1、Identifying clients needs and challenges in rare disease clinical developmentClinical researchppdIntroduction 3 Demographics 5 Outsourcing with a Contract Research Organization(CRO)12Patient Resources&Drug Development 18Clinical Trial Details 25 Contents2 Rare diseases survey 1INTRODUCTIONRARE DISEA

2、SES SURVEY3 Rare diseases survey by 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 co

3、ntract research organization(CRO).The goal of the study was to identify clients needs and the challenges they are facing in terms of rare disease clinicaldevelopment.4 Rare diseases survey study was designed to be global but with a strong bias toward North America-based respondents,who comprise 70%o

4、f the audience;20%of the audience 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 instituti

5、ons/universities(4%).91%of 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

6、organizations are working 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 SENIORITY

7、Of respondents are director level or above87%5 Rare diseases survey 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

8、%of 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 st

9、udies 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

10、the 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 requiremen

11、ts”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%report

12、ed 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

13、”(64%)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 diseas

14、e clinical trials are using 3+CROs55%Of respondents said that their companies are currently utilizing CROs as part of their rare disease drug development efforts86%6 Rare diseases survey section of the survey was dedicated to answering an assortment of questions around topics such as patient advocac

15、y 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 are utilized to c

16、reate 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 resource.21%of respo

17、ndents 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 of a registry.The m

18、ost 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 included as part of

19、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 respondents feel that

20、 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 Oncology-focused r

21、are disease clinical trial in the past 24 months.47%7 Rare diseases survey 2DEMOGRAPHICSRARE DISEASES SURVEY8 Rare diseases survey or Other Academic Institution4%2.1 Survey Participants Company/Organization TypeResearch and Development(R&D)Laboratory6%72%18%Biopharmaceutical CompanySmall Molecule Ph

22、armaceutical Company2.2 Survey Participants Regional Location70%North America20%Europe10%Asia Pacific9 Rare diseases survey Manager1%2.3 Survey Participants Years of Life Science Experience2.5 Survey Participants Department/Work Area2.4 Survey Participants Job Title54%Research and Development (R&D)1

23、5%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 than 10 yearsExecutive(VP,President,CEO,CIO,CTO,COO,etc.)Ma

24、nager/Senior Manager12%Between 3 and 5 years1%Between 5 and 10 years8%10 Rare diseases survey 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 establishes the criteria for the contractor selection process70%of

25、 respondents said they are part of the decision-making group that conducts research into/interviews/selects a potential partnership opportunityIs your company currently developing and/or commercializing a drug ortherapy that is used in the treatment of patients with a rare disease?2.6 Survey Partici

26、pants Companys Involvement with Rare Disease2.7 Survey Participants Role When Deciding on/Working with Outsourcing Partners100%Yes70%9%21%11 Rare diseases survey 3OUTSOURCING WITH A CONTRACT RESEARCH ORGANIZATION(CRO)RARE DISEASES SURVEY12 Rare diseases survey company has multiple therapeutics for r

27、are 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 of development.My company has multiple therapeutics for rare disease(s)in the late

28、stages ofdevelopment.My company has a single therapeutic for rare disease(s)in the late stages of development.3.1 Survey Participants Companys Size3.2 Survey Participants 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 empl

29、oyees)Large company(2,500+employees)13 Rare diseases survey Partnering with a Contract Research Organization(CRO)Is your company currently utilizing any Contract Research Organizations(CROs)as part of your rare disease drug(s)development?3.3a Partnering with a CRO Number of Contract PartnersWith how

30、 many CROs are you currently working?NOTE:Total number of respondents is 86;the respondents who answered“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 prompt

31、ed to answer this.3.3b Partnering with a CRO Number of Contract PartnersWith how many CROs does your company expect to work when it does begin to outsource projects?29%1 CRO43%2 CROs21%3-4 CROs5+CROs7%14 Rare diseases survey Current Outsourcing Activity(CROs):Research NeedsApproximately what percent

32、age of your research needs is your company currently outsourcing?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 Activi

33、ty(CROs):Research NeedsApproximately what percentage 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

34、 NeedsRespondents15 Rare diseases survey will decrease8%I am not sure1%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 n

35、umber 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 answe

36、r 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%YesNo16 Rare diseases survey Decision Drivers:Choosing a CRO Part

37、ner 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 driver they felt was most important.The lower the number,the m

38、ore 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 capabilities3.94Communication3.94Relationships with patient advocacy groups3.97

39、AccessibilityTherapeutic expertisev2.94Agility/flexibility17 Rare diseases survey 4PATIENT RESOURCES&DRUG DEVELOPMENTRARE DISEASES SURVEY18 Rare diseases survey Establishing Relationships with Patient Advocacy GroupsDoes your company build and maintain its own relationships with patient advocacy gro

40、ups,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%of respondents said their company has an internal unit that establishes relationships wi

41、th 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 resources to create and maintain relationships with patient advocacy groups.10%5%8%33%2%2

42、0%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 fundingIncrease in enrollment rateTransparency/agility/faster responsesAwareness and educ

43、ation about rare diseasesTeam motivationPatient recruitmentNOTE:Total number of respondents is 100;multiple answers were allowed.40%23%37%54%A better understanding of patients needs19 Rare diseases survey Using Natural History StudiesWhich of the following uses of natural history studies are you con

44、sidering 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 use of a disease or patient registry?(Multiple answers were allowed)Development of clinical outcome assessmentsIdentifying

45、 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 trial design/identify key sub-groupsWe are not considering a registryOtherUncover unmet needs and market potentialSupport

46、regulatory and HTA submissionsPre-clinical portfolio prioritization/investment decisionsSupport go-to-market activities4.4 Novel Approaches to Drug DevelopmentWhich of the following novel approaches to drug development are of interest to your company for use in clinicaltrials?(Multiple answers were

47、allowed)4.5 Clinical Trial Site LocationsIs your company open to conducting clinical trials in less-conventional geographic regions at sites that are(a)relatively new to clinical trials but(b)appropriately supported for success?External/Synthetic control armsBasket/bucket trialsPlatform studiesUmbre

48、lla trialsMaster observational trials(MOTs)Other44%37%35%20%2%46%63%20%17%YesNoNot Sure21 Rare diseases survey 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 answ

49、ers were allowed)59%Concerns with costs54%53%Increasing complexity in clinical trial designHeterogeneity 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 natur

50、al historyLow prevalence ofconditionOtherFinding qualified staff to handle increased technological demandsAccess to appropriate patient pools62%22 Rare diseases survey 5CLINICAL TRIAL DETAILSRARE DISEASES SURVEY23 Rare diseases survey Rare Disease Clinical Trials by Therapy AreaAcross which therapeu

51、tic areas has your company run rare disease clinical trials over 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 diseaseRe

52、spiratory diseaseNon-malignant disorders24 Rare diseases survey 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 Conduct

53、ing Rare Disease Clinical TrialsFrom the following 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 barrie

54、rsEducating patients and their family/caregivers5%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 TrialsRespondents25 Rare diseases

55、survey How CRO Partners Can Help 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 pa

56、tient database16%16%Relationships 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 andefficiencyA

57、bility to conduct decentralized clinical trialsRegulatory supportEngagement with patient advocacy groups and KOLsKnowledge of/access within therapeutic areas of interestImproved technology and technical expertiseOtherHelp in improving clinical trial design26 Rare diseases survey fully decentralized

58、clinical trial(DCT)that leverages digital tools and supporting services,such as eConsent,eCOA,home healthcare,etc.to enable the patient to complete some or all scheduled visits from their home5.4 Types of Clinical Trials Conducted for Rare Disease TherapiesWhat types of clinical trials has your comp

59、any conducted 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

60、or decentralized 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 TrialsResponden

61、ts27 Rare diseases survey 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 sof

62、twareElectronic 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 satel

63、lite clinical trial sitesWearable technologies28 Rare diseases survey 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 using again?(Multiple

64、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 trials?(Multiple ans

65、wers 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/health visits17%In-home

66、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%Electronic informed consent

67、/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 survey Patient Requirements for In-Person Visits During Clinical T

68、rialsDo 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 resources does your company use to network a

69、nd 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%Transportation to/from clinical trial sitesMobilit

70、y assistanceAssistance with medical equipmentAssistance with reimbursement needs35%Medication reminders30 Rare diseases survey Additional Resources Used to Increase/Share Knowledge Online ForumsWhich online forums or community sites do you find most beneficial?(Multiple answers were allowed)NOTE:Tot

71、al 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 list.Rare disease forumsTwitterOnline forumsLinkedInPatient advo

72、cacy sitesDisease specific groupsCompany websiteRare Disease Clinical Research Network(RDCRN)FacebookConferences16%7%15%7%13%4%4%4%4%13%31 Rare diseases survey Additional Resources Used to Increase/Share Knowledge Conferences&EventsWhich conferences/events do you find most beneficial?(Multiple answe

73、rs 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 above highlights the Top 17 answers from that list.Other9%Orphan Drug and Rare Disease confe

74、renceDisease specific conferences9%11%World conferencesWorld Congress on Rare DiseasesAmerican Thoracic SocietyAmerican Society of Hematology(ASH)American Association of Cancer Research(AACR)American Society of Clinical Oncology(ASCO)European Conference on Rare DiseasesNational Organization for Rare

75、 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 Oncology(ESMO)7%5%3%7%7%4%4%4%3%9%3%8%5%3%32 Rare diseases survey Additional Resources Used t

76、o 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 answered“Social media”in Question 5.7.were prompted to answer this.There is a total of 74 items mentioned in this ques

77、tion;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%2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.08.2024 Learn more at