertheybeclinicalpractitionersor
basicscientistswhowanttoextendtheir worktohumans,butalsousersofclinicalresearch
findingswhether theybeclinicalpractitionersordecisionmakersinthehealthsystem.
This book is divided into six parts. The first part is concerned with how to frame a
clinicalresearchquestionandchoosetheappropriatedesign,biasesthatmayoccurinclinical
research,andtheethicsassociatedwithdoingaresearchprojectinhumans.
Parts II–IV discuss issues of design, measurement, and analysis that pertain to various
research questions, including determination of risk in longitudinal studies, assessment of
therapyinrandomizedcontrolledtrials,andevaluationofdiagnostictests.
PartVpresentsmethodsinthevariouscomponentsofevidence-baseddecision-making
andPartVIisfocusedoninterventionsthatcouldchangehealth-relatedbehaviors.Inview
of the recent emphasis of granting agencies on knowledge translation and patient engage-
mentChapters25,29,and32shouldbeofinterest.
The content varies from the basics of clinical epidemiology to more advanced chapters
examiningbias(Chapter3),theanalysisoflongitudinalstudies(Chapters7and8),rando-
mized trials (Chapters 16 and 17), and genetic epidemiology (Chapter 19). Examples and
case studies have been encouraged particularly in Part VI on Changing Health-Related
Behaviors.
Allthecontributorstothisvolumearepracticingclinicalepidemiologists,whohopethe
reader willjointhemindoingresearchfocusedonimprovingclinicaloutcomes.
St.John’s,NL,Canada PatrickS.Parfrey
BrendanJ.Barrett
v
Contents
Preface ..................................................................... v
Contributors................................................................. xi
1 OnFramingtheResearchQuestionandChoosingtheAppropriate
ResearchDesign........................................................ 1
PatrickS.ParfreyandPietroRavani
2 BiasinClinicalResearch ................................................. 17
SusanStucklessandPatrickS.Parfrey
3 DefinitionsofBiasinClinicalResearch .................................... 35
GeoffreyWarden
4 ResearchEthicsforClinicalResearchers ................................... 53
JohnD.Harnett
5 Ethical,Legal,andSocialIssues(ELSI)inClinicalGeneticsResearch ......... 65
DarylPullmanandHollyEtchegary
6 LongitudinalStudies1:DeterminantsofRisk .............................. 83
SeanW.Murphy
7 LongitudinalStudies2:ModelingDataUsingMultivariateAnalysis........... 103
PietroRavani,BrendanJ.Barrett,andPatrickS.Parfrey
8 LongitudinalStudies3:DataModelingUsingStandardRegression
ModelsandExtensions .................................................. 125
PietroRavani,BrendanJ.Barrett,andPatrickS.Parfrey
9 LongitudinalStudies4:MatchingStrategiestoEvaluateRisk ................ 167
MatthewT.James
10 LongitudinalStudies5:DevelopmentofRiskPredictionModels
forPatientswithChronicDisease......................................... 179
NavdeepTangriandClaudioRigatto
11 RandomizedControlledTrials1:Design .................................. 193
BryanM.Curtis,BrendanJ.Barrett,andPatrickS.Parfrey
12 RandomizedControlledTrials2:Analysis ................................. 213
RobertN.FoleyandPatrickS.Parfrey
13 RandomizedControlledTrials3:MeasurementandAnalysis
ofPatient-ReportedOutcomes........................................... 229
MichelleM.RichardsonandKlemensB.Meyer
14 RandomizedControlledTrials4:Planning,Analysis,
andInterpretationofQuality-of-LifeStudies............................... 247
RobertN.FoleyandPatrickS.Parfrey
vii
viii Contents
15 RandomizedControlledTrials5:BiomarkersandSurrogates/Outcomes...... 261
ClaudioRigattoandBrendanJ.Barrett
16 RandomizedControlledTrials6:DeterminingtheSampleSize
andPower forClinicalTrialsandCohortStudies........................... 281
TomGreene
17 RandomizedControlledTrials7:OnContamination
andEstimatingtheActualTreatmentEffect................................ 307
PatrickS.Parfrey
18 EvaluationofDiagnosticTests............................................ 319
BrendanJ.BarrettandJohnM.Fardy
19 GeneticEpidemiologyofComplexPhenotypes............................. 335
DarrenD.O’RiellyandProtonRahman
20 QualitativeResearchinClinicalEpidemiology.............................. 369
StephanieThompsonandKaraSchick-Makaroff
21 Evidence-BasedDecision-Making1:CriticalAppraisal....................... 389
LaurieK.Twells
22 Evidence-BasedDecision-Making2:SystematicReviews
andMeta-Analysis ...................................................... 405
AminuBello,BenVandermeer,NatashaWiebe,
AmitX.Garg,andMarcelloTonelli
23 Evidence-BasedDecisionMaking3:HealthTechnologyAssessment.......... 429
DariaO’Reilly,RichardAudas,KaitrynCampbell,
MeredithVanstone,JamesM.Bowen,LisaSchwartz,
NazilaAssasi,andRonGoeree
24 Evidence-BasedDecisionMaking4:ClinicalPracticeGuidelines.............. 455
TaeWonYi,SineDonnellan,andAdeeraLevin
25 Evidence-BasedDecisionMaking5:KnowledgeTranslation
andtheKnowledgetoActionCycle....................................... 467
BradenJ.Manns
26 Evidence-BasedDecisionMaking6:AdministrativeDatabases
asSecondaryDataSourceforEpidemiologicandHealth
ServiceResearch........................................................ 483
TanvirTurinChowdhuryandBrendaR.Hemmelgarn
27 Evidence-BasedDecisionMaking7:HealthEconomics
inClinicalResearch ..................................................... 501
BradenJ.Manns
28 Evidence-BasedDecision-Making8:APrimeronHealthPolicy
forResearchers ......................................................... 517
VictorMaddalenaandMaisamNajafizada
29 ChangingHealth-RelatedBehaviors1:Patient-OrientedResearch
andPatientEngagementinHealthResearch ............................... 537
CatherineStreet,LaurieK.Twells,ToniLeamon,
LynnTaylor,andHollyEtchegary
Contents ix
30 ChangingHealth-RelatedBehaviors2:OnImprovingtheValue
ofHealthSpending ..................................................... 553
Karen Dickson,RobertWilson,OwenParfrey,andPatrickS.Parfrey
31 ChangingHealth-RelatedBehaviors3:LessonsfromImplementation
Science ................................................................ 571
AmandaHall,HelenRichmond,KristaMahoney,andJamesMatthews
32 ChangingHealth-RelatedBehaviors4:RealizingImpactofHealth
ResearchThroughKnowledgeTranslation................................. 597
KateLambert,KristaMahoney,andPatrickS.Parfrey
33 ChangingHealth-RelatedBehaviours5:OnInterventionstoChange
PhysicianBehaviours.................................................... 613
CherylEtchegary,LynnTaylor,KristaMahoney,
OwenParfrey,andAmandaHall
34 ChangingHealth-RelatedBehaviors6:Analysis,Interpretation,
andApplicationofBigData.............................................. 631
RandyGiffenandDonaldBryant
Index ...................................................................... 645
Contributors
NAZILA ASSASI • ProgramforAssessmentofTechnologyinHealth(PATH),St.Joseph’s
HealthcareHamilton,Hamilton,ON,Canada;DepartmentofClinicalEpidemiology
andBiostatistics,FacultyofHealthSciences,McMasterUniversity,Hamilton,ON,
Canada
RICHARDAUDAS • DivisionofCommunityHealthandHumanities,FacultyofMedicine,
MemorialUniversityofNewfoundland,St.John’s,NL,Canada
BRENDANJ.BARRETT • ClinicalEpidemiologyUnit,FacultyofMedicine,Memorial
UniversityofNewfoundland,St.John’s,NL,Canada
AMINU BELLO • DivisionofNephrology,DepartmentofMedicine,UniversityofAlberta,
Edmonton,AB,Canada
JAMESM.BOWEN • ProgramforAssessmentofTechnologyinHealth(PATH),St.Joseph’s
HealthcareHamilton,Hamilton,ON,Canada;DepartmentofClinicalEpidemiology
andBiostatistics,FacultyofHealthSciences,McMasterUniversity,Hamilton,ON,
Canada
DEONALD BRYANT • QualityofCareNL,FacultyofMedicine,MemorialUniversityof
Newfoundland,St.John’s,NL,Canada
KAITRYNCAMPBELL • ProgramforAssessmentofTechnologyinHealth(PATH),St.Joseph’s
HealthcareHamilton,Hamilton,ON,Canada;DepartmentofClinicalEpidemiology
andBiostatistics,FacultyofHealthSciences,McMasterUniversity,Hamilton,ON,
Canada
TANVIRTURINCHOWDHURY • DepartmentofFamilyMedicine,CummingSchoolof
Medicine,UniversityofCalgary,Calgary,AB,Canada;DepartmentofCommunity
HealthSciences,CummingSchoolofMedicine,UniversityofCalgary,Calgary,AB,
Canada
BRYANM.CURTIS • DepartmentofMedicine(Nephrology),FacultyofMedicine,Memorial
UniversityofNewfoundland,St.John’s,NL,Canada
KARENDICKSON • QualityofCareNL,DepartmentofMedicine,MemorialUniversity,
St.John’s,NL,Canada
SINEDONNELLAN • DivisionofNephrology,UniversityofBritishColombia,Vancouver,BC,
Canada
CHERYLETCHEGARY • QualityofCareNL,FacultyofMedicine,MemorialUniversityof
Newfoundland,St.John’s,NL,Canada
HOLLY ETCHEGARY • StrategyforPatient-OrientedResearch(SPOR—CIHR),Facultyof
Medicine,MemorialUniversity,St.John’s,NL,Canada
JOHNM.FARDY • DepartmentofMedicine,MemorialUniversityofNewfoundland,
St.John’s,NF,Canada
ROBERTN.FOLEY • USRDSCo-ordinatingCenter,UniversityofMinnesota,Minneapolis,
MN,USA
AMITX.GARG • DivisionofNephrology,DepartmentofEpidemiologyandBiostatistics,
WesternUniversity,London,ON,Canada
RANDYGIFFEN • IBMCanada,Ottawa,ON,Canada
RONGOEREE • ProgramforAssessmentofTechnologyinHealth(PATH),St.Joseph’s
HealthcareHamilton,Hamilton,ON,Canada;DepartmentofClinicalEpidemiology
xi