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1、RARE DISEASES SURVEY RESULTS1 Introduction pg.22 Demographics pg.73 Outsourcing with a Contract Research Organization(CRO)pg.114 Patient Resources&Drug Development pg.175 Clinical Trial Details 2 Rare Diseases SurveyPART 1INTRODUCTIONRARE DISEASES SURVEYIntroduction13 Rare Diseases SurveySponsored b

2、y PPD,part of Thermo Fisher ScientificThis survey was conducted in April 2023 among 100 respondents who work in some capacity within the rare disease sector and who currently outsource(or plan to outsource within the next 2 years)some of their research needs to a contract research organization(CRO).

3、The goal of the study was to identify clients needs and the challenges they are facing in terms of rare disease Introduction14 Rare Diseases SurveyDemographicsThe study was designed to be global but with a strong bias toward North America-based respondents,who comprise 70%of the audience;20%of the a

4、udience are from Europe,while the remaining 10%are in the Asia Pacific region.The bulk of the audience(72%)works at a biopharmaceutical company,while 18%are employed by a small molecule pharmaceutical company;the remaining 10%are at R&D laboratories(6%)or academic institutions/universities(4%).91%of

5、 the audience noted that they have at least 10 years of experience within the life science sector,and 87%are in a director-level position or higher,representing an approach that supports high relevance and strong data integrity.By design,only respondents whose companies or organizations are working

6、within rare disease,and whose companies or organizations are either currently partnering with a CRO or plan to do so within the next 24 months,were able to complete the survey.RESPONDENT EXPERIENCEOf respondents have 10 years life sciences experiences91%RESPONDENT SENIORITYOf respondents are directo

7、r level or above87%Introduction15 Rare Diseases SurveyTrendsWhen asked if they are currently partnering with a CRO as part of their rare disease drug development plan,86%of respondents noted that they were:55%of that group of respondents said they were working with at least 3 contract partners.44%of

8、 these respondents expect their outsourcing activity to increase over the upcoming 24 months,while 47%felt that their partnerships would likely remain at the current level.The decision drivers that were identified as being the most important when selecting a CRO to partner with on rare disease studi

9、es were“Therapeutic expertise”,“Direct experience with cell&gene therapies”,“Reliability”,“Agility/flexibility”,and“End-to-end service”.47%of respondents reported that their companies conducted an oncology-focused rare disease clinical trial within the most recent 24 months;Genetic Disorders was the

10、 second highest therapy area at 42%,with Neurology in third(33%).The most frequently selected challenge faced by the audience when conducting rare disease clinical trials was“Recruiting patients for the trial”,which was chosen by 52%of the participants.“Complexity of the clinical trial requirements”

11、was a distant second at 16%.Decentralized clinical trials within the rare disease space were used to varying degrees by respondents companies:11%reported that a fully decentralized model was used for at least one of their rare disease clinical trials over the most recent 12 months,while 57%reported

12、that at least one“hybrid”clinical trial,involving both traditional and decentralized aspects,was conducted over the same period.ECRF and eConsent software,at 44%and 42%,respectively,were the most used decentralized elements.Respondents reported that“Coordination of visits or assessments over time”(6

13、4%)and“Transportation to&from clinical trial sites”(51%)were the top needs for patients during their in-person clinical trial visits.Of respondents expect their outsourcing activity to increase over the next 24 months44%Of respondents whose companies are currently outsourcing work for rare disease c

14、linical trials are using 3+CROs55%Of respondents said that their companies are currently utilizing CROs as part of their rare disease drug development efforts.86%Introduction16 Rare Diseases SurveyAnother section of the survey was dedicated to answering an assortment of questions around topics such

15、as patient advocacy groups,natural history studies,patient&disease registries,tokenization,and drug development.40%of respondents noted that relationship building between their companies and patient advocacy groups are handled by an internal unit;37%said that a mix of internal and external resources

16、 are utilized to create and maintain these relationships.Among the study participants who said that their companies utilized natural history studies(79%of the audience),“Development of clinical outcome assessments”(57%)and“Identifying patient population”(56%)were named as the top two uses for the re

17、source.21%of respondents claimed that they were neither using nor considering using a natural history study to build their clinical development program.Patient/disease registries appear to be a useful tool among the study audience,as only 9%of respondents said that they are not considering the use o

18、f a registry.The most frequently given reason for utilizing a patient/disease registry was“Clinical trial design/identify key sub-groups”(66%);the next highest reason,“Uncover unmet needs and market potential”,was selected by 40%of respondents.Of the novel approaches to drug development that were in

19、cluded as part of the study,“External/Synthetic control arms”drew the most interest at 46%;“Basket/bucket trials”were second with 44%.With none of the given options breaking the 50%mark with participants,this might represent an opportunity to build awareness and knowledge of the various tools.Of res

20、pondents feel that patient recruitment is the most challenging aspect of rare disease clinical trials52%Of respondents reported that their companies have conducted hybrid clinical trials that utilize some decentralized elements over the most recent 12 months.57%Of respondents companies conducted an

21、Oncology-focused rare disease clinical trial in the past 24 months.47%7 Rare Diseases SurveyPART 2DEMOGRAPHICSRARE DISEASES SURVEY8 Rare Diseases SurveyUniversity or Other Academic Institution4%Demographics22.1 Survey Participants Company/Organization TypeResearch and Development(R&D)Laboratory6%72%

22、18%Biopharmaceutical CompanySmall Molecule Pharmaceutical Company2.2 Survey Participants Regional Location70%North America20%Europe10%Asia P9 Rare Diseases SurveyProject Manager1%2Demographics2.3 Survey Participants Years of Life Science Experience2.5 Survey Participants Department/Work Area2.4 Surv

23、ey Participants Job Title54%Research and Development (R&D)15%Business Development5%Drug Production/Manufacturing17%Clinical Operations/Management2%Quality Assurance/Quality Control2%Purchasing/Procurement2%External Manufacturing1%Supply Chain1%Regulatory Affairs38%49%Director/Senior Director91%More

24、than 10 yearsExecutive(VP,President,CEO,CIO,CTO,COO,etc.)Manager/Senior Manager12%Between 3 and 5 years1%Between 5 and 10 years8%10 Rare Diseases Survey9%of respondents said they act as a point-of-contact between the contractor and their company21%of respondents said they are part of a team that est

25、ablishes the criteria for the contractor selection process70%of respondents said they are part of the decision-making group that conducts research into/interviews/selects a potential partnership opportunity2DemographicsIs your company currently developing and/or commercializing a drug ortherapy that

26、 is used in the treatment of patients with a rare disease?2.6 Survey Participants Companys Involvement with Rare Disease2.7 Survey Participants Role When Deciding on/Working with Outsourcing Partners100%Yes70%9%21%11 Rare Diseases SurveyPART 3OUTSOURCING WITH A CONTRACT RESEARCH ORGANIZATION(CRO)RAR

27、E DISEASES SURVEY12 Rare Diseases SurveyMy company has multiple therapeutics for rare disease(s)across various stages of development.My company has multiple therapeutics for rare disease(s)in the early stages of development.My company has a single therapeutic for rare disease(s)in the early stages o

28、f development.My company has multiple therapeutics for rare disease(s)in the late stages ofdevelopment.My company has a single therapeutic for rare disease(s)in the late stages of development.3Outsourcing with a Contract Research Organization(CRO)3.1 Survey Participants Companys Size3.2 Survey Parti

29、cipants Companys Rare Disease Portfolio Status32%21%7%34%6%42%22%36%Small company(Up to 500 employees)Mid-sized company(501 to 2,500 employees)Large company(2,500+employees)13 Rare Diseases Survey3.3 Partnering with a Contract Research Organization(CRO)Is your company currently utilizing any Contrac

30、t Research Organizations(CROs)as part of your rare disease drug(s)development?3.3a Partnering with a CRO Number of Contract PartnersWith how many CROs are you currently working?3Outsourcing with a Contract Research Organization(CRO)NOTE:Total number of respondents is 86;the respondents who answered“

31、Yes”to Question 3.3 were prompted to answer this.86%14%YesNo22%1 CRO23%2 CROs34%3-4 CROs21%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 your comp

32、any expect to work when it does begin to outsource projects?29%1 CRO43%2 CROs21%3-4 CROs5+CROs7%14 Rare Diseases Survey3.3c Current Outsourcing Activity(CROs):Research NeedsApproximately what percentage of your research needs is your company currently outsourcing?3Outsourcing with a Contract Researc

33、h Organization(CRO)NOTE:Total number of respondents is 86;the respondents who answered“Yes”to Question 3.3 were prompted to answer this.Up to 20%13%21%to 40%28%41%to 60%24%61%to 80%24%81%to 100%10%Research NeedsRespondents3.3d Possible Outsourcing Activity(CROs):Research NeedsApproximately what perc

34、entage of your research needs do you expect your company to outsource?NOTE:Total number of respondents is 14;the respondents who answered“No”to Question 3.3 were prompted to answer this.Up to 20%7%21%to 40%21%41%to 60%29%61%to 80%21%81%to 100%21%Research NeedsR15 Rare Diseases SurveyIt will decrease

35、8%I am not sure1%3Outsourcing with a Contract Research Organization(CRO)3.3e Future Outsourcing Behavior(CROs)What do you believe your companys outsourcing activity with CROs will be like 24 months from now compared to its current level?It will increase44%47%It will stay at the same levelNOTE:Total

36、number of respondents is 86;the respondents who answered“Yes”to Question 3.3 were prompted to answer this.Small-sized CRO5%3.4a Preference in CRO SizeWhat size CRO do you prefer to work with?NOTE:Total number of respondents is 55;the respondents who answered“Yes”to Question 3.4 were prompted to answ

37、er this.49%45%Large-sized CROMedium-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%YesN16 Rare Diseases Survey3Outsourcing with a Contract Research

38、 Organization(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?(Respondents were asked to rank their top 5 decision drivers from the 11 possible responses,with choice#1 being the driv

39、er they felt was most important.The lower the number,the more important respondents felt the factor was.)2.831.962.69Direct experience with cell and gene therapiesReliability3.073.263.25End-to-end serviceRelationships with clinical trial sitesCost3.84Digital solutions/technical capabilities3.94Commu

40、nication3.94Relationships with patient advocacy groups3.97AccessibilityTherapeutic expertisev2.94Agility/17 Rare Diseases SurveyPART 4PATIENT RESOURCES&DRUG DEVELOPMENTRARE DISEASES SURVEY18 Rare Diseases Survey4Patient Resources&Drug Development4.1 Establishing Relationships with Patient Advocacy G

41、roupsDoes your company build and maintain its own relationships with patient advocacy groups,or does it rely on third parties to establish connections?4.1a Benefits of Relationships with Patient Advocacy GroupsWhat benefits have you experienced through your connection with patient advocacy groups?40

42、%of respondents said their company has an internal unit that establishes relationships with patient advocacy groups.23%of respondents said their company looks to other organizations to build connections with patient advocacygroups.37%of respondents said their company uses both internal and external

43、resources to create and maintain relationships with patient advocacy groups.10%5%8%33%2%20%9%3%6%24%2%12%Collaborative opportunitiesSupport and empowermentBetter communicationFirst-hand experience and feedback from patientsInnovation/technologiesAccess to patients for clinical trialsAccess to grant

44、fundingIncrease in enrollment rateTransparency/agility/faster responsesAwareness and education about rare diseasesTeam motivationPatient recruitmentNOTE:Total number of respondents is 100;multiple answers were allowed.40%23%37%54%A better understanding of patients 19 Rare Diseases Survey4Patient Res

45、ources&Drug Development4.2 Using Natural History StudiesWhich of the following uses of natural history studies are you considering to inform your clinical developmentprogram?(Multiple answers were allowed)4.3 Using Disease or Patient RegistriesIn which of the following areas are you considering the

46、use of a disease or patient registry?(Multiple answers were allowed)Development of clinical outcome assessmentsIdentifying patient populationIdentifying or developing biomarkersDesigning external control studiesWe are not considering a natural history study56%37%35%21%57%66%9%1%40%35%35%34%Clinical

47、trial design/identify key sub-groupsWe are not considering a registryOtherUncover unmet needs and market potentialSupport regulatory and HTA submissionsPre-clinical portfolio prioritization/investment decisionsSupport go-to-market 20 Rare Diseases Survey4Patient Resources&Drug Development4.4 Novel A

48、pproaches to Drug DevelopmentWhich of the following novel approaches to drug development are of interest to your company for use in clinicaltrials?(Multiple answers were allowed)4.5 Clinical Trial Site LocationsIs your company open to conducting clinical trials in less-conventional geographic region

49、s at sites that are(a)relatively new to clinical trials but(b)appropriately supported for success?External/Synthetic control armsBasket/bucket trialsPlatform studiesUmbrella trialsMaster observational trials(MOTs)Other44%37%35%20%2%46%63%20%17%YesNoNot S21 Rare Diseases Survey4Patient Resources&Drug

50、 Development4.6 Challenges Facing the Rare Disease Drug Development SpaceWhat do you consider to be the biggest challenges facing rare disease drug development in the upcoming 10-year period?(Multiple answers were allowed)59%Concerns with costs54%53%Increasing complexity in clinical trial designHete

51、rogeneity of patient population with certain rare diseases41%8%30%37%2%21%Expanded regulatory requirementsDetermining responsibility in clinical trial governanceLack of understanding of rare diseases natural historyLow prevalence ofconditionOtherFinding qualified staff to handle increased technologi

52、cal demandsAccess to appropriate patient pools62%22 Rare Diseases SurveyPART 5CLINICAL TRIAL DETAILSRARE DISEASES SURVEY23 Rare Diseases Survey5Clinical Trial Details5.1 Rare Disease Clinical Trials by Therapy AreaAcross which therapeutic areas has your company run rare disease clinical trials over

53、the most recent 24 months?(Multiple answers were allowed)47%42%OncologyGenetic disorders33%13%31%8%NeurologyMusculoskeletal disordersImmunologyOphthalmology21%4%6%7%16%2%4%HematologyToxicologyOtherHormonal disordersInfectious diseaseRespiratory diseaseNon-malignant 24 Rare Diseases Survey5Clinical T

54、rial Details5.2 Pediatric Patients within Rare Disease Clinical TrialsApproximately what percentage of the rare disease clinical trials that you have conducted over the most recent 24 months included pediatric patients?5.3 Challenges Faced when Conducting Rare Disease Clinical TrialsFrom the followi

55、ng list,what do you feel is the most difficult or challenging aspect of rare disease clinical trials?16%1%9%1%52%Recruiting patients for the trialComplexity of the clinical trial requirementsDevelopment and access to patient registriesRegulatory barriersEducating patients and their family/caregivers

56、5%8%2%6%Patient retentionIdentifying a diverse patient populationCollection and management of dataLength of the trial startup process0%29%Up to 20%33%21%to 40%20%41%to 60%6%61%to 80%6%81%to 100%6%Rare Disease Clinical TrialsR25 Rare Diseases Survey5Clinical Trial Details5.3a How CRO Partners Can Hel

57、p Address Challenges with Rare Disease Clinical TrialsWhat actions would you like to see from a CRO partner to help navigate through these challenges?(Multiple answers were allowed)24%18%Assistance with patient recruitment/enrollmentPatient identification/maintain a patient database16%16%Relationshi

58、ps with/access to regional clinical trial sitesDemonstrate overall expertise/experience,guidance,andcommitment10%9%7%1%5%9%8%5%8%4%Greater/improved communication and listeningAssistance with patient retention andcomplianceGreater adaptability/flexibility andefficiencyAbility to conduct decentralized

59、 clinical trialsRegulatory supportEngagement with patient advocacy groups and KOLsKnowledge of/access within therapeutic areas of interestImproved technology and technical expertiseOtherHelp in improving clinical trial 26 Rare Diseases SurveyA fully decentralized clinical trial(DCT)that leverages di

60、gital tools and supporting services,such as eConsent,eCOA,home healthcare,etc.to enable the patient to complete some or all scheduled visits from their home5Clinical Trial Details5.4 Types of Clinical Trials Conducted for Rare Disease TherapiesWhat types of clinical trials has your company conducted

61、 for rare disease therapies during the most recent 12-month period?(Multiple answers were allowed)5.5 Utilizing Decentralized Elements in Rare Disease Clinical TrialsApproximately what percentage of the rare disease clinical trials that were conducted during this period utilized remote or decentrali

62、zed elements?A traditional clinical trial that is fully conducted at a designatedsite48%A“hybrid”clinical trial that incorporated at least some decentralized clinical trialelements57%11%0%19%Up to 20%31%21%to 40%32%41%to 60%12%61%to 80%3%81%to 100%3%Rare Disease Clinical TrialsR27 Rare Diseases Surv

63、ey5Clinical Trial Details5.5a Types of Decentralized Elements Used in Decentralized Rare Disease Clinical Trials Which decentralized elements did you utilize when conducting a hybrid or fully remote rare disease clinical trial?(Multiple answers were allowed)42%44%Electronic informed consent/eConsent

64、 softwareElectronic case report forms/eCRF software37%31%Electronic clinical outcomes assessment(eCOA)Telemedicine services26%23%Electronic patient-reported outcomes/ePro softwareIn-home nurse services/health visits9%38%9%Pharmacy-based clinicsRemote patient monitoringMobile clinics16%14%Remote or s

65、atellite clinical trial sitesWearable 28 Rare Diseases Survey5Clinical Trial Details5.5b Future Plans for Reusing Decentralized ElementsWhich of the following decentralized elements that were utilized when conducting a hybrid or fully remote rare disease clinical trial would you be interested in usi

66、ng again?(Multiple answers were allowed)5.5c Future Plans for Trying Previously Unused Decentralized ElementsWhich of the following decentralized elements that were not utilized when conducting a hybrid or fully remote rare disease clinical trial would you be interested in using in future clinical t

67、rials?(Multiple answers were allowed)Electronic clinical outcomes assessment(eCOA)32%Electronic clinical outcomes assessment(eCOA)8%Other0%Other2%Mobile clinics4%Mobile clinicsWearable technologies11%Wearable technologies29%Telemedicine services23%Telemedicine services14%In-home nurse services/healt

68、h visits17%In-home nurse services/health visits16%31%Pharmacy-based clinics2%Pharmacy-based clinicsRemote or satellite clinical trial sites11%Remote or satellite clinical trial sites15%17%Remote patient monitoring26%Remote patient monitoring23%Electronic informed consent/eConsent software12%Electron

69、ic informed consent/eConsent softwareElectronic patient-reported outcomes/ePro software17%Electronic patient-reported outcomes/ePro softwareElectronic case report forms/eCRF software31%Electronic case report forms/eCRF software14%22%22%29 Rare Diseases Survey5Clinical Trial Details5.6 Patient Requir

70、ements for In-Person Visits During Clinical TrialsDo patients in your rare disease clinical trials require any of the following services for their in-person clinic visits?(Multiple answers were allowed)5.7 Additional Resources Used to Increase/Share Knowledge about Rare DiseaseWhich of the following

71、 resources does your company use to network and share knowledge about rare diseases?(Multiple answers were allowed)Conferences/eventsSeminars/webinarsOnline forums or community sitesSocial media67%55%50%74%64%Coordination of visits or assessments over time31%28%29%28%Device/software training51%Trans

72、portation to/from clinical trial sitesMobility assistanceAssistance with medical equipmentAssistance with reimbursement needs35%Medication 30 Rare Diseases Survey5Clinical Trial Details5.7a Additional Resources Used to Increase/Share Knowledge Online ForumsWhich online forums or community sites do y

73、ou find most beneficial?(Multiple answers were allowed)NOTE:Total number of respondents is 55;respondents who chose“Online forums or community sites”in Question 5.7 were prompted to answer this.There is a total of 83 mentions in this question;the table above highlights the Top 10 answers from that l

74、ist.Rare disease forumsTwitterOnline forumsLinkedInPatient advocacy sitesDisease specific groupsCompany websiteRare Disease Clinical Research Network(RDCRN)FacebookConferences16%7%4%15%7%4%13%4%13%4%31 Rare Diseases Survey5Clinical Trial Details5.7b Additional Resources Used to Increase/Share Knowle

75、dge Conferences&EventsWhich conferences/events do you find most beneficial?(Multiple answers were allowed)NOTE:Total number of respondents is 74;respondents who chose“Conferences/events”in Question 5.7 were prompted to answer this.There is a total of 119 items mentioned in this question;the table ab

76、ove highlights the Top 17 answers from that list.Other9%Orphan Drug and Rare Disease conferenceDisease specific conferences9%11%World conferencesWorld Congress on Rare DiseasesAmerican Thoracic SocietyAmerican Society of Hematology(ASH)American Association of Cancer Research(AACR)American Society of

77、 Clinical Oncology(ASCO)European Conference on Rare DiseasesNational Organization for Rare Disorders(NORD)American Society of Gene&Cell Therapy(ASGCT)European Society of Gene&Cell Therapy(ESGCT)Patient organizationLarge scale multinational eventsRare Disease Day ForumEuropean Society for Medical Onc

78、ology(ESMO)7%5%3%7%7%4%4%4%3%9%3%8%5%3%32 Rare Diseases Survey5Clinical Trial Details5.7c Additional Resources Used to Increase/Share Knowledge Social MediaWhich social media platforms do you find most beneficial?(Multiple answers were allowed)NOTE:Total number of respondents is 50;respondents who a

79、nswered“Social media”in Question 5.7.were prompted to answer this.There is a total of 74 items mentioned in this question;the table above highlights the Top 13 answers from that list.FacebookShare4RarePatient advocacyLinkedInTikTokSearch advertisingPatient groupsTwitterLinks to patient groupsPharmaceutical publicationInstagramMedical conference,symposium,and publications44%4%2%30%2%2%2%2%6%48%4%2%