艾昆緯-使用患者體驗(yàn)數(shù)據(jù)評估醫(yī)療干預(yù)措施-第7章

UsingPatientExperienceDatatoEvaluate

MedicalInterventions

Generating,understandingandusingpatient

experiencedatawithinandalongsideclinicaltrials.

EDITOR:Dr.MatthewReaney

ThisfilecontainsonlyChapter7ofthisbook.Fora

fullcopyofthebookpleaseemailPCSBD@

Pleasecitethischapteras:RoborelDeClimensA,CasamayorM,

FarrellM,ReaneyM(2023).Generatingpatientexperiencedata

(PED)incomplexpopulationsandprograms;examplesofpediatrics,centralnervoussystem(CNS),cancerandraredisease.InReaneyM(ed.)UsingPatientExperienceDatatoEvaluateMedical

Interventions.Generating,understandingandusingpatient

experiencedatawithinandalongsideclinicaltrials.IQVIA.Pages88-127.

=IQVIA

TableofContents

1Patientexperiencedata(PED)ininterventiondevelopment–whatisit,whyshouldyoucare,andhowcanthisbookhelp?

MattReaney

Section1:Generatingpatientexperiencedata

2Makinguseofexistingdata–approachestoidentifying,appraisingandinterpretingsecondarypatientexperiencedata(PED)

ObinnaOnwude,BetsyWilliams

3Understandingpatientexperiencesthroughqualitativeresearch

RobertKrupnick,KimberlyKelly

4Collectingpatientexperiencedata(PED)withclinicaloutcomeassessments(COAs)

OrenMeyers,IsabelleGuillemin,MattReaney

5Patientpreferenceresearchtounderstandpatients’expectationsandexperiences

AnaMariaRodriguez-Leboeuf,LaurieBatchelder,StephaniePhilpott,WillingsBotha

6Theroleofdigitaltechnologyingeneratingandcapturingpatientexperiencedata(PED)

PipGriffiths,MichaelPosey

7Generatingpatientexperiencedata(PED)incomplexpopulationsandprograms;examplesofpediatrics,centralnervoussystem(CNS),cancerandraredisease

AudeRoboreldeClimens,MontseCasamayor,MeaganFarrell,MattReaney

Section2:Understandingpatientexperiencedata

8Theimportanceofensuringvalid,reliable,responsiveandinterpretablepatientexperiencedata(PED)

ChristinaDaskalopoulou,KonstantinaSkaltsa

9Usingprinciplesofbehavioralsciencetounderstandandenhancepatientexperiencedata(PED)

KateHamilton-West,RachelBruce,SandravanOs

Page3

Page9

Page20

Page43

Page57

Page75

Page88

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1|UsingPatientExperienceDatatoEvaluateMedicalInterventions

Section3:Usingpatientexperiencedata

10Integrationofpatientexperiencedata(PED)intoregulatoryandpayerdecision-making

JoyWhitsett,MattReaney,LiviaLai

11Disseminatingpatientexperiencedata(PED)tothemedicalandscientificcommunity

EmilyRuzich,CathyYang,TwinkleKhera,AliseNacson,ShwetaShah,PaulWilliams

12Understandinghowtocommunicatepatientexperiencedata(PED)topatients

FranceGinchereauSowell,MattReaney

Section4:Developingapatientexperiencedatastrategy

13Apatientexperiencedata(PED)roadmapforpatient-focusedinterventiondevelopment,determinationandevaluation

JulietDavis,JamesTurnbull,MattReaney

Authorbiographies

Page168

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SECTION1:GENERATINGPATIENTEXPERIENCEDATA

CHAPTER7

Generatingpatientexperiencedata(PED)incomplexpopulationsandprograms;examplesofpediatrics,centralnervoussystem(CNS),

cancerandraredisease

AROBORELDECLIMENS,MCASAMAYOR,MFARRELL,MREANEY

Keytakeaways

?Pediatricpopulations

?Childrenshouldbeabletoengageinqualitativeandquantitativepatientexperiencedata(PED)researchfromage8.

?Between5and8,validityofself-reportisquestionableandshouldbebasedonverysimple,concrete,current,andsufficientlyremarkableevents.

?Askingconcretemomentaryassessmentsis

recommendedforyoungchildrenandnewdigitaltechnologiescouldhelpwiththis.

?Whenchildrenareunabletoself-report,observablefactsshouldbeevaluatedbyexternalpersons,andproxyevaluationavoided.

?Populationswithcentralnervoussystem(CNS)disorders

?PatientswithCNSdisordersexperienceawidearray

ofphysical,cognitiveand/orsensorylimitationsthatcanmakeitdifficultforthemtoreflectandreporton

theirexperiences.Further,measurementofthepatientexperienceinCNSdisordersisoftendonethrough

instrumentsthatdonothavesufficientevidenceforcontentvalidityandpsychometricproperties.

?Despitethesechallenges,reliableandinformative

PEDcanbegeneratedinCNSdisorderswithcareful

considerationofthespecificneedsofthepatient

population,theconceptsofinterest,andtheapproachtomeasurement.

?Cancerprograms

?PEDisincreasinglyimportantinthedevelopmentofanti-cancerdrugs,andincancercare.However,generationofrobustandinformativePEDis

complicatedbytheincreasinguseofaccelerateddrugdevelopmentprogramsandbasketand

superumbrellatrials,highattritionrates,anda

substantialamountofmissingPEDduetodiseaseprogression,treatment-relatedtoxicityanddeath.

?YetPEDcollectionispossibleandinformativewithappropriateplanning.

?Rarediseaseprograms

?Rarediseasesareoftencomplex,multisystemicandchronic.Mostpeoplewithararediseasewillnotbecuredintheirlifetimes,andthusidentifyingwaystoimprovequalityoflife(QoL)isimportant.

?Smallheterogenoussamples,non-traditional

interventiondevelopmentprograms,andpediatricpopulationsmakemeasurementofQoLintrials

difficult.However,acarefullyconsideredmixed-

methodsapproachtoQoLdatacollection,coupledwithotherPED,canbevaluablefordecision-makinginraredisease.

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AdviceforresearchersinterestedincollectingPEDincomplexpopulations

andprograms

?Pediatricpopulations

?TheabilitytocollectvalidPEDwithchildrenwill

varyacrossagegroupsanddevelopmentalstages.PrecautionsandinteractiveapproachesshouldbeusedtomakechildrenandadolescentscomfortableandimprovetheirengagementinPEDresearch.

?Theuseofinnovative,participatoryandfriendly

digitalhealthtoolsadaptedtothepediatric

populationmayimprovecomplianceandcollectionofrobustdata.

?PopulationswithCNSdisorders

?ResearchersmustevaluatewhetherpatientswithCNSdisordershavesufficientcapacitytoprovideaccurateandreliablereportsoftheirexperiences.

?QualitativeinterviewscanbeconductedwithpatientswithselectCNSdisorders,althoughadaptations

andaccommodationsmaybeneededforthosewithcommunicativelimitations.Quantitativeapproachesmayneedtocombineinformationfrommultiple

sourcestodevelopthemostcomprehensivepictureofthepatientexperience.

?Cancerprograms

?PEDincanceroftencomesfrompatient-reported

outcome(PRO)instruments.Carefulplanningis

essentialfordataanalysisandinterpretation.If

acceleratedapprovalislikelytobesought,standalonestudiesmaybeneededtosupportPROinterpretation,includingthegenerationofpsychometricevidence.

?OtherformsofPEDcanbeusefultosupplementPROdata,includingpreferenceresearch.

?Rarediseaseprograms

?PROinstrumentsshouldbeusedtocollect

informationonQoL,wherepossible,fromrare

diseasepopulations.Datacollectedfromcliniciansandcaregiversshouldalsobeconsideredto

supplementknowledgefromPROs.OtherPEDisalsorelevantandimportantinrarediseaseandshouldbeconsidered.

?Strategiesshouldbeconsideredtoincreasethereliability,validityandgeneralizabilityoffindingsfromPEDinraredisease.

“Youguysgiveup?Orareyouthirstyformore?”

—KevinMcCallisterinHomeAlone(1990film)

Priorchaptersinthisbookhaveoutlinedtheapproachestogeneratingreliable,valid,meaningfulandinterpretablepatientexperiencedata(PED)fromqualitativeand

quantitativeexploration.Theseapproachesare,inmost

cases,triedandtestedacrossdrugdevelopmentprogramsfromvariousinstitutionsandinterventiondevelopers/

sponsors.However,therearesomepopulationsinwhichthecollectionofPEDisabitmoredifficult.Thisincludes

collectionofPEDfrompeoplewithsymptomsthatmakereportingexperiencesdifficult.Thisincludespeoplewithspeechissues,physicallimitationsimpactingoninabilitytocompletesurveysorclinicaloutcomeassessments

(COAs),orcognitiveimpairment.Twospecificpopulations

inwhichthecollectionofPEDneedsspecialconsiderationarechildrenandthosewithneurologicalorpsychiatric

indications(summarizedas“centralnervoussystem(CNS)disorders”).ThischapterwilldiscussthecollectionofPEDinchildrenandamongpeoplewithCNSdisorders.

Inaddition,priorchaptersinthisbookhavefocusedon

collectingPEDinatraditionalmodelofdrugdevelopment;thatisastructuredprogressionfromPhaseI,through

PhaseIIandPhaseIIIforregistration.Insomediseases,includingoncologyandraredisease,thismaynotbe

possibleornecessary.Thischapterwillthereforealsodiscusstheimpactofnoveldevelopmentpathwayson

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thegenerationanduseofPEDinthecontextofoncology,andprovideashortdiscussiononrarediseasesforwhomgenerationofPEDhavesomeadditionalnuancesthatareworthhighlighting.

GeneratingPEDin

pediatricpopulations

AROBORELDECLIMENS

Chapter3ofthisbookdiscusstheroleofqualitative

insightsasthecornerstoneofPED,explaininghow

discussionswithpatientsareessentialtounderstand

perspectivesofdisease,preferencesandpriorities

fortreatment,andexperiencesofresearchstudies.

Subsequentchapters(Chapters4–6)speaktotherole

ofPEDderivedfromstructuredoutcomemeasuresand

surveyscompletedbythepatient(patient-reported

outcome(PRO)instruments;patientpreferenceresearch)ordeliveredthroughtechnologywornbythepatient.

TheseallinherentlyassumethatreliableandvalidPEDcanbegeneratedfromthepatientpopulation.ThismaynotalwaysbethecasewhentryingtogeneratePED

fromchildren.

Childrenaredefined–forthepurposeofpediatric

research–asthosebetweentheagesof0and17.This

isquitealargeagerange,withsignificantdifferences

inmotor,cognitive,linguistic,socialandemotional

developmentininfants,youngchildren,adolescents,andyoungadults.1,2Acknowledgingthisbroadagerange,

itisimportanttoexplorewhetherchildrencanreliablyreporttheirexperiences,andhowtogeneratePEDaboutthosewhocannot.

HOWRELEVANTISPEDINPEDIATRIC

INTERVENTIONDEVELOPMENT?

Someinterventionsaredevelopedspecificallyforuseinchildren,whileothersaredevelopedforuseinadultsandusedoff-labelinchildren.3,4However,childrenarenot“l(fā)ittleadults,”andinterventionsforchildrenneed

specificconsiderationstoensurethattheyaremeetingthepriorities,needsandpreferencesofchildrenand

theircaregivers.Thisinformationcanbeobtained

throughPED.ForthecollectionofPED,researchers

generallyrecommenddirectreportsfrompatients,5

butthisisnotalwayseasyinpediatricresearchand

thus,childrenarenotalwaysaskedtosharetheirownexperiencesandinsighttoinformthedevelopmentofinterventions.6,7Therearesomeinitiatives,including

theInternationalChildren’sAdvisoryNetwork(iCAN),

thatareseekingtochangethisthroughgeneratinga

networkofyoungpersons’advisorygroups(YPAGs)

forinterventiondevelopment.Researchsupportssuchinitiatives,showingthepotentialforimprovedsuccessinthetrialprocess(includingrecruitmentandretention)andsubsequenthealthoutcomeswheninvolving

childrenindesign.8,9Researchersandfamiliesalike

requiretrainingandeducationtobeabletoinvolvethepediatricpatientinPEDresearchinasensitivemanner.10

WHATMAKESPEDDIFFICULTTOGENERATEIN

PEDIATRICPOPULATIONS?

ThegenerationofrobustPEDreliesongoodscientific

researchmethods(asdescribedinotherchaptersofthisbook)andapatientpopulationwillingandabletoreflectonandsharetheirinsights,experiences,priorities,

attitudes,knowledgeandhealth-relatedbehaviors.

Somechildren–especiallyyoungchildren–maynot

beabletounderstandquestionsthatresearchers

tendtoasktoelicitPED,norarticulatetheirthoughtsandfeelingsinawaythatiseasilyinterpretableto

researchers.Thatdoesnot,however,meanthatrobustPEDcannotbecollectedinpediatricintervention

development.Rather,caremustbetakentomaximizequality.9,11,12

HOWDOICOLLECTROBUSTPEDIN

PEDIATRICRESEARCH?

Itisgenerallyagreedthatchildren’sunderstandingofhealth-relatedconceptsincreaseswithage,2,13andwhiledevelopmentalevolutionmaynotbethesameinall

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children,14ageisareasonablewayinwhichtoinitiallyclassifypediatricpopulationsforPEDresearch.Theageatwhichchildrencanprovidereliableinformationinaqualitativeinterview(asdescribedinChapter3)and

theageatwhichachildcanreliablyrespondtoaPROinstrumentorpreferencesurvey(seeChapters4and5)aretwodifferentissues.1

QualitativePEDresearch

Whileitisimpossibletocollecthealthinformation

directlyfrominfants,2childrenasyoungas4or5maybecapableofprovidingsomespecificinformation

onconcreteaspectsoftheirhealthstatus(about

eventsthataresufficientlyremarkable).1But,itisnot

untilage8thatchildrenaregenerallyabletoexplain

simpleconceptslike“pain”intheirownwords1,2;and

eventhenonlyabouttheircurrentorveryrecent

experience.1,15Atthisstage(i.e.,fromage8),itwould

thereforebereasonabletoengagechildreninsimple

conceptelicitationresearch;thatisqualitativeresearchtoexplorethepatientexperienceofmanagingillness,diseaseoracondition(seeChapter3fordetails).Bytheageof12,mostchildrencanunderstandandanswer

questionsaboutabstractconceptsorhypothetical

events1andthuscanbeexpectedtoanswerthe

sametypesofquestionsinqualitativeresearchas

adults.However,socialandemotionalaspectsmaybe

consideredwithcautioninadolescents(age12–17),as

theymaynotbewillingtoshare.2Thisisparticularly

truewhenthediscussioncouldcauseembarrassmentor

shame,suchaswithsexualfunctioning.16

Specialattentionshouldbepaidtostandardsforgoodpracticeinconductinginterviewswitholderchildren(age8–11)andadolescents(age12–17).Forexample,theinterviewershouldlearnthediscussionguidewelltoadaptandimprovise,17usewarm-upexercisesto

makechildrenfeelcomfortable,18useage-appropriatelanguage,1,14,16,19–21andaskquestionsinaneutral

mannerwithsimplewording,avoidingclinicalterms.16Foradolescents(age12–17),thereareadditional

considerations.Forexample,itisimportanttoavoid

“child-like”termsduringtheinterview.16Itmayalsobe

beneficialtohaveaninterviewerofthesamesexto

encourageopenandhonestdiscussion,particularly

aboutsensitivetopics.Theinterviewershouldemphasizeatthebeginningandduringtheinterviewthatthe

adolescentisfreetonotansweranyquestionstheyarenotcomfortablewith.

Itisnotuntilage8thatchildren

aregenerallyabletoexplain

simpleconceptslike“pain”in

theirownwords.

Aschildrenhavelimitedattentionspan14,19,22and

caneasilybecomebored,itisalsoimportanttouse

interactivetechniquestoengagechildreninqualitativeinterviews.14,17,19,23Theuseofdrawings,photographs,illustrations,props,andtoyscouldbebeneficialfor

childrenage8–11,especiallyiftheyareshy.14,18,21Askingthemfirsttodrawapicturedescribingtheirconditionoritsimpact,andthenexplainit,encourageschildrentoshareandhelpstartingthediscussion.1,14,15,18,21

Similarly,photo-elicitationinterviewshavebeen

showntobeparticularlybeneficialwhenworkingwithchildrenwithpoorwrittenorverballiteracy.17,21,24–26

Socialmediacanalsobeusedtoengageadolescents,provideastartingpointforconversation,andfacilitateanunderstandingofotherpeople’sexperiences.27

NHS(NationalHealthService)Digital’sWidening

DigitalParticipationProgrammefurtherrecommendsconsideringdigitaltechnologiesasanenablerfor

improvinghealthserviceswithadolescents.27Evenwiththesetechniques,thelengthofinterviewsshouldbe

shorterforadolescentsthanitisforadults,andevenshorterstillforchildrenage8–11.1,23Severalbreaks

shouldalsobemadeduringaninterview.1

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Focusgroups–involvingadiscussionbetweenaresearcherandasmallgroupofparticipantsto

exploreissuesbothattheindividuallevelandby

encouragingdiscussionsamongparticipants–can

offeranalternatewayofgeneratingPEDfromchildrenandadolescents.Ifconductedinsmallgroups(i.e.,

4–6)withchildrenofasimilarage,itmaybeeasierforchildrenandadolescentstoshareinformation.1,16,28,29

However,thegroupsettingcouldalsoinhibitsometoshare,andsocialdesirabilitymaybeexacerbated,especiallyforadolescents.1,2,16

Whilechildrenfromtheageof8canengageinsome

degreeofconceptelicitationresearch,preference

explorationmethods(qualitativeapproachesto

collectingdescriptivepreferenceinformationby

exploringpeople’sidiosyncraticexperiencesand

decisions)maybemoredifficultamongchildren.

Preferenceexplorationmethods,describedindetail

inchapter5,ofteninvolvecognitivelycomplextasks,whichmaybemoredifficultthansimpleconcept

elicitationquestionsforadolescentsandchildren.30

Ahighlevelofimaginationcanalsobeobservedin

children,whichcanbechallenginginpreference

discussions.However,priorresearchhasshownthat

childrenareabletocommunicatetheirpreferencesininterviewsifmethodsusedaresimpleandunderstood.Forexample,vignettescanbeusedininterviewsto

providechildrenconfidenceinsharingtheirpersonalexperiences,ratherthanaskingtheiropinionson

hypotheticalsituations.31,32

Itiscommontohaveparentsorcaregiverspresent

ininterviewswithchildrenage8–11,andadolescents

uponrequest(fortheparent/caregiver’soradolescents’comfort).However,thepresenceoftheparentor

caregivermayinhibitthechild–andparticularlytheadolescent–fromsharingrelevantexperiencesorfeelings.1,2,22Further,itisimportantthattheparent/caregiverdoesnotinterveneduringthediscussionbetweenthechild/adolescentandtheresearcherto

giveeithertheirownexperienceorremindthechild/adolescentofanexperienceoftheirown.2,16Thisruns

theriskofdistortingthePED.Assuch,iftheparent/

caregiverispresent,itisrecommendedtousearoomwithaone-waymirror1orasktheparenttositbehindtheirchild.2,22Whenaresearchquestiondictatesthatbothachildandtheirparent/caregiverbeinterviewed,itisbettertointerviewthemseparately.1,2

QuantitativeCOAresearch

PROinstrumentscapturedataaboutthestatusof

apatient’shealthconditionorhealth-relatedtopic

directlyfromthepatient,withoutinterpretationofthe

patient’sresponsebyaclinicianoranyoneelse.33Aswithqualitativeresearch,mostresearchersusechildren’s

ageasaproxyfortheirabilitytoreliablyself-reporttheirexperiencesonaPROinstrument.1,2Itisgenerallythoughtthatfromtheageof5childrenmaybeabletocomplete

PROinstrumentsthatarecarefullyandappropriately

targetedtotheirage(i.e.,useage-relatedvocabulary)andconsiderlanguagecomprehension,andcomprehensionofthehealthconceptmeasured,22,34althoughreliabilityandvalidityareoftenquestionableuntilage8.2

Whenpossible,then,researchersshouldusePRO

instrumentstocapturePEDdirectlyfromchildrenage5andabove,unlesstheyareunabletoreliablyreportontheconceptofinterest.5However,relativelyfew

pediatricPROinstrumentsexist,andresearchersoftenuseadultinstrumentsforchildren.Adultinstruments

maycontainquestionsthatmaybeirrelevantto

children,ordifficultforchildrentounderstand,and

thus“instrumentdevelopmentwithinfairlynarrowagegroupingsisimportanttoaccountfordevelopmentaldifferences.”35Asforadults(seedetailsinChapter

4),theselectionordevelopmentofaPROinstrument

shouldbecarefullyconsideredforchildren.SpecificconsiderationsforPROinstrumentsintendedtobe

administeredtochildrenbetween5and18include:

?Look:Pediatricinstrumentsshouldbemoreappealingandless“official-looking”thanadultinstruments.21

Illustrationsratherthanwordsmayalsohelpmaintainyoungchildren’sinterestandincreaseunderstanding.1,2

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?Conceptualcoverage:Youngchildrenmaybecapableofprovidingsomespecificinformationonconcrete

aspectsoftheirhealthstatus,whileonlyolderchildrenandadolescentsareabletoreliablyreporttheir

symptoms1,2;Adolescentscanalsoreliablyanswerquestionsaboutabstractconcepts.1

?Complexityofinstructionsanditems:Lengthof

sentencesandnumberofsentencesshouldbeshorterandsyntaxsimplerwhengeneratingPROinstrumentsinpediatrics.2Forexample,wheneditingvocabularyoftheadultPatient-ReportedOutcomesMeasurementInformationSystem(PROMIS)itembankforpediatrics,severaladaptationsweremadeforproblematicterms

(e.g.,“irritable”waschangedto“cranky”;“socialactivities”to“activitieswithfriends”).36

?Age-appropriateresponsescales:ManyPRO

instrumentsdevelopedforyoungerpopulations

includefacialexpressions(e.g.,Wong-BakerFACES?

PainRatingscale37),circlesfromincreasingsizesor

thermometers.Onthecontrary,visualresponsechoicesshouldbeavoidedforadolescents,astheycouldbe

perceivedoverlychildish.2Whenverbalresponsecategoriesareofferedinstead,fewerandsimplerresponseoptionsarerecommendedinpediatriccomparedtoadultPROinstruments,22asyoungerchildrengenerallyselectonlyextremeormiddleresponsesonaresponsescale.2,13Moregranularresponseoptionscanbeaddedforolderchildren.

?Recallperiod:MostPROinstrumentsdesigned

foradultshavearecallperiodbetweenthepast

24hoursandthepastmonth.However,anyrecall

periodmaybechallengingforchildrenyoungerthan11yearsold.1Askingchildrentoanswermomentaryassessmentsorconcrete,salientandremarkable

eventsispreferred.2

?Interviewer-administration:Foryounger

childrenwherecompletionofaPROinstrumentischallenging,anintervieweradministrationcouldbeconsidered;anadult(parentornurse)canassistthe

childreadingandcompletingtheinstrument,afterreceivingclearinstructionstoavoidinfluencingthechildintheiranswers.1

?Modality:TheuseofelectronicPROsanddigital

diariescanfacilitatedatacollection(seeChapter6),andcellphonetextmessagingcanbeusedto

remindparticipantstocompletethePROinstrumentsinatimelymanner.Electronicdevicescouldalsobeusedtoensurethatchildrenwithlowliteracylevelsarenotexcluded,asitemscanbepresentedtothe

childorallyviaacomputer-administrationoran

application.1,22,38Theuseofothercomputer-assistedtechnologies,suchastouchscreenandvideosor

games,isalsoincreasinglyconsideredforcollectingPEDwithchildren.2,22,38,39

Wherepossible,bothchildself-

reportandobservablereportare

recommendedashavingdifferent

reporters(patients,clinicians,parentsorteachers)mayprovideacomplementaryandcomprehensivepicture.1

Inpediatricclinicaltrialstosupportthedevelopment

ofanintervention,childrenwitharangeofagesand

developmentalstagesarelikelytobeincluded.InclusionofmultipleversionsofaPROinstrumentfordifferent

agegroupsmayintroduceunwantedmeasurement

variability22;rather,itisidealtohaveonePROinstrumenttomeasureaPEDoutcomeofinterestforparticipants

ofallagesinthetrial.Butage-specificvariantsofthesameinstrumentcanbedevelopedwithwordingor

presentationsadaptedtoagegroups.Forexample,thePaediatricQualityofLifeInventory(PedsQL?)hasfourself-completedvariants;oneforchildrenages5–7,oneforages8–12,anotherforages13–18,anda“young

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adult”versionfor18-to25-year-olds.34Thecontentof

thedifferentvariantsisconceptuallythesame,with

developmentallyappropriatewording,andallvariantsarescoredonthesame(T-score)metric.Assuch,itis

plausibletocombinetheassessmentsacrossgroups

ofparticipantsrespondingtothedifferentvariants.1

Forlengthytrials,importantdevelopmentalchanges

couldunfoldduringthetrialasachildevolvesand

grow.Forexample,ifaparticipantisage7atbaselineandcompletestheages5–7versionofthePedsQL?,

atthefollow-upvisittheymaybeage9andeligibletocompletetheages8–12versionofthePedsQL?.Whileitmakessenseforachildtocompletetheage-appropriateversion,aslongasthefollow-upperiodiswithinone

ortwoyearsoftheoriginaladministrationandthe

instrumentisstillcontentvalidfortherespondent,itmaymakesensetokeepthesameversionforthechildthroughoutthetrialforoperationalefficiency.

Whenresearchcannotrelyonself-reportfromchildren–i.e.,whentheyareundertheageof5orhavelearning

difficultiesmitigatingtheirabilitytocompleteaPROinstrument–itmaystillbepossibletogeneratesomerelevantPEDinformationfromparents,clinicians,

teachersorothercaregivers5usingotherCOAs.In

thiscontext,itisimportanttodistinguishbetween

PEDreportedbytheparents,clinicians,teachers

orothercaregiversasiftheywerethepatient,and

PEDthatreflectsobservations.Theformerisknownas“proxyreporting.”Althoughnotuncommon(theaforementionedPedsQL?alsohasparentalproxy

itemsinreportversionsforages2–4,5–7,8–12and

13–18),thisisgenerallydiscouraged,22,35asitrequiresthattherespondentmakesinferencesaboutthe

patientsthemselvesandthismaynotbereflectiveofwhatthepatientmaybetrulythinkingorfeeling(i.e.,researcherscannotassumethatsomebodyelsewillbeabletoevaluatethechild’slevelofpain,theirlevelofsatisfaction,ortheiremoti