Methods for exploring and eliciting patient preferences in the medical product lifecycle: a literature review.

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feature

Methods

for

exploring

and

eliciting

patient

preferences

in

the

medical

product

lifecycle:

a

literature

review

VikasSoekhai1,7,8,§_,_Chiara_Whichello1,7,§_,_Bennett_Levitan2_,_Jorien_Veldwijk1,7_,_Cathy_Anne_Pinto3_,

BasDonkers4,7,IsabelleHuys5,ElinevanOverbeeke5,JuhaeriJuhaeri6and

EstherW.deBekker-Grob1,7,debekker-grob@eshpm.eur.nl

Preference

studies

are

becoming

increasingly

important

within

the

medical

product

decision-making

context.

Currently,

there

is

limited

understanding

of

the

range

of

methods

to

gain

insights

into

patient

preferences.

We

developed

a

compendium

and

taxonomy

of

preference

exploration

(qualitative)

and

elicitation

(quantitative)

methods

by

conducting

a

systematic

literature

review

to

identify

these

methods.

This

review

was

followed

by

analyzing

prior

preference

method

reviews,

to

cross-validate

our

results,

and

consulting

intercontinental

experts,

to

confirm

our

outcomes.

This

resulted

in

the

identification

of

32 unique

preference

methods.

The

developed

compendium

and

taxonomy

can

serve

as

an

important

resource

for

assessing

these

methods

and

helping

to

determine

which

are

most

appropriate

for

different

research

questions

at

varying

points

in

the

medical

product

lifecycle.

Introduction

Thereisanemergingconsensusthatthepatient perspective should be incorporated within decisions inthemedicalproductlifecycle(MPLC;seeGlossary)

[1–4],wherethemedicalproductlifecycleinthis studyisdefinedasthelifecyclesofdrugs,biologics andmedicaldevices.Broadlyencouragingthe in-volvementofpatientshas,therefore,become in-creasinglyimportant[5,6].Takingthepatientvoice intoconsiderationhasnot only becomeincreasingly importantforcompaniesthatdevelopnewmedical productsbutalsofortheauthoritiesthatassess, regulateanddecidewhichproductsareeffective, safe,well-toleratedandcost-effective[7–16].

Toincorporatethepatientvoice,patient preferencesneedtobeexplicitlyexploredor

elicitedthroughrevealed-orstated-preference methods.Inthispaper,preferenceexploration methodsaredefinedasqualitativemethodsthat collectdescriptivedatathroughparticipantor phenomenonobservation,examiningthe sub-jectiveexperiencesanddecisionsmadeby participants.Elicitationmethodsaredefinedas quantitativemethodscollectingquantifiable dataforhypothesistestingandotherstatistical analyses.Whereastheuseof revealed-prefer-encemethodsstillrepresentsamethodological challengeinhealth,manydifferentmethods existtoassessstatedpreferencesofpatients

[17,18].Anup-to-datecompendiumofdifferent stated-preferencemethodstoexploreorelicit patientpreferenceswithintheMPLCismissing.

Therehavebeenfewpublicationsonwhat methodscanbeusedtoassesspatient prefer-encesinascientificway,inthecontextofthe MPLCspecifically.In2001,Ryanetal.[19] pro-videdanoverviewofmethodsknownatthe timeforelicitingpublicpreferencesfor health-care.In2015,theMedicalDeviceInnovation Consortium(MDIC)developedanoverviewof differentpreferenceelicitationmethodsaspart oftheirframeworkonincorporationofpatient preferencesintoregulatoryassessmentsof medicaldevices[20].Althoughboth publica-tionsmadeusefulcontributions,thestudyfrom Ryanetal.[19]doesnotreflectmethods de-velopedsince2001,andthestudyfromthe MDIC[20]didnotincludepreference

explora-Features

PERSPECTIVE

PERSPECTIVE DrugDiscoveryTodayVolume24,Number7July2019

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tionmethodsoruseasystematicapproachfor identifyingpreferenceelicitationmethods. Therefore,theaimofourstudywastodevelop anup-to-datecompendiumandtaxonomyof explorationandelicitationpreferencemethods withintheMPLCcontext.Thiswillbean im-portantsteptofurtherdrivetheincorporationof patientpreferencesforward,inadditiontothe studyofvanOverbeekeetal.[6],andin de-velopingguidanceonwhenandhowtoassess patientpreferencesscientificallyinthecontext ofdecision-makingintheMPLC.

Compendiumofpreferencemethods

Asystematicliteraturereviewwasconducted, followedbyananalysisofpriorreviewsbyRyan etal.[19]andfromtheMDIC[20]andexpert consultationswithinternationalpreference experts,toidentifyallpotentialpreference ex-plorationandelicitationmethodswithinthe contextoftheMPLC.Inthispaper,abroad defi-nitionofapreferencemethodwasused:any methodthatenabledustogaininsightintoa patient’srelativedesirabilityoracceptabilityof specifiedalternatives;orchoicesamong treat-mentalternativesoroutcomes;orotherattributes thatdifferamongalternativehealthinterventions

[7].Ultimately,208paperswereanalyzedduring thesystematicliteraturereviewtoidentify pref-erence exploration and elicitation methods within thecontextoftheMPLC.Moreinformationabout theapproachusedinthesystematicliterature

reviewisprovidedinAppendixA.1(see Supple-mentarymaterialonline).Analphabetical over-viewofallreviewedfull-textpapersislistedin AppendixB(seesupplementarymaterialonline).

Weidentified19differentmethods:five ex-plorationmethodsand14elicitationmethods, inthesystematicliteraturereview.Themost frequentlycitedexplorationmethodsincluded focusgroups(n=29,13.9%)and (semi-)struc-turedindividualinterviews(n=47,22.6%), whereasmostcitedelicitationmethodpapers includeddiscretechoiceexperiments(n=57, 27.4%)andthevisualanalogscale(n=12,5.8%). Contingentvaluation(n=11,5.3%),standard gamble(n=11,5.3%)andtimetrade-off(n=11, 5.3%)werealsofrequentlyincludedinthe an-alyzedpapers.Fourstudiesincludedbest–worst scalingtype1,2(n=4,2%).

Throughtheanalysisofthepreference methodreviewsofRyanetal.[19]andtheMDIC

[20],andaftercondensingseveralofthese methods,weidentified23preference explora-tionandelicitationmethods.Thisselection in-cludedninepreferenceexplorationand14 elicitationmethods.Fromthese23preference methods,13methodswerealsoidentifiedinour systematicliteraturereview(56%).Theexpert consultationsconfirmedthemethodsidentified inthesystematicliteraturereviewandinthe analysisofpriorpreferencemethodreviews. Also,consensuswasreachedonincludingfour additionalelicitationmethods.Theexpert

con-sultationsalsoresultedintheexclusionof methodsfocusingonscale-related(e.g.,Likert scales)ordecision-makingframework-related(e. g.,multicriteriadecisionanalysis)techniques, becausethesetechniqueswereregardedas inconsistentwithourdefinitionofapreference method.Asdescribedabove,weidentified19 methodsthroughthesystematicliterature re-view,the23methodsthroughtheanalysisof previouslyconductedreviewsandthefour ad-ditionalmethodsviaexpertconsultations.In total,32uniquepreferencemethodswere identified:tenexplorationand22elicitation methods.Table1 summarizesandbriefly describesthesemethods.

Taxonomyofpreferencemethods

Therearemanywaystogrouppreference methods.Inthisstudy,wegroupedthe identi-fiedmethodsaccordingtotheirmannerofdata collectionandthesimilaritiesintheirmethodof analysis.Thisgroupingwasnotintendedtobea formallexiconbutprimarilyservedasa taxon-omytoorganizeresultsandtodevelopa compendiumofpreferenceexplorationand elicitationmethods.Preferenceexploration methodscanbegroupedaccordingtothe numberofparticipantsthemethodutilizesin onesession(Fig.1).(Semi-)structuredindividual interviews,in-depthinterviewsandcomplaints proceduresuseinterviewswithoneparticipant (n=1)inasinglesettingorsession.TheDelphi

TABLE1

Overviewofidentifiedmethods

Method Description Refs

Explorationmethods

Citizens’juriesb Groupofindividualsdiscussingissuesonthebasisofevidenceprovidedbytwotrainedmoderators [24,25]

Complaints

proceduresb

Methodinwhichstakeholderscanregistercomplaintstobeinvestigatedbyexperts [26,27]

Conceptmappingb _Method_that_utilizes_small_groups_of_participants_responding_to_various_topics_or_issues,_while_ensuring_each

respondentisgivenequalopportunitytoexpresstheiropinionsandaddressothergroupdynamicissues

[28,29]

Delphimethoda,b _Structured,_iterative_forecasting_method_involving_a_panel_of_experts_who_provide_anonymous_responses_to

questionnaireswiththeopportunitytorevisetheirresponseswhentheanonymoussummaryofresponse

fromthepriorroundisrevealed

[30,31]

Dyadicinterviewa,b _Method_that_utilizes_two_participants_in_a_single_interview,_responding_to_open-ended_questions_asked_by_an

interviewertoidentifyhowaproduct,serviceoropportunityisperceived

[32,33]

Focusgroupa,b Methodthatutilizesagroupofinteractingindividualsthatprovideinformationaboutaspecificissueto

identifyhowaproduct,serviceoropportunityisperceived

[34,35]

Indepth–individual

interviewa,b

Interviewtechniquethatallowsforanintensivediscussionwithoneintervieweetoexploretheirperspectives

onaparticulartopicortheme,togainadeeperunderstandingofthisparticulartopicortheme.Oftenonlya

limitedamountofquestionsorthemesarepreparedbytheinterviewer,andtherestofthequestionsare

basedontheresponseoftheinterviewee

[36,37]

Nominalgroup

techniqueb

Methodthatutilizesagroupprocessthatinvolvesmakingdecisionsbyvoteandrankingresponsesgivenby

membersofthegroup

[38,39]

Publicmeetingsb _Method_to_gain_public_opinions_on_particular_issues_by_allowing_general_members_of_the_public_to_attend_and

voicetheirresponses

[40,41]

(Semi-)structured

individualinterviewa,b

Interviewtechniquethatallowsnewideastobebroughtupduringtheinterviewasaresultofwhatthe

intervieweesaysinasemi-structuredsetting,whereasinthestructuredsettingtheinterviewerstrictlysticks

toaninterviewguideanddoesnotaskquestionsbasedontheresponseoftheinterviewee

[42,43]

Features

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TABLE1(Continued)

Method Description Refs

Elicitationmethods

Adaptiveconjoint

analysisa

Methodsimilartoregularconjointanalysis,butwithadaptiveconjointchoicetasksbasedontheearlier

choicesmadewithinthesurvey,intheoryallowingthesurveytofocusattentiononthoseattributesorlevels

ofthoseattributesthathavethemostinfluenceonthechoicesofthatindividual.Unlikediscretechoice

experimentsthismethodisfoundedinthetheoryofconjointmeasurement(CM),whichismorefocusedon

thebehaviorofnumbersystemsinsteadofthebehaviorofhumanpreferences

[44,45,81]

Allocationofpointsb _Method_that_involves_asking_respondents_to_rate_their_conditions_on_scales,_while_knowing_the_weights_which

theyattachtodifferentcriteria,indicatingtherelativeimportanceofparticularareasoftheirlives

[46,47]

Analytichierarchy

processa,b

Methodinwhichrespondersassesstherelativeimportanceofpairsofattributes(treatmentendpoints,

properties,criteria,items,objects,etc.)towardachievingagoal,wheretheseresponsesareusedtocompute

aweightforeachattribute

[20,48]

Best–worstscaling

(types1,2,3)a,b

Involvesrespondentsansweringsurveysthatincludelistsofattributesorprofilesandbeingaskedtoindicate

thebest(ormostappealing/important)andtheworst(orleastappealing/important)ofthem.Thismethod

consistsofthreetypes:intype1asetofattributesisshowedthatmightnotreflectthecharacteristicsofany

particulartreatment,ofwhichtherespondentpicksthebestandworst.Type2involvesasituationinwhich

theattributescollectivelycharacterizeaparticularprofileandtherespondentchoosesthebestandworst.In

type3threeormoreprofilesareshownandtherespondentselectsthebestandworstprofiles

[20,49,50]

Constantsumscalingc Constantsumscalingconsistsofacomparativescalewhererespondentsareaskedtoallocateafixedamount

(orconstantsum)ofpoints,dollarsoranythingamongasetofobjectsaccordingtoacriterion

[51,52]

Contingentvaluationa,b Methodtodeterminethewillingnesstopay(WTP),whereindividualsarepresentedwithachoicebetween

nothavingthecommodityvaluedandhavingthecommoditybutforgoingacertainamountofmoney.The

moneybeingthattheyarewillingtoforgotohavethecommodityistheirWTPforthatcommodity.WTPcan

becalculateddirectlyusingathresholdorindirectlyusingadiscretechoiceexperimentforexample

[53,54]

Controlpreference

scalea

Thecontrolpreferencesscale(CPS)isamethodtodeterminethedegreeofcontrolapatientwantsregarding

medicaltreatment.Thepreferenceordersareanalyzedusingunfoldingtheorytodeterminethedistribution

ofpreferencesindifferentpopulationsandtheeffectofcovariatesonconsumerpreferences

[55_–57]

Discretechoice

experimenta,b

Methodthatutilizesanattribute-basedmeasureofbenefit,duringwhichindividualsareofferedaseriesof

hypotheticalchoicesituations(i.e.,choicesets),fromwhichtheyareaskedtochoosebetweentwoormore

profiles.Therearenumerousvariantsofdiscretechoiceexperiments.Incontrasttoconjointanalysis,this

methodreliesonatheoryofthebehaviorofhumanpreferences[forexamplerandomutilitytheory(RUM)]

[58,59,60,81]

Measureofvalueb _Method_used_to_identify_the_optimal_bundle_of_services_to_be_provided_given_resource_constraints._Individuals

areaskedtoallocateafixedamountofresourcesbetweendifferentservices.Theseallocationsareanalyzed

toidentifythetrade-offsindividualsmake

[61]

Outcomeprioritization

toola

Instrumentthatallowsparticipantstoprioritizeoutcomesmakinguseofaspecifictoolaccordingtothe

‘trade-off’principle,implyingthattheyarewillingtocompromiseonthelessimportantoutcomes

[62]

Persontrade-offa,b Anextensionofthetimetrade-off.Withpersontrade-offanindividualevaluatesthehealtheffectsof

interventionsusingpersons(insteadoftime)astheequilibratingmechanism

[63,64]

(Probabilistic)

thresholdtechniquea,b

Methodthatdeterminesthemaximalchangeinoneattributerespondentsarewillingtoaccepttoachievea

givenchangeinanotherattribute

[20,65]

Q-methodologyc Methodthatusesaspeciallydesignedresponsegridtopresentrespondentswithasetofstatementsand

askingthemtoorder,usuallybasedontheextenttowhichtheyagreewiththem

[66,67]

Qualitative

discriminantprocessb

Methodthatinvolvesascoringandrankingprocessbasedondecisionanalysistechnique,involvingthe

definitionofoptionsintermsofqualitativecategories,thenderivinganumericpointestimateandfinally

solvingamaximizationproblemwithgivenconstraints

[68]

Repertorygrid

methoda

Methodusedforelicitingpersonalconstructs(i.e.,whatpeoplethinkaboutagiventopic).Toidentify

preferencesoverlappingandratingtechniquesareused

[69,70]

Self-explicated

conjointc

Methodthatasksexplicitlyaboutthepreferenceforeachattributeratherthanthepreferenceofseveral [71]

Standardgamblea,b _Method_in_which_respondents_are_asked_to_choose_between_a_certain_outcome_and_a_gamble_that_might

resultineitherabetteroutcomewithaprobabilityPoraworseoutcomethantheoriginalwithaprobability

1-P

[72,73]

Startingknown

efficacya

Methodsimilarto(probabilistic)thresholdtechniques,butwithaspecificknownstartingpoint.Thismethod

isspecificallyusedwithinthecontextofthemedicalproductlifecycle

[74]

Swingweightingb Methodforsettingtheweightsinwhichadecision-relevantrangeisspecifiedforeachattribute,andthe

impactof‘swinging’theattributethroughthatentirerangeofvaluesisassignedaweightrelativetothe

impactofswingingtheattributewiththelargestweight

[19,20]

Testtrade-offc Methodthatcanberegardedasanextensionofthetimetrade-offthatisspecificallyusedtoevaluateanew

biomarkerbyusingrisks(insteadoftime)astheequilibratingmechanism

[75,76]

Timetrade-offa,b _Method_that_presents_individuals_with_a_choice_between_living_for_a_period_in_a_specified,_but_less_than

perfect,stateversushavingahealthierlifeforaperiodoftime,wheretimeisvarieduntiltherespondentis

indifferenttothealternatives

[20,77,78]

Visualanalogscalea,b _A_{self-reporting}_instrument_consisting_of_a_line_of_{predetermined}_length_that_separates_extreme_boundaries

ofthephenomenonbeingmeasured

[79,80]

Intotal32uniquemethodswereidentified.

a

Identifiedinsystematicreview(19methods).

b

Identifiedthroughanalysisofpreviouspreferencemethodreviews(23methods).

c

Identifiedwithexpertconsultations(4methods).

Features

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Preference

exploration

Individual methods

Group methods

Individual/group

methods

In-depth individual interview (Semi-)structured

individual interview Delphi method Public meetings

Concept mapping

Focus group

Dyadic interview

Nominal group technique

Complaints procedures Citizens’ juries

Drug Discovery Today

FIGURE1

Groupingofpreferenceexploration(qualitative)methodsintothreegroups:individual,groupandindividual/groupmethods.

Discrete choice experiment/best-worst scaling type 3 Best-worst scaling Type 1,2 Adaptive conjoint analysis Self-explicated conjoint Analytic hierarchy process Measure of value Allocation of points

Starting known efficacy Time trade-off Standard gamble

Person trade-off

Visual analog scale Constant sum scaling

Qualitative discriminant process

Repertory grid method

(Probabilistic) threshold technique Swing weighting Outcome prioritization tool Contingent valuation Control preferences scale Q-methodology Test trade-off

Preference

elicitation

Discrete-choice-based methods Ranking methods Indifference_methods Rating methods

Drug Discovery Today

FIGURE2

Groupingofpreferenceelicitation(quantitative)methodsintofourgroups:discretechoicebased,ranking,indifferenceandratingmethods.

Features

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method,focusgroups,dyadicinterviews,public meetings,nominalgrouptechniqueandcitizen juriestypicallydirectquestionstomorethan oneparticipant(n>1)inasinglesetting. Con-ceptmappingcanemployeitherindividualor groupsettingsfordatacollection(n1).

Preferenceelicitationmethodscanbe groupedintofourdistinctgroups(Fig.2),with methodsfromlefttorightbeingabletoanswer asmallersubsetofresearchquestions[adiscrete choiceexperiment(DCE)isforexampleableto providewillingness-to-pay(WTP)information andprobabilityscoreswhereascontingent val-uationprovidesWTPinformationonly].First, discretechoice-basedmethodstypically exam-inetheimportanceoftrade-offsbetween attri-butesandtheiralternativesthroughaseriesof

choicesetsthatpresent(hypothetical) alterna-tives.Second,ranking(orrelated)methodswere classifiedbasedontheuseofrankingexercises tocapturetheorderofalternativesorattributes withinapresentedset.Third,indifference techniquesaremethodsthatvarythevalueof oneattributeinoneofthealternativesuntilthe participantisindifferent,orhasnopreference, betweenalternatives.Finally,rating(orrelated) methodsaremethodsbasedontheirutilization ofcomparativeratingapproaches,often allow-ingparticipantstoexpressthestrengthoftheir preferencesalongalabeledscale.

Trendsintheuseofpreferencemethods

Withthesystematicliteraturereview,spanning 37yearsofliterature,weobservedanoverall

upwardstrendinthenumberofMPLCpatient preferencestudiesperyear.Themeannumber ofpreferencestudiesincreasedfrom1.1peryear to6.5peryearto20.3peryear.Thisisforthe periods1980–2000,2001–2010and2011–2016, respectively(AppendixC,seesupplementary materialonline).Wealsoobservedthatour includedpapersoriginatedfromalloverthe world,coveringfivedifferentcontinents(Table2

).Themajority(73%)ofpaperswerefromNorth America(n=90)andEurope(n=62).

Analyzingtheseparateuseofpreference explorationandelicitationmethodsovertime, weobservedatrendofpreferenceexploration methodsbeingusedmorefrequentlyinrecent years.Wedidnotconsidertheperiod1980– 2005becausethisperiodonlyincludedafew

TABLE2

Backgroundinformationofidentifiedpatientpreferencemethodsinthesystematicreviewfocusingonthemedicalproductlifecycle

Method Frequency Continentsoforigin Studynumbers

n=19 n=208a (%) Continents(frequency)a n=208

Explorationmethods

Delphimethod 3 (1.4) Asia(2),NorthAmerica(1) 24,107,308

Dyadicinterview 1 (0.5) Africa(1) 269

Focusgroup 29 (13.9) Africa(1),Asia(2),Australia/Oceania

(3),Europe(15),NorthAmerica(8)

2,14,17,18,43,45,71,72,84,97,109,116,119,121, 211,220,222,236,253,269,282,283,286,290,294, 300,308,313,317 Indepth–individual interview 9 (4.3) Asia(1),Australia/Oceania(1),

Europe(3),NorthAmerica(4)

32,41,108,147,173,191,193,211,316

(Semi-)structured

individualinterview

47 (22.6) Africa(2),Asia(6),Australia/Oceania

2,9,17,18,21,30,41,43,57,58,65,67,87,94,100,101, 120,129,141,153,162,164,184,193,198,205,211, 215,217,222,226,229,230,232,239,267,268,269, 272,280,284,285,286,302,306,310,323 Elicitationmethods Adaptiveconjoint analysis 3 (1.4) NorthAmerica(3) 88,89,243 Analytichierarchy process 1 (0.5) Europe(1) 221

Best-worstscaling(types

1,2,3)

4 (1.9) Asia(1),Australia/Oceania(1),North

America(2)

133,180,189,300

Contingentvaluation 11 (5.3) Asia(2),Australia/Oceania(1),North

America(2)

29,35,144,148,155,166,167,180,199,244,298

Controlpreferencescale 3 (1.4) Asia(1),NorthAmerica(2) 147,175,316

Discretechoice

experiment

57 (27.4) Africa(1),Asia(7),Australia/Oceania

19,25,26,34,42,48,57,66,73,79,80,90,100,101, 109,114,117,119,122,133,134,154,155,160,161, 163,166,179,180,184,192,194,200,212,213,215, 218,219,222,227,229,234,238,239,243,246,247, 249,257,264,266,272,281,309,311,312,313 Outcomeprioritization tool 1 (0.5) Europe(1) 304

Persontrade-off 1 (0.5) Europe(1) 274

Repertorygridmethod 1 (0.5) Europe(1) 255

Standardgamble 11 (5.3) Asia(1),Australia/Oceania(1),

Europe(2),NorthAmerica(7)

34,42,155,180,195,200,209,219,237,277,312

Startingknownefficacy 1 (0.5) NorthAmerica(1) 201

(Probabilistic)threshold

technique

2 (1.0) NorthAmerica(2) 42,172

Timetrade-off 11 (5.3) Australia/Oceania(1),Europe(2),

NorthAmerica(8)

33,34,78,155,180,200,209,219,237,277,318

Visualanalogscale 12 (5.8) Asia(2),Europe(3),NorthAmerica

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93,115,168,171,178,195,208,223,278,281,287,314

a

Includedcountriespercontinent:Africa–Kenya,SouthAfrica;Asia–China,Iran,Japan,Malaysia,Singapore,SouthKorea,Taiwan,Thailand,Turkey;Australia/Oceania–Australia;Europe

–France,Germany,UK,Hungary,TheNetherlands,Norway,Spain;NorthAmerica–Canada,USA.

Features

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datapointstocomputerepresentative percen-tages.Fortheperiod2002–2006,33.3%ofthe papersusedapreferenceexplorationmethodto gaininsightsintopatientpreferences (com-putedasthefrequencyofanexplorationor elicitationmethodineachindividualpaper). Thisincreasedto48.8%intheperiod2007–2011 andto45.8%for2012–2016.Amongpreference explorationmethods,theproportionofstudies thatusedfocusgroupsincreasedfrom23%in theperiod2002–2006to35%intheperiod 2012–2016.Theproportionof(semi-)structured individualinterviewsremainedmoreorless constantwith55%intheperiod2002–2006and 52%intheperiod2012–2016,whereasin-depth individualinterviewsdecreasedfrom23%in 2002–2006to8%in2012–2016.Overtime,we alsoobservedmorediversitywithinthegroupof preferenceexplorationmethods.TheDelphi methodanddyadicinterviewsbeganappearing in2007.

Amongpreferenceelicitationmethods,we observedthatthenumberofpapersthatmade useofadiscretechoiceexperimentincreased from38%in2002–2006to58%in2012–2016. Papersthatincludedavisualanalogscale de-creasedfrom16%to3%,andcontingent valu-ationshowedasimilartrend(17%to9%). Standardgambleandtimetrade-offshowedan upwardtrend,from5%and4%in2002–2006to 9%and6%in2012–2016,respectively.Overall, weobservedthat,overtime,amorediverse groupofpreferenceelicitationmethodswas used.

Comparisonofsources

Theresultsofthisstudywerepartlyinlinewith theresultsfoundbyRyanetal.(2001)andthe MDIC(2015)[19,20].Fifty-sixpercent(13outof 23)ofmethodsreportedbyRyanetal.[19]and/ ortheMDIC[20]wereidentifiedinour sys-tematicliteraturereview.Thedifferencesarise because:(i)thesearchinthisstudyfocused specificallyonmethodstoobtainpatient pre-ferencesfordrugsandmedicaldevices,whereas Ryanetal.[19]focusedonpublicviewsonthe provisionofhealthcare;(ii)MDIC[20]excluded preferenceexplorationmethods;and(iii)the MDIC[20]effortdidnotuseasystematic ap-proachforidentifyingmethods.Thetaxonomy ofpreferencemethodsproposedinthisstudyis alsoinlinewithresultsfromMt-Isaetal.[21], Zhangetal.[22]andGonzalezetal.[23],in whichelicitationmethodsweregroupedby rating,rankingandtrade-off(whichincluded choice-basedmethods)techniques,although manyotherwaystogroupthesemethodsare possible.

Resultsfromourstudy’ssystematicliterature review(19preferencemethodsidentified) showedthatmostreviewedpapersusedfocus groups,(semi-)structuredindividualinterviews, discretechoiceexperimentsorthevisualanalog scaletogaininsightsintopatientpreferences. MostofthesestudieswereconductedinNorth AmericaorEurope.Wealsoshowedthatthe meannumberofpatientpreferencestudiesfor drugsandmedicaldevicesincreasedovertime. Furthermore,thisstudyshowedthat,for pref-erenceexplorationandelicitationmethods,a morediversemixofmethods(explorationand elicitationmethods)wasusedovertimeto exploreorelicitpreferences.

Concludingremarks

Inthisstudywedevelopedanup-to-date compendiumandtaxonomyofpreference ex-plorationandelicitationmethodsinthecontext oftheMPLC.Thesystematicreview(19 meth-ods),analysisofpriorconductedpreference methodreviews(23methods)andexpert con-sultations(fourmethods)contributedtothis compendium.Intotal,32uniquemethodswere identified.Preferenceexplorationmethodswere groupedinthreemaingroups,whereasthe preferenceelicitationmethodsweregroupedin fourmaingroups.Becausechoosingwhich methodtousewilldependontheMPLCphase andwhatthemeasuredpreferencesarebeing usedfor,futureresearchmightfocuson deter-miningwhichmethodsaremostappropriateto exploreorelicitpatientpreferences,andunder whatcircumstances,throughoutthedifferent phasesintheMPLC.Inaddition,itmightbeof interestforfutureresearchtofocusonthe specificcombinationsofpreferenceexploration andelicitationmethodsusedinmixed-method studies,andthereasoningbehindsuchstudy designs.

Acknowledgments

TheauthorswouldliketothankJ.Bridges(Johns HopkinsBloombergSchoolofPublicHealth),B. Craig(InternationalAcademyofHealth PreferenceResearch),J.M.Gonzalez(Duke University),T.Hammad(EMDSerono),B.Hauber (RTIHealthSolutions),R.Hermann

(AstraZeneca),R.Johnson(DukeUniversity),U. Kihlbom(UppsalaUniversity),A.Mohamed (Bayer),V.Patadia(Sanofi),S.Russo(European InstituteofOncology)andA.Stiggelbout (LeidenUniversityMedicalCenter)fortheir valuableinputduringthewritingofthispaper. ThisworkhasreceivedsupportfromtheEU/ EFPIAInnovativeMedicinesInitiative[2]Joint UndertakingPREFERgrantno.115966.Thistext

anditscontentsreflecttheauthors’andthe PREFERproject’sviewandnottheviewofIMI, theEuropeanUnionorEFPIA.

AppendicesA–C. Supplementarydata

Supplementarymaterialrelatedtothisarticle canbefound,intheonlineversion,atdoi:

https://doi.org/10.1016/j.drudis.2019.05.001.

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VikasSoekhai1,7,8,§

ChiaraWhichello1,7,§

BennettLevitan2 JorienVeldwijk1,7

CathyAnnePinto3

BasDonkers4,7 IsabelleHuys5 ElinevanOverbeeke5

JuhaeriJuhaeri6

EstherW.deBekker-Grob1,7,*

1

ErasmusSchoolofHealthPolicy&Management, Erasmus University Rotterdam, P.O. Box 1738, Rotterdam,3000DRtheNetherlands

2

JanssenResearch&Development,1125 Trenton-Harbourton Road, PO Box 200, Titusville, NJ, 08560,USA

3_Merck,_Sharpe_&_Dome,₂₀₀₀_Galloping_Hill_Rd.,

Kenilworth,NJ,07033,USA

4_Department_of_Business_Economics,_Erasmus

Uni-versityRotterdam,P.O.Box1738,Rotterdam,3000 DRtheNetherlands

5_Clinical _Pharmacology _and _{Pharmacotherapy,}

UniversityofLeuven,Herestraat49Box521, Leu-ven,3000Belgium

6

Sanofi, 55 Corporate Drive Bridgewater, NJ, 08807,USA

7

ErasmusChoiceModellingCentre,Erasmus Uni-versityRotterdam,P.O.Box1738,Rotterdam,3000 DRtheNetherlands

8

Department of Public Health, Erasmus MC – University Medical Centre, Dr. Molewaterplein 40,3000CARotterdam

*Correspondingauthor.

§_Joint_first_author.

GLOSSARY

Medicalproductlifecycle(MPLC)termto describethelifecyclesofdrugsandmedical devices

Patientpreferencesrelativedesirabilityor acceptabilityofspecifiedalternatives;or choicesamongtreatmentalternativesor outcomes;orotherattributesthatdiffer amongalternativehealthinterventions[7]

Preferenceelicitationmethod quantitativemethodscollecting quantifiabledataforhypothesistesting andotherstatisticalanalyses

Preferenceexplorationmethod

qualitativemethodsthatcollectdescriptive datathroughparticipantorphenomenon observation,examiningthesubjective experiencesanddecisionsmadeby participants

Quality-adjustedlifeyear(QALY)a measureofthestateofhealthofaperson orgroupinwhichthebenefits,intermsof lengthoflife,areadjustedtoreflectthe qualityoflife[77]

Features