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1、Clinical Trial Trends&Insights 2024Intelligently connecting data and insights to navigate the clinical research trends of tomorrowAdvancing health is all about connections.Connecting people,data,insights,and technology for a better,more efficient clinical trial experience.So as the calendar turns ov

2、er to another year,weve asked our scientific and subject matter experts to connect our data with their expertise and share their thoughts about what trends,changes,and innovations they are looking forward to in 2024 and beyond.While the industry continues to adjust in a post-COVID world,as of this p

3、ublication we expect clinical trial starts in 2024 to be up slightly over 2023.Despite this improvement,disconnects remain.Sites continue to struggle with resourcing and the demands of working in multiple technology platforms.Sites,sponsors,and CROs are working to bridge the gap between artificial i

4、ntelligence,machine learning,drug development,and decision making.All stakeholders are focusing on the continued promise of digital health tools that provide researchers with real-time data,as well as new approaches to clinical trials that prioritize the needs of participants first beyond the protoc

5、ol.In these pages,our experts will connect you with insights to prepare you for what we expect to be another progressive year in the clinical research industry.Foreword2 2024 WCG.All Rights Reserved.Diversity,Equity&Inclusion 4Research Site Readiness 15Decentralized&Hybrid Trials 11EmergingOpportuni

6、ties 26Table of Contents3 2024 WCG.All Rights Reserved.Current Opportunities and Outcomes in Rare Disease Clinical TrialsDiversity,Equity&Inclusion4 2024 WCG.All Rights Reserved.Importance Ratings for Diversityvs.Primary Reasons Diversity wasConsidered to be Important*Racial/EthnicGenderEconomicPati

7、ent JoureyPrimary Reason Diversity is Believed to be ImportantMostly Scientific4.1(50)4.0(68)3.4(20)4.0(71)Mostly Ethical3.9(33)3.9(18)3.1(47)3.9(13)Mostly Regulatory3.9(11)3.6(9)2.3(3)3.5(6)Mostly Marketing3.0(1)-(0)2.2(25)3.2(5)The scientific case for diversity is largely settled.Whats left is con

8、tinuing to make the case with key stakeholders in moving DEI initiatives for clinical trials forward in a meaningful way.In this section,our experts will make the case for the significance of DEI in rare disease clinical trials.ack of diversity in clinical trials can impair quality,increase costs,an

9、d put patient safety at risk.Many therapies work differently depending on a persons gender,race,and ethnicity,so without diverse participants,scientists and clinicians have only a limited understanding of the effectiveness and suitability of treatments for underrepresented populations.These critical

10、 differences are eventually discovered after the therapeutic has been approved and is in widespread use.The barriers to diversity have been well-documented,but despite significant progress,the industry still struggles to overcome them.While everyone agrees on the importance of diversity in clinical

11、trials,the 2021 WCG Avoca State of the Industry Report:Diversity in Clinical Research Execution and Participation found that respondents who saw the pursuit of diversity as a scientific or ethical imperative felt more strongly about its importance than those who considered diversity to be important

12、primarily for regulatory or marketing reasons.Table:Mean(N)importance ratings type diversity 1(no importance)to 5(critically important)Diversity,Equity&InclusionL5 2024 WCG.All Rights Reserved.*2021 WCG Avoca State of the Industry Report:Diversity in Clinical Research Execution and Participation*rar

13、e disease affects,by definition,fewer than 200,000 individuals in the United States,and an ultra-rare disease affects many fewer.1 Altogether,there are more than 10,000 identified rare diseases affecting more than 30 million Americans and their families,with similar numbers in other parts of the wor

14、ld.2 Individuals with rare diseases and their families face significant challenges due to such factors as uncertainty in and availability of a diagnosis and potential treatment options that ultimately affect their medical,psychological,economic,and social health.Considering rare disease prevalence,a

15、 lack of diversity,equity,and inclusion(DEI)considerations in research of these conditions,and practices around treating them leads to diminished opportunity for care and poorer outcomes.These include limited access to diagnosis and care,ongoing clinical trials for a person living with a rare diseas

16、e,their child or their partner,and the availability of support for their concerns and ongoing needs.ASharad Adekar Medical Chair,IRB Operations WCGScott J.Hunter,PhDSenior Scientific Expert,Neurodevelopmental Disorders and Pediatric Rare DiseaseWCG Steve Smith President,Patient AdvocacyWCGDiversity,

17、Equity&Inclusion6 2024 WCG.All Rights Reserved.Patients and families from minority groups,and those who come from less economically stable environments often face significant barriers to safe and accessible health care.These can include a lack of supportive resources,such as parental education and a

18、wareness of diagnosis and treatment options,or available access to ongoing clinical trials for their conditions.Further impacting access is the historical mistrust toward clinical research studies many minority communities hold,a lack of community representation,and limited engagement with patient a

19、dvocacy groups that provide supportive guidance and direction.These limitations can readily lead to a delay in diagnosis and access to available treatment options,impacting potential outcomes.A further challenge for many families,both in urban and rural areas,can be more limited access to resources

20、and facilities where diagnosis and intervention take place.Together,these contribute to diminished opportunities for care and poorer outcomes for diverse communities affected by rare diseases.When we think about social determinants of health-related access barriers,we must consider factors related t

21、o healthcare professional education and knowledge regarding rare diseases.It is the case that many families first rely on available sources of primary care,including family and general practice physicians or nurse practitioners,who may not be as informed about current information regarding rare and

22、ultra-rare diseases.Similarly,primary care practices may not have enough resources or advanced technologies available for faster diagnosis and treatment options.These challenges can be further complicated for minority and under-resourced communities when awareness of specialty care and the availabil

23、ity of practitioners versed in understanding the diverse needs of affected individuals is more limited.Importantly,it has been recognized that there is a significant need to provide healthcare professionals and clinical trial investigators with relevant continuing education and training about the im

24、portance of diversity,equity,and inclusion strategies regarding the diagnosis and treatment of individuals with rare diseases.Starting early with medical and health care training,the integration of curricula regarding the social and behavioral determinants of health has begun to contribute to better

25、 coordination of elements of care,leading to faster diagnosis and,when available,access to developing and approved treatments.Furthermore,improving resources and availability of specialist care and adding greater diverse community representation,such as connecting with patient advocacy groups,have i

26、mproved outcomes for minority persons with rare disease and their caregivers.More specifically,sponsors running clinical trials,including pharmaceutical companies and the individual investigators conducting their studies,have been directed to think more clearly and to state explicitly within their s

27、tudy objectives and design how they will Diversity,Equity&Inclusion7 2024 WCG.All Rights Reserved.directly address diversity and equity considerations.Unique in 2024 is the extent to which emerging practices regarding equitable clinical trials have begun to standardize as drug developers and other s

28、takeholders become familiar with the methods of diversification required of them,which will ultimately lead to a required increase in the numbers of diverse participants who are represented in and serve as beneficiaries of treatment research.During protocol development,sponsors and investigators are

29、 also encouraged to carefully assess research methodologies and approaches,research outreach and recruitment methods,and improve the availability of adequate resources to accommodate minority populations for ease of recruitment.Community-based participatory research methods can support this effort,e

30、nsuring that individuals from minority communities with rare diseases are included in the design and implementation of the research from the start,and by identifying and resolving potential clinical biases.Community-based participatory research uses collaborations between research organizations,inve

31、stigators,and community members throughout all aspects of a research project.This approach is important given its commitment to engaging and representing intersectionally diverse populations affected by rare and more common diseases and fostering greater engagement across minority communities.Trial

32、diversity and rare disease drug development together will benefit from the growing collaboration by the FDA regarding the necessity for diverse and equitable representation in clinical trials and biotech companies increasing familiarity with the resulting best practices in development.Tools to reduc

33、e the diagnostic odyssey will continue to reduce the cost and burden of rare diseases,while advocates work with policymakers will better open access to these tools and strategies.The new DEPICT Act passed recently by the U.S.Congress requires the FDA to require sponsors to submit Diversity Action Pl

34、ans with their Phase III or other pivotal trials.The FDA has urged both large pharmaceutical companies and the growing number of smaller biotech drug developers involved in rare disease research to reach out and work with them early in Diversity,Equity&Inclusion8 2024 WCG.All Rights Reserved.Unique

35、in 2024 is the extent to which emerging practices regarding equitable clinical trials have begun to standardize as drug developers and other stakeholders become familiar with the methods of diversification required of them.the investigatory process to develop protocols and find solutions to the chal

36、lenges this new requirement presents.The DEPICT requirement comes at a time when the industry is a few years further down the road from the events of 2020,including the COVID-19 pandemic,which awakened a new,sincere investment in being more inclusive in health care across all stages of development a

37、nd ultimate treatment,including clinical research.2024 is a specific year to watch this investment translate directly into best practices.Additionally,better tools in genomic screening are available now,which help reduce the diagnostic odyssey and connect rare disease patients with new treatment opt

38、ions and early interventions that can save their lives and prevent unnecessary damage from the disease.Policies to open access to these fresh solutions more broadly across diverse affected communities will remain an active focus for rare disease advocates and collaborating policymakers in 2024.A gro

39、wing body of evidence shared recently has shown how policies that help families of rare diseases also benefit society.Two studies commissioned by the EveryLife Foundation for Rare Diseases3,4 will be used in dialogue with policymakers:one quantifies the cost burdens on families and society of rare d

40、iseases,and the other quantifies the avoidable costs of the diagnostic odyssey.Legislation is in the works to provide access to treatments and diagnostic tools to people regardless of zip code and income level.Watch for legislative efforts to open access to newborn screening,including rapid genome s

41、equencing,genetic counseling,and early intervention services.Small patient populations have always hampered Diversity,Equity&Inclusion9 2024 WCG.All Rights Reserved.rare disease research.As we expand our definition of who participates in the research and its benefits,the population sizes grow.Lastly

42、,and important to how the process regarding greater diversity and equity in clinical research will unfold,the FDA has already issued draft guidance on enhancing the diversity of clinical trial populations,providing recommendations to sponsors to enroll representative numbers of participants from und

43、errepresented racial,ethnic,gender diverse,and economically diverse populations in the United States.This is being further supported by Institutional Review Boards(IRBs)reviewing current research proposals with an eye toward DEI and representative justice.IRBs play a key role in determining the avai

44、lability of clinical trials for minority populations in rare diseases.One of the criteria for approval is consideration of the equitable selection of research subjects.ADDITIONAL WCG THOUGHT LEADERSHIPDiversity,Equity&InclusionARTICLEFDAs Path Toward Diversity in Clinical Trials:The DEPICT Act and S

45、ponsor ResponsibilityPODCAST Breaking Down Barriers:How DE&I,Technology,and Protocol Design Impact Enrollment SuccessPODCAST The Importance of Diversity,Equity,Inclusion,and Intersectionality in Clinical ResearchAs per the Belmont Report,no individual group should be absolutely included or excluded

46、from clinical study without justifiable scientific or ethical reasoning.IRBs can review the submitted justification and study design for scientific and ethical validity,10 2024 WCG.All Rights Reserved.and ensure adequate safeguards and protections are in place for the study population more broadly.T

47、his will lead to both greater recruitment of diverse participants and clearer knowledge regarding potential outcomes.The Decentralization of Vaccines in 2024 and Beyond Decentralized&Hybrid Trials11 2024 WCG.All Rights Reserved.FDA has created a framework that includes workshops,demonstration projec

48、ts,stakeholder engagement,a website,and internal processes to evaluate DCTs and hybrid trials.In this section,our expert outlines the impact of DCTs and hybrid trials on vaccines,and how we can continue to bring the promise of more patient-centric care through DCTs.he COVID-19 pandemic drove interes

49、t in decentralized(DCT)and hybrid trials.As the world emerges from the global pandemic,the utility of DCT elements continues as DCT and hybrid trials enable the delivery of a more participant-centric approach to research,as treatments can be delivered remotely,sponsors can engage with a more diverse

50、 patient population,and recruitment efforts can be accelerated.But while digital devices,mobile applications,and online communication methods are useful tools,they are not complete solutions.The launch of Apples ResearchKit facilitated the enrollment of thousands of participants into research progra

51、ms on a wide variety of chronic diseases,but several weeks later,about 90%of initial enrollees had dropped out of the project.As such,its important for the industry to determine the right mix of virtual and on-site care.From a regulatory perspective,if the protocol is designed as a DCT or a hybrid t

52、rial,some or all research activities may occur at locations that are not traditional clinical trial sites.For example,a local clinic,a mobile unit,or the participants house may serve as the location for some research activities.In 2023,the U.S.FDA issued its guidance on the design and implementation

53、 of DCTs and updated its position on using electronic systems,records,and signatures in clinical investigations.In addition to guidance development,the DCT Adoption 2020-2021*04%20%09%01%DCT Adoption 202201%03%13%48%36%36%30%Much more utilization than previously36%Somewhat more20%About the same09%So

54、mewhat less01%Much less utilization than previously04%Dont know/NA36%Much more utilization than currently48%Somewhat more13%About the same03%Somewhat less00%Much less utilization than currently01%Dont know/NA30%According to a Clinical Trials Arena analysis,the incorporation of decentralization compo

55、nents in clinical trials was expected to rise by the end of 2023.Insights for 2024Decentralized&Hybrid TrialsT12 2024 WCG.All Rights Reserved.*Clinical Trials Arena DCT Adoption Trackereveloping protective vaccines for endemic and emerging infectious diseases is a top priority for global health and

56、a promising area for innovation with decentralized clinical trials(DCTs).A DCT is a trial where some or all research activities occur at locations other than a traditional clinical trial site.These activities often include digital screening,remote enrollment,and remote participant monitoring.The res

57、ponse to the COVID-19 pandemic included the rollout of a variety of innovative hybrid approaches to vaccine trials.As one example,participants were able to collect dried blood spots in the home setting for immune monitoring and to determine the development of vaccine-induced antiviral antibodies.The

58、 potential for remote monitoring for the efficacy of infection and safety events has been clearly demonstrated,and these approaches will play more prominent roles in the coming years.An emerging area under consideration is direct-to-participant investigational product delivery.With the ongoing devel

59、opment of oral,intranasal,and microneedle array patch vaccine delivery systems,at-home vaccine self-administration is becoming more plausible.The FDA is currently considering allowing at-home self-administration of an approved intranasal live attenuated influenza vaccine.However,for investigational

60、products,significant concerns related to safety,shipment,stability,privacy,blinding,and compliance create barriers to the rapid adoption of comprehensive direct-to-participant vaccine testing approaches.DDaniel Kavanagh,PhD,RAC Senior Scientific Advisor,Gene Therapy,Vaccines,and Biologics WCG Decent

61、ralized&Hybrid Trials13 2024 WCG.All Rights Reserved.Many of the most promising new vaccine technologies involve genetically modified products such as recombinant or synthetic mRNA or DNA,or viral and bacterial vectors.In the U.S.,Institutional Biosafety Committees(IBCs)usually oversee the safe hand

62、ling,administration,and disposal of genetically modified investigational vaccines.IBCs are traditionally based at research institutions and fixed clinical trial sites,and IBC approval requires assessment of facilities,equipment,and procedures at each site.One solution to the limitations outlined abo

63、ve is the use of mobile research units.Mobile facilities housed in vans and trailers can bring research capabilities closer to the participants while allowing for careful management of product handling,safety,and privacy concerns.Importantly,with appropriate documentation and advanced planning,mobil

64、e research units can be reviewed and approved by an IBC,bringing cutting-edge research to neighborhoods and community settings.Regarding pandemic preparedness,flexible and adaptable clinical trial platforms will be critical for an agile and timely response to the next emergency.Increasing decentrali

65、zation of vaccine trials in 2024 and beyond will play a key role in enabling that flexibility.Decentralized&Hybrid TrialsADDITIONAL WCG THOUGHT LEADERSHIPPODCAST Diversity&Inclusion with Technology in Decentralized Trials14 2024 WCG.All Rights Reserved.Supporting Research Sites and the Road AheadRes

66、earch Site Readiness15 2024 WCG.All Rights Reserved.several key factors,including protocol design,training,participant recruitment and enrollment,and more.Likewise,sites can optimize and grow their research businesses by harnessing solutions that improve site capacity,reduce start-up delays,and less

67、en their administrative burden.By leveraging site enablement solutions,sites can increase operational efficiency,recruit,and retain more participants sooner,deliver quality data,and improve their financial performance.Read on for insights into how sponsors and CROs can better enable the research sit

68、es conducting their studies and how sites can address site capacity constraints.he evolution of clinical research has been dependent on the conversion of Healthcare Organizations(HCOs)to clinical research sites,along with the conversion of Healthcare Providers(HCPs)to Principal Investigators(PIs).Th

69、ese conversions are critical to advancing medicine and providing cutting-edge treatment to patients.However,the current clinical research landscape has become increasingly intricate due to complex protocols,demanding data collection needs,and constricted study timelines,causing a slowdown in the con

70、version of HCOs and HCPs.As a result,the number of clinical trials now being initiated exceeds the amount of clinical research sites available to conduct these trials.Because of this,active clinical research sites have become saturated and are facing unrelenting pressure to meet protocol endpoints w

71、ithin the timelines dictated.With only 15%of HCOs conducting clinical research according to industry sources,sponsors and CROs must ensure existing study sites are fully enabled to conduct efficient studies so protocol endpoints can be successfully met.To do this,sponsors and CROs must enable their

72、study sites from the very beginning.Effective site enablement must address 14182224Months longer on average to complete a clinical trial in 2023 compared to 2020102020202220212023Average Length(Months)of Clinical Trials*Research Site ReadinessT16 2024 WCG.All Rights Reserved.*WCG Data Intelligence&2

73、022 Tufts Center for the Study of Drug Developmenthe tumultuousness of the pandemic years has lessened,giving rise in 2024 to an intense focus on people,timelines,and quality in conducting clinical research.Near the end of 2023,research sites reported more stabilization in the research workforce,con

74、sistent with the U.S.Bureau of Labor Statistics report of declining resignations in healthcare(see graphic).Since 2020,the most critical concern at research sites has been workforce retention and recruitment.The availability of qualified research professionals will remain a top concern in 2024.Innov

75、ations in workforce development will continue to expand through research sites,professional organizations,and partnerships as the industry highlights the role of the clinical research professional as an intentional career choice(see above).Aside from workforce issues,clinical trials sites continue t

76、o face numerous TA Look Ahead at Research Sites in 2024Sandy Smith,RN,MSN,AOCN Senior Vice President,Clinical Solutions and Strategic PartnershipsWCGheadwinds ranging from capacity limitations to increasingly complex protocol designs and the inability to meet trial enrollment targets and timelines.T

77、hese factors contribute to the increasing trend in clinical trial completion taking an average of ten months longer to complete in 2023 vs.2020.5 U.S.Trial Starts and Healthcare Resignations*Workforce Development InnovationsSite-based training/InternshipsCollaborations with universities forcertifica

78、te programsCommunity college training programsTraining high school graduates as CRCsRemote/virtual training for regulatoryfunctionsNew investigator trainingInitiation of new research sites0501001502002503003504000100200300400500600700JanMarMayJulSepNovJanMarMayJulJanMarMayJulSepNovMayJulSepNovJanMar

79、2020202120222023Trial StartsResignationsResearch Site Readiness17 2024 WCG.All Rights Reserved.*2023 WCG Data Intelligence&2023 U.S.Bureau of Labor Statistics Report on Declining Healthcare ResignationsWhile the impact of The Great Resignation has lessened,clinical research sites and pharmaceutical

80、company partners are mutually invested in accelerating clinical trial activation.Focusing on oncology,a therapeutic area representing over 40%of the sponsored trials opened in 2023,5 guidance from the National Cancer Institute(NCI)suggests a target activation timeline of 90 days.Some sites report me

81、eting or exceeding this target,but far more are establishing action plans to reduce their activation times currently surpassing 100,200 or even 300 days.Across all therapeutic areas,there is disparity in median time for trial activation,defined as time from site selection to completion of contract.F

82、or the past 3 years for Phase I-III trials,the median timeframe for Academic Medical Centers(AMCs)and hospitals is 8.12 months vs.independent sites/physician practices with a median of 4.37 months.5 With many steps and variables in the start-up process,one task consuming weeks to months is the negot

83、iation process for both budgets and contracts.Budget negotiation timelines trended an average of eight days longer in 2023,5 likely impacted by higher site costs due to inflation.Concentrated efforts on improving activation timelines through workflow optimization will include use of centralized ethi

84、cal and biosafety review,deployment and linking enabling technologies,evaluating options for outsourcing administrative services,and enhanced communication in negotiations.Research Site Readiness18 2024 WCG.All Rights Reserved.While the impact of The Great Resignation has lessened,clinical research

85、sites and pharmaceutical company partners are mutually invested in accelerating clinical trial activation.he time it takes to finalize clinical trial agreements(CTAs)is among the most pressing challenges in clinical trials,but our industry proved during the COVID-19 pandemic that rapid improvement i

86、s possible.CTAs that previously took months to negotiate before the onset of the TRecapturing the Progress Made with CTA Negotiations During the PandemicLuke Goodpaster Director,Site NetworkWCG Collin Gruener Operations Manager,Site Network WCG Research Site Readinesspandemic were suddenly finalized

87、 in a matter of days.However,CTA negotiations have largely reverted to pre-pandemic practices just as research on treatments and vaccines have allowed society to return to some form of normalcy.With trial volume normalizing after a post-pandemic boom and pharmaceutical sponsors and healthcare provid

88、ers reconsidering their clinical trial portfolios and resources,those involved in clinical trial contracting have an opportunity to refine their processes and standards in a way that would bring us closer to those rapid turnarounds we saw during the pandemic.We envision stakeholders seeking more opp

89、ortunities to use a template or another source of agreed-upon language,including previous CTAs between parties and industry-recognized templates like those from ACTA and MAGI.While some sponsors may,for example,deem ACTAs approach to GDPR or IP ownership insufficient to protect their interests,these

90、 types of issues do not preclude the use of templates.It just means some terms,but not all,may require adjustments before final agreement is reached.At the very least these resources can provide a great starting point,allowing the parties to focus on key issues,as was the case when the industry turn

91、ed to those resources for rapid start-up on COVID-19 trials.Contract teams also will look for ways to become more efficient in 2024 when templates are not used,and two CTA negotiations have largely reverted to pre-pandemic practices just as research on treatments and vaccines have allowed society to

92、 return to some form of normalcy.19 2024 WCG.All Rights Reserved.ways to improve efficiency are through reprioritization of workload and revising internal standards,such as the CTA playbook.By prioritizing CTAs that are nearly complete ahead of new CTAs,you can shorten negotiation timelines and prev

93、ent overaccumulation of contracts on your to-do list.In other words,do not automatically relegate incoming tasks to the bottom of the priority list;focus on reducing the size of your to-do list by resolving those CTAs that can be finalized quickly.Just as we prioritized our vaccine and treatment tri

94、als during the pandemic,prioritizing those CTAs that are closest to finalization can have a tremendous effect on turnaround.Drafting an adaptable CTA playbook that works within a wide variety of CTA templates will help set your contracts team up for efficient CTA negotiations in 2024 and beyond.Focu

95、s on key words,phrases,and concepts,with examples of agreeable text to help guide the CTA reviewer.Avoid mandating Research Site ReadinessDrafting an adaptable CTA playbook that works within a wide variety of CTA templates will help set your contracts team up for efficient CTA negotiations in 2024 a

96、nd beyond.20 2024 WCG.All Rights Reserved.long blocks of text that are required verbatim.Allowing your contracts team to work key ideas and terms into the existing template language leads to more productive conversations with the opposing side.The first step to accomplishing that goal is strategical

97、ly drafting or revising your CTA playbook.Lastly,with efficiency as a priority,teams should be willing to pick up the phone earlier and more frequently.Too often“negotiations”happen in the comment bubbles,especially when the reviewer is short on time.This method,while convenient,is inefficient.It le

98、ads to prolonged discussions and more rounds of ineffective back and forth.Simply removing and replacing language with comments such as“not approved”is not negotiation,and it certainly does not constitute collaboration.A phone call allows both parties to clarify points in real-time,which leans more

99、toward collaborative problem-solving than combative dispute.The decrease in CTA negotiation time that was experienced during the pandemic was more easily obtainable as all parties had the same shared goal of supporting COVID-19 clinical trials.We cannot forget that patients who suffer from any disea

100、se or disorder also experience a disruption to their daily lives.While one specific health issue may not have as widespread of an impact as a pandemic,all parties should be able to continue to share this same goal.Focusing on common goals can change negotiations from an adversarial relationship to a

101、 team approach.It is important to move away for the“us vs.them”mentality.The clinical research ecosystem requires all parties of a CTA to work together to be successful.Research Site Readiness21 2024 WCG.All Rights Reserved.Vaccine research will forever be linked to the COVID-19 pandemic.For all the

102、 heartache and troubles the pandemic brought us,it showed the collective power our industry holds when unified for a singular focus.While the pandemic brought new awareness to vaccine research,it also elevated the expectations of sites without accounting for their own aspects of pandemic challenges.

103、At the height of COVID-19 vaccine research,everyone was on board:site staff,sponsors,CROs,vendors,and most of all,patients,unified for a singular focus.Our industry,and the world,saw incredible speed and results.Now that the world has progressed and individual factors are no longer aligned,the goal

104、of vaccine research stays the same.Because of this,the next year will see the continued trend of high expectations,with traditional resource constraints in place,bringing the need to focus on enhancing efficiency at sites.Sites can maximize resources by leaning into the lessons learned through the p

105、andemic.One practice that enabled the speed of pandemic vaccine research is the clear delegation of duties at sites.Recruitment and retention needs are not mutually SITE READINESS IN VACCINE TRIALS:Lessons Learned from the COVID-19 Pandemic Tyler Bye Director,Site Solutions and Product StrategyWCGRe

106、search Site Readiness22 2024 WCG.All Rights Reserved.exclusive,but sites should clearly assign the tasks associated across the patient journey to team members.These specific tasks require not just delegation,but dedication from team members.When the team knows who carries each responsibility,members

107、 can focus on their specific tasks and work efficiently.The concept sounds simple,but putting it into a repeatable practice takes effort and specialization.The next year of vaccine development will continue to focus on respiratory diseases,including new variants of influenza and COVID-19.The year wi

108、ll also see a growing focus on new and emerging diseases such as dengue fever and Zika,and advances in bacterial and viral indications,including meningococcal,hepatitis,and chicken pox.Each specific indication will require sites to focus on healthy but at-risk populations.Sites must continue engagin

109、g with the target population to bring awareness of these vaccine areas and ensure the efficacy endpoints can be achieved.The industry desires efficiency.Sites can expect sponsors to work on bringing efficiencies through new and consolidated technology.From a sites perspective,some will work,some wil

110、l not,and the ongoing trend of technical issues will persist.Still,sites should work openly through the challenges and approach changing technologies with the basics of the scientific method.There is a hypothesis that changing technology will benefit research.To continue to advance as an industry,we

111、 all need to go through the methodology to determine if the results support the hypothesis.Vaccine research in 2024 will continue to press forward,and sites will continue to find ways to be more efficient in their processes.The pandemic proved what is achievable,and efficiencies were born from the p

112、rocess.With the mindset of continuous process improvement,patients will be the beneficiaries of the work to come.Research Site Readiness23 2024 WCG.All Rights Reserved.Patient Recruitment Barriers in Oncology Jamie Harper,MHA,CCRP Vice President,Site Solutions and Engagement WCGAs the industry conti

113、nues to recognize advancements in oncology,it also continues to recognize the challenges in patient recruitment,a vital component in bringing these advancements to market.Patient recruitment comprises two distinct components identification and enrollment with each component bringing forth its own se

114、t of challenges.While often overlooked,understanding how patients will enter the study is the first step in developing a successful recruitment strategy to identify potentially eligible patients.The referral pathway can be internal to the research site through electronic medical records(EMR),through

115、 physicians within the same healthcare organization(HCO),or externally through physicians outside the HCO.Internal referral pathways using EMR are the easiest means to identify potential patients,but it takes time and experience.Building a comprehensive query within the EMR can be challenging due to

116、 limitations of the platform and experience of the research staff.Entering complex eligibility criteria commonly found in oncology studies often requires medical record“superusers”to build algorithms for capturing the appropriate patient population.It takes ample resources with the required expertis

117、e to properly query the sites EMR to obtain a list of highly eligible patients for review and potential enrollment.A properly managed referral pathway continues to the enrollment stage of recruitment.Once highly eligible patients are identified,ensuring they are informed and engaged is critical to s

118、uccessful enrollment and retention.Building a rapport and solid foundation for communication requires patience,understanding,and time.There is a heavy focus on the administrative components of clinical research and the increase in those requirements.The administrative tasks completed in the backgrou

119、nd are essential to maintaining regulatory compliance but may cause study teams to sacrifice the time needed to build relationships with enrolling patients.Assigning a designated team to address administrative and compliance requirements is impractical for most research sites.Collaborating with a Re

120、search Site Readiness24 2024 WCG.All Rights Reserved.While often overlooked,understanding how patients will enter the study is the first step in developing a successful recruitment strategy to identify potentially eligible patients.vendor can ensure potential research participants are well-informed

121、and engaged while also supporting administrative and regulatory compliance.As complex eligibility requirements continue to be a component of clinical oncology research protocols,developing a strong and comprehensive patient recruitment plan is the first step to mitigating patient identification and

122、enrollment barriers.Utilizing internal resources in combination with the right external partners can help research sites continue the drive forward in conducting clinical research and propel oncology treatments to the next level.Research Site ReadinessADDITIONAL WCG THOUGHT LEADERSHIPSURVEY REPORT 2

123、023 Clinical Research Site Challenges Survey ReportPODCAST Unlocking Site Potential:Reducing Site Burden and Enhancing Clinical Trial Efficiency25 2024 WCG.All Rights Reserved.Psychedelics,Artificial Intelligence,and Digital BiomarkersEmerging Opportunities26 2024 WCG.All Rights Reserved.Digital Bio

124、markers Digital biomarkers are behavioral and physiological data such as heart rate,physical activity,and step counts collected using digital devices.These measurements can be reflective of treatment response,enabling a better understanding of disease progression,and they have the potential to repla

125、ce time-consuming assessments that can be difficult for patients.While this technological shift could introduce risks to clinical trials,it offers long-term benefits to patients and researchers alike when safely developed,assessed,and adopted.Psychedelics A recent search of clinicaltrials.gov found

126、nearly 200 clinical trials of psychedelics currently registered.The FDA published its first draft guidance on this topic in 2021,and an expanding body of research suggesting that psychedelics may treat CNS disorders like substance abuse,depression,chronic pain,and schizophrenia has sparked a renewed

127、 interest in recent years by drug developers and investors.This emerging area of research presents exciting opportunities to address important unmet medical needs for many disorders,including CNS.While there are some significant challenges,the potential to make significant strides in improving patie

128、nts lives provides a sense of optimism for the future of the field.On the following pages,our experts will dive deeper into these emerging topic areas of interest.Insights for 2024Artificial Intelligence The reach and impact of artificial intelligence(AI)throughout the healthcare industry has been s

129、teadily growing.From disease diagnosis to the tailoring of treatment plans,AI is starting to appear everywhere.But where is AIs place in clinical trials?According to the Tufts Center for the Study of Drug Development,a typical Phase III clinical trial generates more than 3.5 million data points.This

130、 finding highlights the need for artificial intelligence to manage these massive data sets to deliver insights for better,more timely decision making.From a regulatory perspective,in 2023 the FDA published a discussion paper outlining the current and future applications of AI and machine learning(ML

131、)in drug development.This paper addressed core issues like human-led governance,data quality,and model development standards.It emphasized a risk-based approach tailored to AI/ML use,emphasizing the importance of accountability and transparency.The FDA is requesting stakeholder feedback to help info

132、rm future regulatory activities.It is anticipated that AI will impact all areas of clinical research including protocol development and ethical review of research.Emerging Opportunities27 2024 WCG.All Rights Reserved.he past five years have been full of important milestones for neuroscience clinical

133、 development,from the approval of the first rapid-acting antidepressant,Janssens esketamine(Spravato)to the publication of the industrys first late-stage clinical trial of psilocybin by Compass Pathways.6 Many of these trends will continue,but they will be accompanied by larger,epoch-scale changes t

134、hat may permanently alter the way we do research.Psychedelics,the most exciting new trend in neuroscience,are not exactly new they have been used all over the world for thousands of years,predating the rise of modern industrial medicine.Legal and conceptual frameworks to allow their use in neuroscie

135、nce research have only recently permitted widespread study,bringing them to the brink of potential approval by the FDA for therapeutic applications.In parallel with the rise of psychedelics and the continued advances in new treatments for schizophrenia,PTSD,and anxiety disorders,we have seen the evo

136、lution of a new area of technologies TMark Opler,PhD,MPH Chief Research Officer,Clinical Research Solutions WCG collectively referred to as digital biomarkers.The confluence of these two trends,coupled with recent research on novel mechanisms of action,will likely help foster large-scale changes in

137、how neuroscience studies are conducted,particularly in the field of psychiatry.The central challenges of psychiatry clinical trials have been two-fold:first,the eternal limitations of finding appropriate research participants for disorders that are diagnosed based on subjective criteria and syndromi

138、c presentations;and second,the Past Promises,the Future Hope of Neuroscience Clinical Research Emerging Opportunities28 2024 WCG.All Rights Reserved.difficulty of determining efficacy with measurement tools which require significant training and resources to use correctly,and even in the hands of an

139、 expert are often inaccurate and prone to bias.Rapid-acting antidepressant research has catalyzed a re-examination of study endpoints7 and hints at a new method for bridging the gap between objective evaluations,which will enable efficient,sensitive evaluations at scale and the existing validated me

140、thods of symptom assessment has emerged in the form of vocal analytics.This field has roots in the 1960s and 70s when researchers used pauses in speech as a measure for neuromotor function.8Recent publications by Johns Hopkins University and others point to the use of simple,valid measures,such as s

141、peech latency,as being useful methods for automating objective markers of pathology in depression and bipolar disorder.Future work on this approach may lead to smaller,more accurate trials that have meaningfully better statistical power(Siegel et al.)and can be conducted more efficiently and ethical

142、ly.9The next year will bring more reports of continued success with psychedelics and a further crystallization of their unique modality.They may increasingly come to be appreciated as“synaptogenics,”or compounds that promote the reorganization of the brain,while also demonstrating renewed attention

143、to non-drug therapies as most psychedelics are being packaged with various structured psychotherapies and supportive therapies.An unintended benefit of the psychedelic revolution Emerging Opportunities29 2024 WCG.All Rights Reserved.might well be the renewed interest in holistic approaches to treatm

144、ent and a rediscovery of the psychiatric patient as a psychosocial entity,not simply a neurobiological test subject.Progress is not without its problems.While the past decades have been a time of incredible technological transformation of neuroscience research,the impact on investigative site staff

145、has been challenging.A poster by Cohen et al.10 shows that the stress of managing technology burdens falls disproportionately on the shoulders of those clinicians assessing patient symptoms.The demand for high-throughput data collection at scale with strong reliability and accuracy,on top of all the

146、 other tasks required by investigators,may finally force the field to consider the limits of this approach.In conclusion,2024 may be the year that neuroscience clinical research rediscovers its future in the promises of the past.From the approval of psychedelics known to humanity for thousands of ye

147、ars for their potent neuroactive properties to the renewal of speech analysis as a method of evaluating patient status and the promise of smaller,more humane studies that allow patients and investigators to fully participate in the benefits of our work.With hope and optimism that is hopelessly old-f

148、ashioned,we look forward to the coming year.2024 may be the year that neuroscience clinical research rediscovers its future in the promises of the past.Emerging Opportunities30 2024 WCG.All Rights Reserved.Psychedelics:From Powerful Potential to the Psychiatrists PracticeCurrien MacDonald,MD,CIP Med

149、ical Chair Director,IRB Operations WCGThe list of mental illnesses with high disease burdens and limited effective treatments may be shrinking soon.Psychedelics show great promise in being able to address multiple illnesses plaguing society,and the research investigating this is not fringe science b

150、lowing smoke.For example,in 2023 the Office for Human Research Protections held its sixth annual exploratory workshop11 on the ethical and practical considerations of psychedelics research.Also,the FDA granted breakthrough therapy designation in 201712 to the Multidisciplinary Association for Psyche

151、delic Studies(MAPS)for post-traumatic stress disorder,and those active MAPS trials may lead to an approval soon.Moreover,the FDAs first draft,Guidance on Clinical Trials with Psychedelic Drugs,13 released in June 2023,shows the way forward for others looking to harness the potential power of these s

152、ubstances and bring them to market.Truth,it may be a long trip.Psychedelics are still Schedule I drugs,and clinical trials with that class are challenging without other associated issues.While psychedelics may have some utility as just another medicine,their power is likely best realized in a specif

153、ic,somewhat unique psychotherapy combination.Research experts in this area,such as those at MAPS and Johns Hopkins University,14 have amassed substantial experience;however,realizing psychedelics full potential will take many more extensive trials.Classically trained psychiatrists will need new know

154、ledge,training,and support to conduct these trials.Additional stafflikely also requiring substantial training and supportwill be needed to monitor and gather what is often subjective data.Having those tools and being ready for the necessary challenges will ensure that the promise of psychedelics isn

155、t just a pipe dream.Emerging Opportunities31 2024 WCG.All Rights Reserved.ADDITIONAL WCG THOUGHT LEADERSHIPPODCAST Delve into the World of Psychedelic Research and Ethical InquiryThe Impact of Artificial Intelligence(AI)on Ethical Review by IRBs Donna Sny yder,MD,MBE Executive Physician,WCGThe integ

156、ration of Artificial Intelligence into all aspects of society is moving at lightspeed and is certain to impact the ethical review of clinical research.Currently,there are no federal regulations specific to AI for Institutional Review Boards(IRBs)to follow,but the development of guidance and best pra

157、ctices is likely to evolve over the next few years as IRBs encounter AI applications in all aspects of protocol review.Many research projects that include AI may be exempt from IRB review,either because the study involves secondary use of data without interaction with a human subject or the data is

158、de-identified.However,The Secretarys Advisory Committee on Human Research Protections(SACHRP)recently opined on this issue and noted that the“AI/ML machine learning and BD big data research expose the limits of the traditional concept of identifiability that serves as the basis for privacy protectio

159、ns under the Common Rule.”15 As technology advances,what was considered private in the past may no longer be,so IRBs may need to evaluate the data sources used in research more closely and consider whether adequate protections are in place before establishing that a protocol is exempt from IRB revie

160、w.Once a protocol is determined to require IRB review,there are a variety of areas where AI might be incorporated into the research.For example,AI may be incorporated into software as part of a medical device.16 It may be used as part of drug and biologics development.17 It could be used to help sel

161、ect what participants might be enrolled in a clinical trial,or it may be integrated into digital technologies18 as a study outcome measure.The IRB is tasked with evaluating how AI will impact the IRBs analysis of benefit and risk,particularly harm related to the validity and bias of models used to t

162、rain AI19,as well as how the privacy of the individual will be protected in the research.The IRB is also tasked with ensuring that the informed consent form and process includes information on the use of AI as part of the research if AI is being used to make treatment decisions during the study and

163、is clear on any secondary use of data after the study is complete.IRBs may consider having an individual with expertise in AI/ML available for consultation for complex protocols utilizing AI to ensure that participants are adequately protected.Emerging Opportunities32 2024 WCG.All Rights Reserved.De

164、ploying AI Across the Clinical Trial Life Cycle Paul J.Mancinelli,PhD Chief Technology Officer,WCGArtificial Intelligence(AI)techniques across many differentiating industries and domains have grown significantly over the last few years.As in other industries,AI offers great promise across the clinic

165、al trial landscape to address common challenges like recruiting and retaining clinical trial participants,identifying the right sites for a particular study,and predicting outcomes to improve the likelihood of a clinical trials success.Looking ahead to 2024,technological advancements in AI,particula

166、rly with generative AI and Large-Language Models(LLMs),will continue to grow due to computing availability and the improvement of algorithmic techniques.These advancements in AI,coupled with technology,can address the many challenges and pain points across clinical trials.According to the National I

167、nstitutes of Health,20 traditional clinical trial design and execution are time-consuming and inefficient because of many manual processes,leading to only 10%associated success rate of drugs that receive FDA approval.Given that the drug pipeline has more than 6,000 molecules in clinical development,

168、sponsors require a massive amount of energy,resources,and funds to get these through all phases of trials.The current technology landscape provides the industry an opportunity to pivot to data and AI-driven solutions that are highly enabled by technology.This means not just digitizing a paper proces

169、s but reimagining the clinical trial life cycle.The key challenges in a trial life cycle have remained the same for several years and will only continue to get more intractable if not addressed.Finding and keeping patients,determining optimal sites,data and document management,as well as detecting a

170、nomalies in data and the associated time to take corrective actions,will be further exacerbated if the replication of inefficient trial designs continues to reinforce this suboptimal overall process.Emerging Opportunities33 2024 WCG.All Rights Reserved.Several critical areas of the clinical trial li

171、fecycle exist where AI solutions,including Machine Learning(ML)and LLMs,can alleviate these challenges.Within design and start-up,there is a massive opportunity to use components of AI during document creation and management,such as document review,trial and protocol design,consent,contracts,and stu

172、dy review.LLMs trained on historical documents,combined with indexing,can generate optimal index search criteria,creating a powerful tool to generate documents efficiently and find information quickly.These techniques offer massive time savings in document management and locating key data points and

173、 elements within documents.True intelligent automation or augmented intelligence,through the application of effective AI techniques,makes staff more efficient and effective,offering significant improvements in efficiency across an entire organization.Traditional ML algorithms can be used for site fe

174、asibility,site identification,and patient recruitment.In a broader sense,when given a trial protocol,plus historical trial and site performance data,one can determine the optimal set of sites and patients for a given trial.Using ML to identify sites and optimize the identification of patients,there

175、is a 70%improvement in average response rates from sites willing and able to join trials versus an industry average of 30-50%.By implementing ML techniques,studies have also seen patient recruitment accelerated by 44%.In the area of evidence generation,such as electronic Clinical Outcome Assessment(

176、eCOA)and electronic Patient-Reported Outcome(ePRO),monitoring for and detecting anomalies is crucial for keeping a clinical trial on track with a higher likelihood for success.Applying ML to eCOA and ePRO data in real-time as it is flowing in allows anomalies to be detected and addressed quickly and

177、 efficiently.With these techniques,clinical endpoints have a 65%reduction in error rates,drastically improving the likelihood of a trials success.Through the use of AI from ML to generative AI clinical trials can run more effectively,efficiently,and with higher quality.The application of AI to well-

178、known challenges in clinical trials will alleviate the issues to a great degree in the year ahead and bring life-saving drugs and therapies to society more quickly,ultimately saving lives.Emerging Opportunities34 2024 WCG.All Rights Reserved.Using Machine Learning to identify sites and optimize the

179、identification of patients,there is a 70%improvement in average response rates from sites willing and able to join trials versus an industry average of 30-50%.1.Bainbridge,M.N.(2020).Determining the incidence of rare disease.HumanGenetics,139,569-574.DOI:10.1007/s00439-020-02 135-5.2.Smith,C.I.E.,Be

180、rgman,P.,&Hagey,D.W.(2022).Estimating the number ofdiseases the concept of rare,ultra-rare,and hyper-rare.iScience,25,104698.DOI:10.1016/i.isci.20 22.104 698.3.The national economic burden of rare disease in the United States in 2019.4.The Cost of Delayed Diagnosis in Rare Disease-a Health Economic

181、Study.5.WCG Data Intelligence,2023.6.Goodwin et al,Single Dose Psylocibin for a Treatment-Resistant Episode ofMajor Depression New England Journal of Medicine,November 3,2022.7.Yavorsky et al,Recommendations for Selection and Adaptation of RatingScales for Clinical Studies of Rapid-Acting Anti-Depre

182、ssants,Front Psychiatry,June 2023.8.E.Szabadi et al,Elongation of Pause Time in Speech:A Simple,ObjectiveMeasure of Motor Retardation in Depression,Cambridge University Press,January 2018.9.Cohen et al,Measuring Depression Using an Automated Natural SpeechAnalysis Pipeline:A Potential Clinical Trial

183、 Enrichment Tool,Poster Presentedat the 2022 ISCTM Meeting.10.Cohen,E.A.,Montero,J.,Grindell,V.M.,Wyka,K.,Hassman,H.H.,Walling,D.P.,Blanchard,C.L.,Opler,M.G.,Michoulon,D.,and Ereshefsky,L.(November,2023).Global raters work stress and task burnout:An empirical explorationof our primary endpoint evalu

184、ators.Poster presented at the Annual CNS Summit Scientific Meeting,Boston,MA.11.Department of Health and Human Services,Office for Human ResearchProtections,2023 OHRP Exploratory Workshop,Exploring the Ethical&Practical Considerations of Psychedelics Research,September 2023.12.Multidisciplinary Asso

185、ciation for Psychedelic Studies,FDA GrantsBreakthrough Therapy Designation for MDMA-Assisted Therapy for PTSD,Agrees on Special Protocol Assessment for Phase 3 Trials.13.U.S.Food and Drug Administration,FDA Issues First Draft Guidance onClinical Trials With Psychedelic Drugs,June 23,2023.14.Johns Ho

186、pkins Medicine,Psychedelic Research and Psylocybin Therapy.15.U.S.Department of Health and Human Services,Office for Human ResearchProtections,SACHRP Committee Recommendations.16.U.S.Food and Drug Adminstration,Medical Device,Digital Health Center of Excellence.17.U.S.Food and Drug Administration,Ar

187、tificial Intelligence and MachineLearning in Drug Development,May 2023.18.U.S.Food and Drug Administration,Digital Health Technologies for DrugDevelopment,December 2023.19.Cristos Andreas Makridis et al,Informing the ethical review of humanresearch subjects utilizing artificial intelligence,Frontier

188、s,September 2023.20.Duxin Sun et al,Why 90%of drug development fails and how to improve it?,Science Direct,February 2022.References35 2024 WCG.All Rights Reserved.WCG is a global leader of solutions that measurably improve and accelerate clinical research.Biopharmaceutical and medical device compani

189、es,contract research organizations(CROs),research institutions,and sites partner with us for our unmatched expertise,data intelligence,and purpose-built technology to make informed decisions and optimize study outcomes,while maintaining the highest standards of human participant protection.WCG raises the bar by pioneering new concepts,reimagining processes,fostering compliance and safety,and empowering those who perform clinical trials to accelerate the delivery of medical therapies and devices that improve lives.For more information,please visit or follow us on X WCGClinical or LinkedI