2023年罕见病行业调研报告深度洞察行业外包需求英文版PPD324mb

RAREDISEASESSURVEY

RESULTSIntroductionpg.

2Demographicspg.

7OutsourcingwithaContractResearchOrganization(CRO)pg.

11PatientResources&DrugDevelopmentpg.

17ClinicalTrialDetailspg.

22PART1INTRODUCTIONRAREDISEASES

SURVEY2RareDiseases

Survey

Introduction1SponsoredbyPPD,partofThermoFisher

Scientific3RareDiseases

Survey

ThissurveywasconductedinApril2023among100respondentswhoworkin

somecapacitywithintheraredisease

sectorandwhocurrentlyoutsource(orplantooutsourcewithinthenext2years)some

oftheirresearchneedstoacontract

researchorganization(CRO).Thegoalofthestudywastoidentifyclients’needsandthechallengestheyarefacingintermsofrarediseaseclinical

development.Introduction1DemographicsThestudywasdesignedtobeglobalbutwithastrongbiastowardNorthAmerica-basedrespondents,whocomprise70%oftheaudience;

20%

of

the

audience

are

from

Europe,

while

the

remaining10%areintheAsiaPacificregion.Thebulkoftheaudience(72%)worksatabiopharmaceuticalcompany,while18%areemployedbyasmallmoleculepharmaceuticalcompany;theremaining10%areatR&D

laboratories

(6%)

or

academic

institutions/universities(4%).

91%oftheaudiencenotedthattheyhaveatleast10yearsofexperiencewithin

the

life

science

sector,

and

87%

are

in

a

director-level

positionor

higher,

representing

an

approach

that

supports

high

relevance

andstrongdata

integrity.Bydesign,onlyrespondentswhosecompaniesororganizationsareworkingwithinraredisease,andwhosecompaniesororganizationsare

either

currently

partnering

with

a

CRO

or

plan

to

do

so

within

thenext

24

months,

were

able

to

complete

the

survey.RESPONDENTEXPERIENCE91%Ofrespondentshave>10

yearslifesciences

experiencesRESPONDENTSENIORITY87%Ofrespondentsare

directorlevelor

above4RareDiseases

Survey

Introduction1TrendsWhenaskediftheyarecurrentlypartneringwithaCROaspartoftheir

rare

disease

drug

development

plan,

86%

of

respondents

notedthattheywere:55%ofthatgroupofrespondentssaidtheywereworking

with

at

least3

contract

partners.44%

of

these

respondentsexpecttheiroutsourcingactivitytoincreaseovertheupcoming24months,while

47%

felt

that

their

partnerships

would

likely

remain

atthecurrentlevel.ThedecisiondriversthatwereidentifiedasbeingthemostimportantwhenselectingaCROtopartnerwithonrarediseasestudieswere“Therapeuticexpertise”,“Directexperiencewithcell&genetherapies”,“Reliability”,“Agility/flexibility”,and“End-to-end

service”.47%ofrespondentsreportedthattheircompaniesconductedanoncology-focusedrarediseaseclinicaltrialwithinthemostrecent24months;GeneticDisorderswasthesecondhighesttherapyareaat

42%,

with

Neurology

in

third(33%).

The

most

frequently

selectedchallengefacedbytheaudiencewhenconductingrarediseaseclinicaltrialswas“Recruitingpatientsforthetrial”,whichwaschosenby52%oftheparticipants.“Complexityoftheclinicaltrialrequirements”

was

a

distant

second

at

16%.Decentralized

clinical

trials

within

the

rare

disease

space

were

usedto

varying

degrees

by

respondents’

companies:

11%

reported

thatafullydecentralizedmodelwasusedforatleastoneoftheirrarediseaseclinicaltrialsoverthemostrecent12months,while57%reportedthatatleastone“hybrid”clinicaltrial,involvingbothtraditionalanddecentralizedaspects,wasconductedoverthe

sameperiod.ECRFandeConsentsoftware,at44%and42%,respectively,werethemostuseddecentralizedelements.Respondentsreportedthat“Coordinationofvisitsorassessmentsovertime”(64%)and“Transportationto&fromclinicaltrialsites”(51%)werethetopneeds

for

patients

during

their

in-person

clinical

trial

visits.44%Ofrespondentsexpecttheiroutsourcingactivityto

increaseoverthenext24

months55%Ofrespondentswhosecompaniesarecurrentlyoutsourcingworkfor

rarediseaseclinical

trialsareusing3+

CROs86%OfrespondentssaidthattheircompaniesarecurrentlyutilizingCROsaspartof

theirrarediseasedrugdevelopment

efforts.5RareDiseases

Survey

Introduction1Anothersectionofthesurveywasdedicatedtoansweringanassortment

of

questions

around

topics

such

as

patient

advocacygroups,naturalhistorystudies,patient&diseaseregistries,tokenization,anddrug

development.40%ofrespondentsnotedthatrelationshipbuildingbetweentheircompaniesandpatientadvocacygroupsarehandledbyaninternalunit;37%saidthatamixofinternalandexternal

resourcesareutilizedtocreateandmaintainthese

relationships.Amongthestudyparticipantswhosaidthattheircompaniesutilizednaturalhistorystudies(79%oftheaudience),“Developmentofclinicaloutcomeassessments”(57%)and“Identifyingpatientpopulation”(56%)werenamedasthetop

twousesfortheresource.21%ofrespondentsclaimedthattheywereneitherusingnorconsideringusinganaturalhistorystudyto

buildtheirclinicaldevelopment

program.Patient/diseaseregistriesappeartobeausefultoolamongthestudyaudience,asonly9%ofrespondentssaidthattheyarenotconsideringtheuseofaregistry.Themostfrequentlygiven

reasonforutilizingapatient/diseaseregistrywas“Clinicaltrialdesign/identifykeysub-groups”(66%);thenexthighestreason,“Uncoverunmetneedsandmarketpotential”,wasselectedby40%ofrespondents.Ofthenovelapproachestodrugdevelopmentthatwere

includedaspartofthestudy,“External/Syntheticcontrolarms”drewthemostinterestat46%;“Basket/buckettrials”weresecondwith44%.

With

none

of

the

given

options

breaking

the

50%

markwithparticipants,thismightrepresentanopportunityto

buildawarenessandknowledgeofthevarious

tools.52%Ofrespondentsfeelthatpatientrecruitmentisthemost

challengingaspectofrarediseaseclinical

trials57%Ofrespondentsreportedthat

theircompanieshaveconductedhybridclinicaltrialsthatutilizesomedecentralizedelementsoverthemostrecent12months.47%Ofrespondents’companiesconductedanOncology-focusedraredisease

clinicaltrialinthepast24

months.6RareDiseases

Survey

PART2DEMOGRAPHICSRAREDISEASES

SURVEY7RareDiseases

Survey

Universityor

OtherAcademic

Institution4%8RareDiseases

Survey

Demographics22.1SurveyParticipant’sCompany/Organization

Type6%Researchand

Development(R&D)

Laboratory18%72%Biopharmaceutical

CompanySmallMoleculePharmaceuticalCompany2.2SurveyParticipant’sRegional

Location70%North

America20%Europe10%AsiaPacificProject

Manager1%2Demographics2.3

Survey

Participant’s

Years

of

Life

Science

Experience2.5SurveyParticipant’sDepartment/Work

Area2.4

SurveyParticipant’sJobTitle54%Researchand

Development(R&D)15%BusinessDevelopment5%Drug

Production/Manufacturing17%ClinicalOperations/Management2%QualityAssurance/Quality

Control2%

Purchasing/Procurement2%External

Manufacturing1%Supply

Chain1%Regulatory

Affairs38%49%Director/Senior

Director91%Morethan10

yearsExecutive(VP,President,

CEO,CIO,CTO,COO,

etc.)12%Manager/Senior

ManagerBetween

3

and

5

years1%9RareDiseases

Survey

8%Between5and

10years10RareDiseases

Survey

9%ofrespondentssaidtheyactasapoint-of-contactbetween

thecontractorandtheir

company2.7

Survey

Participant’s

Role

When

Deciding

on/Working

with

Outsourcing

Partners70%ofrespondentssaidtheyarepartofthedecision-makinggroupthatconductsresearchinto/interviews/selectsapotentialpartnershipopportunity2Demographics2.6

Survey

Participant’s

Company’s

Involvement

with

Rare

DiseaseIsyourcompanycurrentlydevelopingand/orcommercializingadrugortherapythatisusedinthetreatmentofpatientswitharare

disease?100%Yes70%本报告来源于三个皮匠报告站（）,由用户Id:262617下载,文档Id:177782,下载日期:2024-10-1521%ofrespondentssaidthey

arepartofateamthatestablishesthecriteriaforthecontractorselection

process21%9%PART

3OUTSOURCINGWITH

ACONTRACTRESEARCHORGANIZATION

(CRO)11

RareDiseases

Survey

RAREDISEASES

SURVEYMycompanyhasmultiple

therapeuticsforraredisease(s)acrossvariousstagesof

development.Mycompanyhasmultipletherapeuticsforraredisease(s)intheearlystages

ofdevelopment.Mycompanyhasasingle

therapeuticforraredisease(s)intheearly

stagesof

development.Mycompanyhas

multipletherapeuticsforraredisease(s)

inthelatestagesof

development.Mycompanyhasa

singletherapeuticforraredisease(s)

inthelatestagesof

development.3OutsourcingwithaContractResearchOrganization

(CRO)3.1SurveyParticipant’sCompany’s

Size3.2

Survey

Participant’s

Company’s

Rare

Disease

Portfolio

Status32%21%7%34%6%42%12

RareDiseases

Survey

22%36%Small

company(Upto500

employees)Mid-sized

company(501to2,500

employees)Largecompany(2,500+

employees)3.3

Partnering

with

a

Contract

Research

Organization

(CRO)IsyourcompanycurrentlyutilizinganyContractResearchOrganizations(CROs)aspartof

yourrarediseasedrug(s)

development?3.3a

Partnering

with

a

CRO

–

Number

of

Contract

PartnersWithhowmanyCROsareyoucurrently

working?3OutsourcingwithaContractResearchOrganization

(CRO)NOTE:

Total

number

of

respondents

is

86;

the

respondents

who

answered

“Yes”

to

Question

3.3

were

prompted

to

answer

this.86%Yes14%No34%3-4

CROs23%2

CROs22%1

CRO21%5+

CROsNOTE:

Total

number

of

respondents

is

14;

the

respondents

who

answered

“No”

to

Question

3.3

were

prompted

to

answer

this.3.3b

Partnering

with

a

CRO

–

Number

of

Contract

PartnersWith

how

many

CROs

does

yourcompany

expect

to

work

when

it

does

begin

to

outsourceprojects?43%2

CROs29%1

CRO21%3-4

CROs7%5+

CROs13

RareDiseases

Survey

3.3cCurrentOutsourcingActivity(CROs):

ResearchNeedsApproximatelywhatpercentageofyourresearchneedsisyourcompanycurrently

outsourcing?3OutsourcingwithaContractResearchOrganization

(CRO)NOTE:

Total

number

of

respondents

is

86;

the

respondents

who

answered

“Yes”

to

Question

3.3

were

prompted

to

answer

this.Upto

20%13%21%to

40%28%41%to

60%24%61%to

80%24%81%to

100%10%Research

NeedsRespondents3.3dPossibleOutsourcingActivity(CROs):Research

NeedsApproximately

what

percentage

of

your

research

needs

do

you

expect

your

company

tooutsource?NOTE:

Total

number

of

respondents

is

14;

the

respondents

who

answered

“No”

to

Question

3.3

were

prompted

to

answer

this.Upto

20%7%21%to

40%21%41%to

60%29%61%to

80%21%81%to

100%21%Research

NeedsRespondents14

RareDiseases

Survey

8%Itwill

decreaseIamnot

sure1%3OutsourcingwithaContractResearchOrganization

(CRO)3.3eFutureOutsourcingBehavior

(CROs)What

do

you

believe

your

company’s

outsourcing

activity

withCROs

will

be

like

24

months

from

nowcomparedtoitscurrent

level?44%Itwill

increase47%Itwillstayatthesame

levelNOTE:

Total

number

of

respondents

is

86;

the

respondents

who

answered

“Yes”

to

Question

3.3

were

prompted

to

answer

this.5%NOTE:

Total

number

of

respondents

is55;the

respondents

who

answered

“Yes”

to

Question

3.4

were

prompted

to

answer

this. Small-sized

CRO3.4aPreferenceinCRO

SizeWhat

size

CRO

do

you

prefer

to

work

with?49%Large-sized

CRO45%Medium-sized

CRONot

Sure6%3.4

Decision

Driver:

Size

of

the

Contract

Research

Organization

(CRO)When

considering

a

partnership

with

a

CRO,

do

you

have

a

preference

in

the

size

of

the

contract

organization?55% 39%15

RareDiseases

Survey

YesNo3OutsourcingwithaContractResearchOrganization

(CRO)3.5

Decision

Drivers:

Choosing

a

CRO

Partner

for

Rare

Disease

StudiesWhat

factors

appeal

to

you

when

making

your

choice

for

a

CRO

partner

for

Rare

Disease

studies?(Respondentswereaskedtoranktheirtop5decisiondriversfromthe11possibleresponses,withchoice#1beingthedrivertheyfelt

was

most

important.

The

lower

the

number,

the

more

important

respondents

felt

the

factor

was.)2.831.962.69Directexperiencewithcellandgene

therapiesReliability3.073.263.25End-to-end

serviceCost3.84Digital

solutions/technicalcapabilitiesCommunication3.94Relationshipswith

patientadvocacy

groupsRelationshipswithclinicaltrial

sites3.94 3.9716

RareDiseases

Survey

AccessibilityTherapeutic

expertisev2.94Agility/flexibilityPART

4PATIENT

RESOURCES17

RareDiseases

Survey

&DRUG

DEVELOPMENTRAREDISEASES

SURVEY4PatientResources&Drug

Development4.1EstablishingRelationshipswithPatientAdvocacy

GroupsDoes

your

company

build

and

maintain

its

own

relationshipswith

patient

advocacy

groups,

or

does

it

rely

on

third

partiestoestablish

connections?40%ofrespondentssaidtheircompanyhasaninternal

unitthatestablishesrelationshipswithpatientadvocacy

groups.23%ofrespondentssaidtheircompanylookstootherorganizationstobuildconnectionswithpatientadvocacy

groups.37%ofrespondentssaidtheircompanyusesbothinternalandexternalresourcestocreateandmaintainrelationshipswithpatientadvocacy

groups.5%8%2%20%3%6%2%12%Collaborative

opportunitiesSupportand

empowermentBetter

communicationFirst-handexperience

andfeedbackfrom

patientsInnovation/technologiesAccesstopatients

forclinical

trialsAccesstogrant

fundingIncreaseinenrollment

rateTransparency/agility/fasterresponsesAwarenessand

educationaboutrare

diseasesTeam

motivationPatient

recruitmentNOTE:

Total

number

of

respondents

is

100;

multiple

answers

were

allowed.40%23%37%4.1aBenefits

of

Relationships

with

Patient

Advocacy

GroupsWhat

benefits

have

you

experienced

through

your

connection

with

patient

advocacy

groups?54%

33%

24%18

RareDiseases

Survey

Abetterunderstandingofpatients’

needs10% 9%Developmentof

clinicaloutcome

assessments57%Identifyingpatient

population56%Identifyingordeveloping

biomarkers37%Designingexternalcontrol

studies35%4PatientResources&Drug

Development4.2UsingNaturalHistory

StudiesWhich

of

the

following

uses

of

naturalhistory

studies

are

you

considering

to

inform

your

clinicaldevelopment

program?(Multipleanswerswere

allowed)4.3UsingDiseaseorPatient

RegistriesIn

which

of

the

following

areas

are

you

considering

the

use

of

a

disease

or

patient

registry?(Multipleanswerswere

allowed)Wearenot

consideringanaturalhistory

study21%66%19

RareDiseases

Survey

Clinicaltrialdesign/identifykey

sub-groups40%35%Pre-clinical

portfolioUncoverunmet

needsprioritization/andmarket

potentialinvestment

decisions35%SupportregulatoryandHTA

submissions34%Support

go-to-marketactivities9%Wearenot

consideringa

registry1%OtherExternal/Syntheticcontrol

arms46%Basket/bucket

trials44%Platform

studies37%Umbrella

trials35%Masterobservationaltrials

(MOTs)20%Other2%4PatientResources&Drug

Development4.4

NovelApproachestoDrugDevelopmentWhich

of

the

following

novel

approaches

to

drug

development

areof

interest

to

your

company

for

use

inclinical

trials?(Multipleanswerswere

allowed)4.5ClinicalTrialSite

LocationsIsyourcompanyopentoconductingclinicaltrialsinless-conventionalgeographicregionsatsitesthat

are(a)

relatively

new

to

clinical

trialsbut

(b)

appropriately

supported

for

success?63%

20%

17%20

RareDiseases

Survey

YesNoNot

Sure4PatientResources&Drug

Development59%Concernswith

costs53%21

RareDiseases

Survey

Increasingcomplexityinclinicaltrial

designHeterogeneityofpatientpopulation

withcertainrare

diseases30%Expanded

regulatoryrequirements8%Determining

responsibilityinclinicaltrial

governanceLackof

understandingofrarediseases’natural

historyLow

prevalenceof

condition2%Other21%Findingqualified

stafftohandleincreasedtechnologicaldemandsAccesstoappropriatepatient

pools41% 37%4.6

Challenges

Facing

the

Rare

Disease

Drug

Development

SpaceWhat

do

you

consider

to

be

the

biggest

challenges

facing

rare

disease

drug

development

in

the

upcoming10-year

period?(Multipleanswerswere

allowed)62% 54%PART5CLINICAL

TRIALDETAILSRAREDISEASESSURVEY22

RareDiseases

Survey

5ClinicalTrial

Details47%OncologyGenetic

disorders33%23

RareDiseases

Survey

13%31%8%NeurologyMusculoskeletaldisordersImmunologyOphthalmology4%7%5.1RareDiseaseClinicalTrialsbyTherapy

AreaAcross

which

therapeutic

areas

has

your

company

run

raredisease

clinicaltrials

over

the

most

recent

24

months?(Multipleanswerswere

allowed)21% 16% 42%2%4%HematologyToxicology6%OtherHormonal

disordersInfectious

diseaseRespiratory

diseaseNon-malignant

disorders5ClinicalTrial

Details5.2

PediatricPatientswithinRareDiseaseClinicalTrialsApproximately

what

percentage

of

the

rare

disease

clinical

trialsthatyou

have

conducted

over

the

most

recent

24monthsincludedpediatric

patients?5.3

Challenges

Faced

when

Conducting

Rare

Disease

Clinical

TrialsFrom

the

following

list,

what

do

youfeelisthe

most

difficult

or

challenging

aspect

of

rare

disease

clinical

trials?52% 16% 9%Recruitingpatientsforthe

trialComplexityoftheclinical

trialrequirements1%Developmentand

accesstopatient

registriesRegulatory

barriers1%Educatingpatients

andtheir

family/caregivers5%Patient

retention8%Identifyinga

diversepatient

population2%Collectionandmanagementof

data6%Lengthofthe

trialstartup

process0%29%Upto

20%33%21%to

40%20%41%to

60%6%61%to

80%6%81%to

100%6%RareDiseaseClinical

TrialsRespondents24

RareDiseases

Survey

5ClinicalTrial

Details5.3a

How

CRO

Partners

Can

Help

Address

Challenges

with

Rare

Disease

Clinical

TrialsWhat

actions

would

youliketo

see

from

a

CRO

partner

to

help

navigate

through

these

challenges?(Multipleanswerswere

allowed)24% 18%Assistancewithpatientrecruitment/enrollment16%25

RareDiseases

Survey

16%Relationships

with/accesstoregionalclinicaltrial

sitesDemonstrateoverall

expertise/experience,

guidance,and

commitment9%7%5%Patientidentification/maintainapatient

database10%

9%8%5%4%Greater/improvedcommunication

andlisteningAssistancewithpatient

retentionand

complianceGreater

adaptability/flexibilityand

efficiency1%Abilitytoconductdecentralizedclinical

trialsRegulatory

supportEngagement

withpatientadvocacygroupsand

KOLsKnowledge

of/accesswithintherapeuticareasof

interestImproved

technologyandtechnicalexpertise8%OtherHelpinimprovingclinicaltrial

designAfullydecentralizedclinicaltrial(DCT)thatleveragesdigitaltoolsandsupportingservices,suchas

eConsent,eCOA,homehealthcare,etc.to

enablethepatienttocompletesomeorallscheduledvisitsfromtheir

home5ClinicalTrial

Details5.4

Types

of

Clinical

Trials

Conducted

for

Rare

Disease

TherapiesWhat

types

of

clinical

trialshasyour

company

conducted

for

rare

disease

therapiesduring

the

most

recent12-month

period?(Multipleanswerswere

allowed)5.5

Utilizing

Decentralized

Elements

in

Rare

Disease

Clinical

TrialsApproximately

what

percentage

of

the

rare

disease

clinical

trialsthatwere

conducted

during

thisperiod

utilizedremoteordecentralized

elements?Atraditionalclinicaltrialthatis

fullyconductedatadesignated

site48%A“hybrid”clinicaltrialthat

incorporatedatleastsomedecentralized

clinicaltrialelements57%11%0%19%Upto

20%31%21%to

40%32%41%to

60%12%61%to

80%3%81%to

100%3%RareDiseaseClinical

TrialsRespondents26

RareDiseases

Survey

5ClinicalTrial

Details5.5a

Types

of

Decentralized

Elements

Used

in

Decentralized

Rare

Disease

Clinical

TrialsWhich

decentralized

elements

did

you

utilize

when

conducting

a

hybrid

or

fullyremote

rare

disease

clinical

trial?(Multipleanswerswere

allowed)42%44%Electronicinformed

consent/eConsent

softwareElectroniccasereportforms/eCRF

software37% 31%Electronicclinicaloutcomes

assessment(eCOA)Telemedicine

services23%26%Electronicpatient-reported

outcomes/ePro

softwareIn-homenurseservices/health

visits9%9%Pharmacy-based

clinics38%Remotepatient

monitoringMobile

clinics16%27

RareDiseases

Survey

14%Remoteor

satelliteclinicaltrial

sitesWearable

technologies32%31%31%26%23%17%17%11%11%4%2%0%28

RareDiseases

Survey

5ClinicalTrial

Details5.5bFuturePlansforReusingDecentralized

ElementsWhichofthefollowingdecentralizedelements—thatwereutilizedwhenconductingahybridorfullyremote

rarediseaseclinicaltrial—wouldyoubeinterestedinusing

again?(Multipleanswerswere

allowed)Electronicclinical

outcomesassessment

(eCOA)Electroniccasereportforms/eCRF

softwareElectronicinformed

consent/eConsent

softwareRemotepatient

monitoringTelemedicine

servicesIn-homenurseservices/health

visitsElectronicpatient-reported

outcomes/ePro

softwareWearable

technologiesRemoteorsatelliteclinicaltrial

sitesMobile

clinicsPharmacy-based

clinicsOther5.5c

Future

Plans

for

Trying

Previously

Unused

Decentralized

ElementsWhichofthefollowingdecentralizedelements—thatwerenotutilizedwhenconductingahybridorfully

remoteraredisease

clinical

trial

—

would

you

be

interested

in

using

in

future

clinical

trials?(Multipleanswerswere

allowed)Wearable

technologiesRemotepatient

monitoringPharmacy-based

clinicsMobile

clinicsElectronicpatient-reported

outcomes/ePro

softwareIn-homenurseservices/health

visitsRemoteorsatelliteclinicaltrial

sitesTelemedicine

servicesElectroniccasereportforms/eCRF

softwareElectronicinformed

consent/eConsent

softwareElectronicclinical

outcomesassessment

(eCOA)Other29%23%22%22%17%16%15%14%14%12%8%2%5ClinicalTrial

Details5.7

Additional

Resources

Used

to

Increase/Share

Knowledge

about

Rare

DiseaseWhich

of

the

following

resources

does

your

company

use

to

network

and

share

knowledge

about

rare

diseases?(Multipleanswerswere

allowed)Conferences/eventsSeminars/webinarsOnlineforumsorcommunity

sitesSocial

media67%55%50%74%Coordinationofvisits

orassessmentsover

time31%28%29%28%Device/softwaretraining51%29

RareDiseases

Survey

Transportationto/fromclinicaltrial

sitesMobility

assistanceAssistancewithmedical

equipmentAssistancewithreimbursement

needs5.6

Patient

Requirements

for

In-Person

Visits

During

Clinical

TrialsDo

patients

in

your

rare

disease

clinicaltrialsrequire

any

of

the

following

services

for

their

in-person

clinicvisits?(Multipleanswerswere

allowed)64% 35%Medication

remin