2023年罕見病行業(yè)調(diào)研報(bào)告-深度洞察行業(yè)外包需求（英文版）

RAREDISEASES

SURVEYRESULTS

1Introductionpg.2

2Demographicspg.7

3OutsourcingwithaContractResearchOrganization(CRO)pg.11

4PatientResources&DrugDevelopmentpg.17

5ClinicalTrialDetailspg.22

PART1

INTRODUCTION

RAREDISEASESSURVEY

2RareDiseasesSurvey

1

Introduction

SponsoredbyPPD,partofThermoFisherScientific

ThissurveywasconductedinApril2023

among100respondentswhoworkinsomecapacitywithintherarediseasesector

andwhocurrentlyoutsource(orplanto

outsourcewithinthenext2years)someoftheirresearchneedstoacontractresearchorganization(CRO).Thegoalofthestudywastoidentifyclients’needsandthe

challengestheyarefacingintermsofrarediseaseclinicaldevelopment.

3RareDiseasesSurvey

RESPONDENTEXPERIENCE

91%

Ofrespondentshave>10yearslifesciencesexperiences

RESPONDENTSENIORITY

87%

Ofrespondentsaredirectorlevelorabove

1

Introduction

Demographics

ThestudywasdesignedtobeglobalbutwithastrongbiastowardNorthAmerica-basedrespondents,whocomprise70%ofthe

audience;20%oftheaudiencearefromEurope,whiletheremaining10%areintheAsiaPacificregion.Thebulkoftheaudience(72%)

worksatabiopharmaceuticalcompany,while18%areemployedbyasmallmoleculepharmaceuticalcompany;theremaining10%areatR&Dlaboratories(6%)oracademicinstitutions/universities(4%).91%oftheaudiencenotedthattheyhaveatleast10yearsofexperiencewithinthelifesciencesector,and87%areinadirector-levelpositionorhigher,representinganapproachthatsupportshighrelevanceandstrongdataintegrity.

Bydesign,onlyrespondentswhosecompaniesororganizationsareworkingwithinraredisease,andwhosecompaniesororganizationsareeithercurrentlypartneringwithaCROorplantodosowithinthenext24months,wereabletocompletethesurvey.

4RareDiseasesSurvey

86%

Ofrespondentssaidthattheir

companiesarecurrentlyutilizingCROsaspartoftheirrarediseasedrugdevelopmentefforts.

55%

Ofrespondentswhose

companiesarecurrentlyoutsourcingworkforrarediseaseclinicaltrials

areusing3+CROs

44%

Ofrespondentsexpecttheir

outsourcingactivitytoincreaseoverthenext24months

1

Introduction

Trends

WhenaskediftheyarecurrentlypartneringwithaCROaspartof

theirrarediseasedrugdevelopmentplan,86%ofrespondentsnotedthattheywere:55%ofthatgroupofrespondentssaidtheywere

workingwithatleast3contractpartners.44%oftheserespondentsexpecttheiroutsourcingactivitytoincreaseovertheupcoming24months,while47%feltthattheirpartnershipswouldlikelyremainatthecurrentlevel.ThedecisiondriversthatwereidentifiedasbeingthemostimportantwhenselectingaCROtopartnerwithonrare

diseasestudieswere“Therapeuticexpertise”,“Directexperiencewithcell&genetherapies”,“Reliability”,“Agility/flexibility”,and“End-to-endservice”.

47%ofrespondentsreportedthattheircompaniesconductedan

oncology-focusedrarediseaseclinicaltrialwithinthemostrecent24months;GeneticDisorderswasthesecondhighesttherapyareaat42%,withNeurologyinthird(33%).Themostfrequentlyselectedchallengefacedbytheaudiencewhenconductingraredisease

clinicaltrialswas“Recruitingpatientsforthetrial”,whichwas

chosenby52%oftheparticipants.“Complexityoftheclinicaltrialrequirements”wasadistantsecondat16%.

Decentralizedclinicaltrialswithintherarediseasespacewereused

tovaryingdegreesbyrespondents’companies:11%reportedthat

afullydecentralizedmodelwasusedforatleastoneoftheirrare

diseaseclinicaltrialsoverthemostrecent12months,while57%

reportedthatatleastone“hybrid”clinicaltrial,involvingboth

traditionalanddecentralizedaspects,wasconductedoverthesameperiod.ECRFandeConsentsoftware,at44%and42%,respectively,werethemostuseddecentralizedelements.Respondentsreported

that“Coordinationofvisitsorassessmentsovertime”(64%)and“Transportationto&fromclinicaltrialsites”(51%)werethetopneedsforpatientsduringtheirin-personclinicaltrialvisits.

5RareDiseasesSurvey

52%

Ofrespondentsfeelthatpatient

recruitmentisthemostchallengingaspectofrarediseaseclinicaltrials

57%

Ofrespondentsreportedthattheircompanieshaveconductedhybridclinicaltrialsthatutilizesome

decentralizedelementsoverthemostrecent12months.

47%

Ofrespondents’companiesconductedanOncology-

focusedrarediseaseclinicaltrialinthepast24months.

1

Introduction

Anothersectionofthesurveywasdedicatedtoansweringan

assortmentofquestionsaroundtopicssuchaspatientadvocacygroups,naturalhistorystudies,patient&diseaseregistries,

tokenization,anddrugdevelopment.

?40%ofrespondentsnotedthatrelationshipbuildingbetween

theircompaniesandpatientadvocacygroupsarehandledbyan

internalunit;37%saidthatamixofinternalandexternalresourcesareutilizedtocreateandmaintaintheserelationships.

?Amongthestudyparticipantswhosaidthattheircompanies

utilizednaturalhistorystudies(79%oftheaudience),

“Developmentofclinicaloutcomeassessments”(57%)and

“Identifyingpatientpopulation”(56%)werenamedasthetoptwousesfortheresource.21%ofrespondentsclaimedthattheywereneitherusingnorconsideringusinganaturalhistorystudytobuildtheirclinicaldevelopmentprogram.

?Patient/diseaseregistriesappeartobeausefultoolamongthe

studyaudience,asonly9%ofrespondentssaidthattheyarenotconsideringtheuseofaregistry.Themostfrequentlygivenreasonforutilizingapatient/diseaseregistrywas“Clinicaltrialdesign/

identifykeysub-groups”(66%);thenexthighestreason,“Uncoverunmetneedsandmarketpotential”,wasselectedby40%of

respondents.

?Ofthenovelapproachestodrugdevelopmentthatwereincludedaspartofthestudy,“External/Syntheticcontrolarms”drewthemostinterestat46%;“Basket/buckettrials”weresecondwith

44%.Withnoneofthegivenoptionsbreakingthe50%mark

withparticipants,thismightrepresentanopportunitytobuildawarenessandknowledgeofthevarioustools.

6RareDiseasesSurvey

PART2

DEMOGRAPHICS

RAREDISEASESSURVEY

7RareDiseasesSurvey

2

Demographics

2.1SurveyParticipant’sCompany/OrganizationType

72%

18%

BiopharmaceuticalCompany

SmallMoleculePharmaceuticalCompany

2.2SurveyParticipant’sRegionalLocation

6%

ResearchandDevelopment(R&D)Laboratory

4%

UniversityorOtherAcademicInstitution

70%

20%10%

NorthAmerica

Europe

Asia

Pacific

8RareDiseasesSurvey

2

Demographics

2.3SurveyParticipant’sYearsofLifeScienceExperience

91%

Morethan10years

8%

Between5and10years

1%

Between3and5years

2.4SurveyParticipant’sJobTitle

49%38%

Executive(VP,President,CEO,CIO,CTO,COO,etc.)

Director/SeniorDirector

12%

Manager/SeniorManager

1%

ProjectManager

2.5SurveyParticipant’sDepartment/WorkArea

54%

ResearchandDevelopment(R&D)

15%

Business

Development

17%

Clinical

Operations/Management

5%

DrugProduction/Manufacturing

2%QualityAssurance/QualityControl2%Purchasing/Procurement2%ExternalManufacturing

1%SupplyChain1%RegulatoryAffairs

9RareDiseasesSurvey

2

Demographics

2.6SurveyParticipant’sCompany’sInvolvementwithRareDisease

Isyourcompanycurrentlydevelopingand/or

commercializingadrugortherapythatisusedin

thetreatmentofpatientswithararedisease?

100%

Yes

2.7SurveyParticipant’sRoleWhenDecidingon/WorkingwithOutsourcingPartners

21%ofrespondentssaidtheyarepartofateamthatestablishesthecriteriaforthecontractor

selectionprocess

70%ofrespondentssaidtheyarepartofthedecision-makinggroupthatconductsresearchinto/interviews/selectsapotentialpartnershipopportunity

70%21%9%

9%ofrespondentssaidtheyactasapoint-of-contactbetweenthecontractorandtheircompany

10RareDiseasesSurvey

PART3

OUTSOURCINGWITHACONTRACTRESEARCHORGANIZATION(CRO)

RAREDISEASESSURVEY

11RareDiseasesSurvey

3

OutsourcingwithaContractResearchOrganization(CRO)

3.1SurveyParticipant’sCompany’sSize

42%36%22%

Mid-sizedcompany

(501to2,500employees)

Smallcompany

(Upto500employees)

Largecompany

(2,500+employees)

3.2SurveyParticipant’sCompany’sRareDiseasePortfolioStatus

Mycompanyhasmultipletherapeuticsforraredisease(s)acrossvarious

stagesofdevelopment.

Mycompanyhasasingletherapeuticforraredisease(s)intheearlystagesofdevelopment.

Mycompanyhasmultipletherapeuticsforraredisease(s)intheearlystagesofdevelopment.

32%

34%

21%7%6%

Mycompanyhasmultipletherapeuticsforraredisease(s)inthelatestagesofdevelopment.

Mycompanyhasasingletherapeuticforraredisease(s)in thelatestagesofdevelopment.

12RareDiseasesSurvey

3

OutsourcingwithaContractResearchOrganization(CRO)

3.3PartneringwithaContractResearchOrganization(CRO)

IsyourcompanycurrentlyutilizinganyContractResearchOrganizations(CROs)aspartofyour

rarediseasedrug(s)development?

86%14%

YesNo

3.3aPartneringwithaCRO–NumberofContractPartners

WithhowmanyCROsareyoucurrentlyworking?

34%

23%

22%

21%

3-4CROs

2CROs

1CRO

5+CROs

NOTE:Totalnumberofrespondentsis86;therespondentswhoanswered“Yes”toQuestion3.3werepromptedtoanswerthis.

3.3bPartneringwithaCRO–NumberofContractPartners

WithhowmanyCROsdoesyourcompanyexpecttoworkwhenitdoesbegintooutsourceprojects?

43%

29%

21%

2CROs

1CRO

3-4CROs

7%

NOTE:Totalnumberofrespondentsis14;therespondentswhoanswered“No”toQuestion3.3werepromptedtoanswerthis.5+CROs

13RareDiseasesSurvey

3

OutsourcingwithaContractResearchOrganization(CRO)

3.3cCurrentOutsourcingActivity(CROs):ResearchNeeds

Approximatelywhatpercentageofyourresearchneedsisyourcompanycurrentlyoutsourcing?●ResearchNeeds●Respondents

Upto20%

13%

21%to40%

28%

41%to60%

24%

61%to80%

24%

81%to100%

10%

NOTE:Totalnumberofrespondentsis86;therespondentswhoanswered“Yes”toQuestion3.3werepromptedtoanswerthis.

3.3dPossibleOutsourcingActivity(CROs):ResearchNeeds

Approximatelywhatpercentageofyourresearchneedsdoyouexpectyourcompanytooutsource?●ResearchNeeds●Respondents

Upto20%

7%

21%to40%

21%

41%to60%

29%

61%to80%

21%

81%to100%

21%

NOTE:Totalnumberofrespondentsis14;therespondentswhoanswered“No”toQuestion3.3werepromptedtoanswerthis.

14RareDiseasesSurvey

3

OutsourcingwithaContractResearchOrganization(CRO)

3.3eFutureOutsourcingBehavior(CROs)

Whatdoyoubelieveyourcompany’soutsourcingactivitywithCROswillbelike24monthsfromnowcomparedtoitscurrentlevel?

47%44%

ItwillstayatthesamelevelItwillincrease

8%

Itwilldecrease

1%

Iamnotsure

NOTE:Totalnumberofrespondentsis86;therespondentswhoanswered“Yes”toQuestion3.3werepromptedtoanswerthis.

3.4DecisionDriver:SizeoftheContractResearchOrganization(CRO)

WhenconsideringapartnershipwithaCRO,doyouhaveapreferenceinthesizeofthecontractorganization?

55%

Yes

39%

No

6%

NotSure

3.4aPreferenceinCROSize

WhatsizeCROdoyouprefertoworkwith?

49%

Medium-sizedCRO

Large-sizedCRO

45%

5%

NOTE:Totalnumberofrespondentsis55;therespondentswhoanswered“Yes”toQuestion3.4werepromptedtoanswerthis.

Small-sizedCRO

15RareDiseasesSurvey

3

OutsourcingwithaContractResearchOrganization(CRO)

3.5DecisionDrivers:ChoosingaCROPartnerforRareDiseaseStudies

WhatfactorsappealtoyouwhenmakingyourchoiceforaCROpartnerforRareDiseasestudies?(Respondentswereaskedtoranktheirtop5decisiondriversfromthe11possibleresponses,withchoice#1beingthedrivertheyfeltwasmostimportant.Thelowerthenumber,themoreimportantrespondentsfeltthefactorwas.)

1.96

v2.94

Agility/flexibility

2.69

Therapeuticexpertise

2.83

Reliability

3.07

Directexperiencewithcellandgenetherapies

3.25

3.26

End-to-endserviceCostRelationshipswithclinicaltrialsites

3.84

Digitalsolutions/technicalcapabilities

3.94

Relationshipswithpatientadvocacygroups

3.94

Communication

3.97

Accessibility

16RareDiseasesSurvey

PART4

PATIENTRESOURCES

&DRUGDEVELOPMENT

RAREDISEASESSURVEY

17RareDiseasesSurvey

4

PatientResources&DrugDevelopment

4.1EstablishingRelationshipswithPatientAdvocacyGroups

Doesyourcompanybuildandmaintainitsownrelationshipswithpatientadvocacygroups,ordoesitrelyonthirdpartiestoestablishconnections?

●40%ofrespondentssaidtheircompanyhasaninternalunitthatestablishesrelationshipswithpatientadvocacygroups.

.23%ofrespondentssaidtheircompanylooksto

otherorganizationstobuildconnectionswithpatientadvocacygroups.

●37%ofrespondentssaidtheircompanyusesbothinternalandexternalresourcestocreateandmaintainrelationshipswith

patientadvocacygroups.

37%40%

23%

4.1aBenefitsofRelationshipswithPatientAdvocacyGroups

Whatbenefitshaveyouexperiencedthroughyourconnectionwithpatientadvocacygroups?

24%

Awarenessandeducationaboutrarediseases

12%

33%

First-handexperienceandfeedbackfrompatients

20%

Accesstopatientsforclinicaltrials

Patientrecruitment

6%

Transparency/agility/fasterresponses

2%

54%

Abetterunderstandingofpatients’needs

9%

8%

Accesstograntfunding

Bettercommunication

3%

Increaseinenrollmentrate

2%

Innovation/technologies

Teammotivation

10%

Collaborativeopportunities

5%

Supportandempowerment

NOTE:Totalnumberofrespondentsis100;multipleanswerswereallowed.

18RareDiseasesSurvey

4

PatientResources&DrugDevelopment

4.2UsingNaturalHistoryStudies

Whichofthefollowingusesofnaturalhistorystudiesareyouconsideringtoinformyourclinicaldevelopmentprogram?

(Multipleanswerswereallowed)

57%

Developmentofclinicaloutcomeassessments

56%

37%

35%

IdentifyingpatientpopulationIdentifyingordevelopingbiomarkers

Designingexternalcontrolstudies

21%

Wearenotconsideringanaturalhistorystudy

4.3UsingDiseaseorPatientRegistries

Inwhichofthefollowingareasareyouconsideringtheuseofadiseaseorpatientregistry?(Multipleanswerswereallowed)

35%

Pre-clinicalportfolioprioritization/

investmentdecisions

34%

Supportgo-to-marketactivities

40%

Uncoverunmetneedsandmarketpotential

35%

SupportregulatoryandHTAsubmissions

9%

Wearenotconsideringaregistry

1%

Other

66%

Clinicaltrialdesign/identifykeysub-groups

19RareDiseasesSurvey

4

PatientResources&DrugDevelopment

4.4NovelApproachestoDrugDevelopment

Whichofthefollowingnovelapproachestodrugdevelopmentareofinteresttoyourcompanyforuseinclinicaltrials?

(Multipleanswerswereallowed)

46%

44%

37%

35%

External/SyntheticcontrolarmsBasket/buckettrialsPlatformstudies

20%

UmbrellatrialsMasterobservationaltrials(MOTs)

2%

Other

4.5ClinicalTrialSiteLocations

Isyourcompanyopentoconductingclinicaltrialsinless-conventionalgeographicregionsatsitesthatare

(a)relativelynewtoclinicaltrialsbut(b)appropriatelysupportedforsuccess?

63%

20%

17%

Yes

No

NotSure

20RareDiseasesSurvey

4

PatientResources&DrugDevelopment

4.6ChallengesFacingtheRareDiseaseDrugDevelopmentSpace

Whatdoyouconsidertobethebiggestchallengesfacingrarediseasedrugdevelopmentintheupcoming10-yearperiod?

(Multipleanswerswereallowed)

62%

Accesstoappropriatepatientpools

41%37%

54%

Increasingcomplexityinclinicaltrialdesign

53%

Heterogeneityofpatientpopulationwithcertainrarediseases

59%

Expandedregulatoryrequirements

30%

Lowprevalenceofcondition

21%

Findingqualifiedstafftohandleincreased

technological

demands

2%

Lackofunderstandingofrarediseases’

naturalhistory

8%

Concernswithcosts

Other

Determiningresponsibilityinclinicaltrialgovernance

21RareDiseasesSurvey

PART5

CLINICALTRIALDETAILS

RAREDISEASESSURVEY

22RareDiseasesSurvey

5

ClinicalTrialDetails

5.1RareDiseaseClinicalTrialsbyTherapyArea

Acrosswhichtherapeuticareashasyourcompanyrunrarediseaseclinicaltrialsoverthemostrecent24months?(Multipleanswerswereallowed)

42%

Geneticdisorders

21%16%

HematologyInfectiousdisease

47%

31%

33%

Oncology

4%

8%

Non-malignantdisorders

2%

Musculoskeletal

disordersOphthalmology

6%

Other

Hormonaldisorders

4%

Neurology

13%

Respiratorydisease

Immunology

7%

Toxicology

23RareDiseasesSurvey

5

ClinicalTrialDetails

5.2PediatricPatientswithinRareDiseaseClinicalTrials

Approximatelywhatpercentageoftherarediseaseclinicaltrialsthatyouhaveconductedoverthemostrecent24monthsincludedpediatricpatients?

●RareDiseaseClinicalTrials●Respondents

0%

29%

Upto20%

33%

21%to40%

20%

41%to60%

6%

61%to80%

6%

81%to100%

6%

5.3ChallengesFacedwhenConductingRareDiseaseClinicalTrials

Fromthefollowinglist,whatdoyoufeelisthemostdifficultorchallengingaspectofrarediseaseclinicaltrials?

16%9%

Complexityoftheclinicaltrial

requirementsRegulatorybarriers

52%

8%6%

Lengthofthetrialstartupprocess

Identifyingadiversepatientpopulation

Recruitingpatientsforthetrial

1%1%

Developmentandaccesstopatientregistries

Educatingpatientsandtheirfamily/caregivers

5%2%

Collectionand

Patientretention

managementofdata

24RareDiseasesSurvey

5

ClinicalTrialDetails

5.3aHowCROPartnersCanHelpAddressChallengeswithRareDiseaseClinicalTrials

WhatactionswouldyouliketoseefromaCROpartnertohelpnavigatethroughthesechallenges?(Multipleanswerswereallowed)

24%

18%

Assistancewithpatientrecruitment/enrollment

Patientidentification/

maintainapatientdatabase

16%16%

Demonstrate

overallexpertise/

experience,guidance,andcommitment

Relationshipswith/accesstoregionalclinicaltrialsites

10%

9%

9%

8%

Greater/improved

Engagementwith

Assistancewith

Knowledgeof/access

communicationand

patientadvocacy

patientretention

withintherapeutic

listening

groupsandKOLs

andcompliance

areasofinterest

7%

5%

5%

4%

Greateradaptability/

Improvedtechnology

flexibility

andtechnical

Helpinimproving

andefficiency

expertise

Regulatorysupport

clinicaltrialdesign

1%

8%

Abilitytoconductdecentralized

clinicaltrials

Other

25RareDiseasesSurvey

5

ClinicalTrialDetails

5.4TypesofClinicalTrialsConductedforRareDiseaseTherapies

Whattypesofclinicaltrialshasyourcompanyconductedforrarediseasetherapiesduringthemostrecent12-monthperiod?

(Multipleanswerswereallowed)

57%

A“hybrid”clinicaltrialthatincorporated

atleastsomedecentralizedclinical

trialelements

48%

Atraditionalclinicaltrialthatisfullyconductedatadesignatedsite

11%

Afullydecentralizedclinicaltrial

(DCT)thatleveragesdigitaltoolsand

supportingservices,suchaseConsent,

eCOA,homehealthcare,etc.toenable

thepatienttocompletesomeorall

scheduledvisitsfromtheirhome

5.5UtilizingDecentralizedElementsinRareDiseaseClinicalTrials

Approximatelywhatpercentageoftherarediseaseclinicaltrialsthatwereconductedduringthisperiodutilizedremoteordecentralizedelements?

●RareDiseaseClinicalTrials●Respondents

0%

19%

Upto20%

31%

21%to40%

32%

41%to60%

12%

61%to80%

3%

81%to100%

3%

26RareDiseasesSurvey

5

ClinicalTrialDetails

5.5aTypesofDecentralizedElementsUsedinDecentralizedRareDiseaseClinicalTrials

Whichdecentralizedelementsdidyouutilizewhenconductingahybridorfullyremoterarediseaseclinicaltrial?(Multipleanswerswereallowed)

44%38%

Remotepatientmonitoring

42%

Electroniccasereportforms/eCRFsoftware

37%31%

Electronicinformedconsent/eConsentsoftware

26%

23%

Electronicclinical

Electronicpatient-

outcomesassessment

reportedoutcomes/

In-homenurse

(eCOA)

Telemedicineservices

eProsoftware

services/healthvisits

16%

14%

9%

9%

Remoteorsatellite

clinicaltrialsites

Wearabletechnologies

Pharmacy-basedclinics

Mobileclinics

27RareDiseasesSurvey

5

ClinicalTrialDetails

5.5bFuturePlansforReusingDecentralizedElements

Whichofthefollowingdecentralizedelements—thatwereutilizedwhenconductingahybridorfullyremoterarediseaseclinicaltrial—wouldyoubeinterestedinusingagain?

(Multipleanswerswereallowed)

Electronicclinicaloutcomesassessment(eCOA)

Electroniccasereportforms/eCRFsoftware

Electronicinformedconsent/eConsentsoftware

RemotepatientmonitoringTelemedicineservices

In-homenurseservices/healthvisits

Electronicpatient-reportedoutcomes/eProsoftware

WearabletechnologiesRemoteorsatelliteclinicaltrialsites

MobileclinicsPharmacy-basedclinicsOther

32%

31%

31%

26%

23%

17%

17%

11%

11%

4%

2%

0%

5.5cFuturePlansforTryingPreviouslyUnusedDecentralizedElements

Whichofthefollowingdecentralizedelements—thatwerenotutilizedwhenconductingahybridorfullyremoterarediseaseclinicaltrial—wouldyoubeinterestedinusinginfutureclinicaltrials?

(Multipleanswerswereallowed)

29%

23%

22%

22%

WearabletechnologiesRemotepatientmonitoringPharmacy-basedclinics

Mobileclinics

17%

16%

Electronicpatient-reportedoutcomes/eProsoftware

15%

14%

In-homenurseservices/healthvisitsRemoteorsatelliteclinicaltrialsitesTelemedicineservices

14%

12%

8%

Electroniccasereportforms/eCRFsoftware

Electronicinformedconsent/ eConsentsoftwareElectronicclinicaloutcomesassessment(eCOA)

2%

Other

28RareDiseasesSurvey

5

ClinicalTrialDetails

5.6PatientRequirementsforIn-PersonVisitsDuringClinicalTrials

Dopatientsinyourrarediseaseclinicaltrialsrequireanyofthefollowingservicesfortheirin-personclinicvisits?(Multipleanswerswereallowed)

35%

64%

Medicationreminders

31%29%

CoordinationofvisitsorDevice/softwareAssistancewith

assessmentsovertimetrainingmedicalequipment

51%28%28%

Assistancewith

reimbursementneeds

Transportationto/fromclinicaltrialsitesMobilityassistance

5.7AdditionalResourcesUsedtoIncrease/ShareKnowledgeaboutRareDisease

Whichofthefollowing