Johns Hopkins Institute for Clinical and Translational Research A CTSA Program

1. Johns Hopkins Institute forClinical and Translational Research A CTSA Program Daniel E. Ford, M.D., M.P.H. Director, Institute for Clinical and Translational Research Vice Dean for Clinical Investigation

2. Goals for the Presentation • Introduce national and local CTSA program • Describe plans for IT support of clinical research • Give us advice on how to transform clinical and translational research at Johns Hopkins

3. Impetus for the CTSA Program • Implementing biomedical discoveries made in the last 10 years demands an evolution of clinical science. • New prevention strategies and treatments must be developed, tested, and brought into medical practice more rapidly. • CTSA awards will lower barriers between disciplines, and encourage creative, innovative approaches to solve complex medical problems. • These clinical and translational science awards will catalyze change -- breaking silos, breaking barriers, and breaking conventions.

4. Urgency to Change at Johns Hopkins • Faculty want more training and resources in translational research • Regulatory environment more complex • Not enough trained specialists in translational research (staff and faculty) • Basic scientists cannot easily find clinical research collaborators • Competition from both community hospitals/physician and international sites • Need to preserve academic reputation of Johns Hopkins • Accrual of research participants too slow • Lack of centralized informatics support • No institutional biospecimen bank • Not certain what our role should be in investigator-initiated research as compared to big science (shared data/multicenter) • NIH support is flat and need to show leveraging of resources

5. Building a National CTSA Consortium WA ME MT ND VT MN OR NH ID WI NY MA SD RI WY MI CT NJ IA PA NE NV OH DE IN IL UT MD CO WV VA KS MO CA KY TN AZ OK AR SC NM GA AL MS TX LA AK FL HI Participating Institutions Since 2006 Since 2007

6. Building a National CTSA Consortium FY06 Grantees Center for Clinical and Translational ScienceThe Rockefeller University Center for Clinical and Translational SciencesUniversity of Texas Health Sciences Center at Houston Clinical and Translational Science CenterUniversity of California, Davis Clinical and Translational Science InstituteUniversity of Pittsburgh Clinical and Translational Science Institute University of California, San Francisco Clinical and Translational Sciences InstituteUniversity of Rochester School of Medicine and Dentistry Duke Clinical and Translational Science InstituteDuke University Institute for Translational Medicine and TherapeuticsUniversity of Pennsylvania Irving Institute for Clinical and Translational Research Columbia University Mayo Center for Translational Science ActivitiesMayo Clinic Oregon Clinical and Translational Research InstituteOregon Health and Science University (partnering with Kaiser Permanente) Yale Center for Clinical InvestigationYale University FY07 Grantees Atlanta Clinical and Translational Science Institute Emory University (partnering with Morehouse College) CTSA at Case Western UniversityCase Western University CTSA at Washington University Washington University CTSA at Weill Cornell Medical College Weill Cornell Medical College (partnering with Hunter College) Institute for Clinical and Translational ResearchUniversity Of Wisconsin Madison Institute for Clinical and Translational Research Johns Hopkins Institute of Translational Health Sciences University Of Washington Michigan Institute of Clinical and Health Research University Of Michigan At Ann Arbor North & Central Texas Clinical and Translational Science Initiative University of Texas Southwestern Medical Center - Dallas University Of Chicago CTSAUniversity Of Chicago Univ of Iowa's Inst for Clinical and Translational Science University Of Iowa Vanderbilt Institute for Clinical and Translational Research Vanderbilt University (partnering with Meharry Medical College)

7. CTSAWeb.org

8. Consortium Governance & Organization Governance Manual available at http://ctsaweb.org/Docs/CTSA_Governance_Manual.pdf

9. Translational Pathway 2 1 Diffusion to All Health Care Settings Mechanistic Studies Initial Human Testing Basic Discovery Proof of Efficacy Proof of Effectiveness

10. Institute for Clinical and Translational ResearchInfluence on Scientific Discovery Research Education and Training Enhancing Formulation of Creation of Development Conduct Analysis of Publication Observation Scientific Question Research Team Study Protocol of Study Study Data Impact Study Advisory Boards Protocol Review Translational Science Forums Science Cores Innovation Groups Biostatistical Support Regulatory Support ATIP Research Ethics Clinical Research Units Clinical Research Participant Core Data/Safety Monitoring Community Engagement Program Biomedical Informatics Navigator Program Institute Programs

11. Johns Hopkins Programs Before CTSA • K12 Clinical Research Scholars Program • Graduate Training in Clinical Investigation • T32 Medical Student Training Program • Johns Hopkins GCRC • Inpatient • Outpatient • Pediatric • Kennedy-Krieger Neurobehavioral Research Unit • Bayview GCRC

12. Johns Hopkins Institute for Clinical and Translational Research NIH & other government agencies Research participant Recruitment office Trial Design Advanced Degree-Granting Programs Biomedical Informatics Johns Hopkins ICTR Industry Clinical Resources Participant & Community Involvement Biostatistics Regulatory Support Healthcare organizations

13. Institute for Clinical and Translational Research • Goals • Academic Home for Clinical and Translational Researchers • Coordinate Translational Research Activities • Support Training of Clinical and Translational Research Faculty and Staff • Provide centralized support for research where efficient • Measure and track efficiency of research to address barriers • Director – Daniel Ford, MD, MPH Vice Dean for Clinical Investigation • Location – Small Office at Thames Street

14. Deputy Directors of the Institute for Clinical and Translational Research • Dr. Charles Balch • Dr. Stephen Desiderio • Dr. Elizabeth Jaffee • Dr. Pamela Ouyang • Dr. Neil Powe • Dr. Christopher Saudek • Dr. Pamela Zeitlin

15. Johns Hopkins ICTRParticipating Schools • School of Engineering • School of Medicine • School of Nursing • School of Public Health

16. Research Education, Training, and Career Development Leaders: Neil Powe, Franklin Adkinson, Susan Furth, Edgar Miller, Jon Samet, Scott Zeger, Jennifer Haythornwaite, Jeri Allen and Charles Flexner • Coordinates clinical and translational research training throughout Johns Hopkins • Administers formal degree granting programs in clinical research (Graduate Program in Clinical Investigation, Clinical Epidemiology Program) • Administers predoctoral (TL), postdoctoral (KL) and junior faculty (KL) training awards in clinical and translational research • Coordinates summer and elective experiences in clinical and translational research for medical students • Curriculum design, innovation and development of problem/case and/or team based modules in clinical research • Coordinates clinical research curriculum for medical students across the four years of medical school • Provides technical assistance on study design, study implementation, data collection, data management and data analysis to clinical and translational research trainees in the formal program

17. Basic Science Translational Forum Leaders: Stephen Desiderio and Elizabeth Jaffee • Identify basic science researchers who are considering translation to human biology and/or clinical settings • Identify clinical investigators who wish to collaborate with basic scientists to develop or improve tools for clinical research • Match each of these groups with appropriate clinical or basic research faculty • Nurture groups through facilitated access to experts and institutional resources • Conduct formal follow-up to evaluate effectiveness

18. Clinical Science Translational Forum Leaders: Daniel Ford and Peter Pronovost • Identify clinical researchers who are considering translation to broader community – knowledge transfer • Match these groups with appropriate basic research faculty, community populations, providers and payers • Nurture groups through facilitated access to content experts and institutional resources • Conduct formal follow-up to evaluate effectiveness

19. Clinical Research Units Leaders: Pamela Ouyang (Bayview), Christopher Saudek (JHH Broadway), Pamela Zeitlin (Pediatrics), Michael Cataldo (Neurobehavioral KKI) • Continue to provide the “laboratory” in which over 260 active clinical research protocols currently being accomplished • Inpatient and Outpatient facilities on the Broadway and Bayview campuses (both adult and pediatric); sleep units (Pediatric at Broadway, Adult at the Bayview campus) • Emphasis on investigator-initiated protocols • Provide thorough peer review prior to study initiation • Support investigators with trained research nurses, phlebotomists, and interviewers • Support investigators with computing (including system manager), full nutrition staff and consultations. • Support investigators by funding limited laboratory and imaging costs as Ancillaries

20. Clinical Research Units • Provide specialized services such as exercise (Bayview/GSS), body composition, CV imaging (Bayview), and a Core Laboratory for research immunoassays, mucociliary clearance laboratory, flexible bronchoscopy, BAL and infant lung function laboratory (Pediatrics) • Provide specimen processing • Neurobehavioral CRU (Kennedy Krieger) with functional MRI facilities is also available to researchers • Work with other ICTR units to coordinate areas such as biostatistical consultation and training.

21. Translational Research Navigators Leader: Daniel Ford • Experienced research coordinators will be assigned to research teams to help develop a plan for efficiently moving protocols through regulatory offices • Each research coordinator will have a person assigned to work with them in each of the regulatory-based offices at Johns Hopkins • Will assist research teams in completing appropriate applications and taking advantage of all science opportunities

22. Biostatistical Support Leader: Scott Zeger • Johns Hopkins Biostatistics for Clinical and Translational Research (Biostats CenTeR) • Center coordinates biostatistics support to Johns Hopkins translational research teams • Provides practical advice on study design and biostatistics questions • Has library of previous biostatistics consultations for review before consultation • Encourages consultation at study design phase and before data collection has been completed

23. Advanced Translation Incubator Program - ATIP Leader: Jeffrey Rothstein • Research teams can apply for pilot grant funds to support translational projects • Investigators need to describe a “deliverable” product at the end of funding • Want to pilot a new focus on strict timelines with rapid turnaround • Can apply for up to $100,000 over two years • New investigators, interdisciplinary teams, and research teams with trainees are priorities

24. Clinical Research Management System - CRMS Leaders: Diana Gumas, Daniel Ford, Stuart Ray, Kerry Stewart • Application to register and follow research participants • Linked to eIRB and EPR • Document eligibility and produce CRF • Web-based secure system

25. Clinical Research Management System EPR/Labs Integration External Recruiting Website IRB Integration Protocol Schema / Patient Calendar Budgeting Sponsor Billing Patient/ Protocol Registry Eligibility Screening Billing Compliance Library Research Forms Data Warehouse JHED/Site Minder Integration Investigators Research Nurse Program Manager Data Manager Insurance Clearance Budgeting by Dept. Admin. SoM Leadership Pharmacy Core Facilities Labs Billing Compliance IRB Possible Future Functionality Currently In Use Work In Progress Key:

26. Biomedical Informatics Leaders: Harold Lehmann, Giovanni Parmigiani • Have programs responsible for knowledge management, clinical research informatics, biospecimen informatics, and bioinformatics • Completing needs assessments in each area • Prioritizing development of new biomedical informatics applications

27. Research Participant Retention and Recruitment Office Leader: Cheryl Dennison • Develop tools to monitor the success of various recruitment and retention approaches used within Johns Hopkins • Provide consultations on various IRB-approved recruitment strategies • Develop Johns Hopkins strategic initiatives focused on recruitment and retention

28. Johns Hopkins Data and SafetyMonitoring Board Leaders: Fred Luthardt and Daniel Ford • Help investigators who do not have access to a DSMB develop a data and safety monitoring plan • If a DSMB is needed provide the structure and administrative support for an institutional DSMB • Communicate DSMB decisions to appropriate IRBs

29. Research Ethics Achievement Program Leader: Jeremy Sugarman • Provides ethics consultations for research groups concerning design and conduct of studies • Conducts empirical studies to inform policies related to risks and benefits associated with different study designs and consent procedures

30. Research Subject Advocate Leaders: Liz Martinez and Daniel Ford • Consultation to research teams about best ways to work with research subjects • Work with research teams who need an intermediary for working with dissatisfied research participants

31. Community Engagement Office Leaders: Charles Balch, Kostas Lyketsos, Chad Boult and Peter Pronovost • Community Research Network Office: Focus on expanding sites and providers performing research, e.g. Anne Arundel Medical Center; community physicians • Community/Patient Research Partnership Office: Focus on working with patient disease advocacy groups to set research agenda and enlist help with translational research projects • Secondary Translation Office: Focus on consultation service for research teams as they design efficacy/effectiveness studies. Consumers of research include patients, providers, payers, and hospitals

32. Innovative Methodology Workgroups Leader: Charles Flexner • Identifies important and recurrent clinical research methodologic and biostatistics problems • Directs multidisciplinary work groups to address these issues • Creates new methodologic approaches that are available to the broad Johns Hopkins community

33. Drug/Device/Vaccine Development Leaders: Elizabeth Jaffee and Craig Hendrix • Provides consultation to research teams on using institutional resources to promote drug/device/vaccine development • Provides medicinal chemistry expertise for pre-clinical toxicology • Provides Good Manufacturing Practices (GMP) and near-GMP for synthesis of biological reagents for pre-clinical toxicology and early Phase 1 testing • Provide pharmacologic and biologic endpoint expertise • Individual and group seminars and hands on experience with core technologies

34. Proteomics/Biomarker Core Leader: Jennifer Van Eyk • Integrates discovery and validation strategies and technologies for the development of robust biomarkers • Assists in study design, cohort development, appropriate experimentation and data analysis as part of discovery process • Develops and coordinates new technologies and optimizes/standardizes protocols specifically for biomarker development • Individual and group seminars and hands on experience with core technologies

35. Genetics Translational Technology Core Leader: Gary Cutting • Provide consultation to clinical and translational research teams regarding the feasibility, design, power and costs of projects using molecular technologies • Provide clinical grade services such as DNA banking, sequencing and genotyping for qualified investigators • Can help evaluate test performance in a CLIA-certified environment • Individual and group seminars and hands on experience with core technologies

36. Imaging Core Leader: Katarzyna Macura • Provides consultation for best imaging approach in translational studies • Developing an imaging library in cooperation with Kennedy-Krieger Institute

37. Johns Hopkins ICTR Challenges • Enhancing communication across the multiple programs in the ICTR • Finding best ways to leverage research infrastructure • Relationships with various schools • Relationships with cancer center • Having the discipline to evaluate programs and reallocate resources • Creating an academic home for translational researchers without a physical location • Measuring output or translational research successes • Identifying most productive approaches to working with other academic institutions within CTSA

38. Communicating with the ICTR • ICTR Main Line -- 443-287-ICTR (4287) • ICTR Website – ictr.johnshopkins.edu • News of program roll-outs, and upcoming events. • Additional Information about the ICTR. • Links to the national CTSA program • COMING SOON: A catalog of ICTR services, and a catalog of resources at the JHMI that support clinical and translational research. • COMING SOON: For research subjects – a searchable catalog of current clinical research taking place in the School of Medicine.