Clinical and Translational Research: Expectations and Goals

1. Clinical and Translational Research:Expectations and Goals Daniel E. Ford, M.D., M.P.H. Director, Institute for Clinical and Translational Research Vice Dean for Clinical Investigation

2. Clinical Research at Johns Hopkins • Comprehensive in Scope • Cancer Center • Research in children • Neurosciences • Drug abuse • Investigator Initiated • Large Discovery Pipeline • Expertise in Moving Discoveries to Population • Fortunate to Have Large Number of Trainees Interested in Research

3. Research at ClOffJohns Hopkins Medicine

5. Sponsored Project SourcesJohns Hopkins SOM – Prorated Awards

6. Translational Medicine • T1 – “the transfer of new understandings of disease mechanisms gained in the laboratory into the development of new methods for diagnosis, therapy, and prevention and their first testing in humans.” • T2 – “the translation of results from clinical studies into everyday clinical practice and health decision making.” • IOM Clinical Research Roundtable

7. Translational Research Activities T1 activities included: 1) the translation of basic discovery into mechanistic studies in cell lines or animals, 2) the translation of mechanistic studies into initial human testing, and 3) the translation of initial human testing into proof of efficacy. The T2 activities included 1) the translation of proof of efficacy into proof of effectiveness in a usual care setting, and 2) research aimed at enhancing the adoption of best practices in the community

8. Conclusions 69% responded they are translational researchers (9% other survey but different definition) PhD and fellows report less translational activity Barriers to translational research are as expected but substantial

9. It is premature for me to consider translational research. I have methodological obstacles to making my research more translational. I don’t have the training or expertise I need to make my research more translational. I don’t have the guidance or mentorship I need to make my research more translational. I haven’t identified or secured the colleagues or collaborators I need to make my work more translational. I am not sure what the next translational step is I have not yet identified an appropriate translational research question to pursue. I don’t have the resources (funding, space, equipment, technology) for making my research more translational. Translational research is not part of my research agenda or is not central to the goals of my research program. 5 It is premature for me to consider translational research. I have methodological obstacles to making my research 11 more translational. I don’t have the training or expertise I need to make my work 20 more translational. I don’t have the guidance or mentorship I need to make my research more translational. I haven’t identified or secured the colleagues or 30 collaborators I need to make my work more translational. I am not sure what the next translational step would be for 33 my research. I have not yet identified an appropriate translational 43 research question to pursue. I don’t have the resources (funding, space, equipment, 44 technology) for making my research more translational. Translational research is not part of my research agenda or 84 is not central to the goals of my research program. 0 10 20 30 40 50 60 70 80 90 Number of Respondents (n=205) Barriers to Translational Research (n=205)

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

11. 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

12. 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.

13. Johns Hopkins and Translational Medicine • Need to support researchers at all career levels • Progress in translational medicine requires development of teams of researchers that appreciate all phases • Clinical and translational researchers will continue to develop more technical and specialized approaches. Handoffs require active coordination (project coordinators). • Do to increasing regulations, researchers will need to rely more on systematized solutions at institutional or national level • Academic organizations need to partner better with other organizations to increase efficiency (industry, payers, patient groups) • Need to balance resources/support for early and late translation • Academic centers need better management structure to systematically monitor research process and barriers

14. 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

15. CTSAWeb.org

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

17. Institute for Clinical and Translational Research • Goals • Academic Home for Clinical and Translational Researchers • Expand the Working Relationships of Clinical Investigators with Basic Scientists and Population-oriented Scientists • Coordinate Translational Research Activities to Increase Innovation and Speed of Translation

18. Institute for Clinical and Translational Research • Goals • Support Training of Clinical and Translational Research Faculty and Staff • Provide Centralized Support for Research Where Efficient • Measure and Track Efficiency of Human Subjects Research to Address Barriers • Director – Daniel Ford, MD, MPH Vice Dean for Clinical Investigation

19. Deputy Directors of the Institute for Clinical and Translational Research • Dr. Charles Balch • Dr. Stephen Desiderio • Dr. Charles Flexner • Dr. Elizabeth Jaffee • Dr. Pete Miller • Dr. Pamela Ouyang • Dr. Jeff Rothstein • Dr. Pamela Zeitlin

20. 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

21. Johns Hopkins ICTR Progams • Translational research training • K junior faculty/fellows • Medical students • Research staff and community physicians • Clinical Research Units • 1200 inpatient research bed days • Over 10,000 outpatient research visits • Pediatric and adult beds including Bayview

22. Johns Hopkins ICTR Progams • Biostatistical Consultation • Innovative Methodology Workgroups • Clinical Research Ethics Consultations • Data and Safety Monitoring Services • Translational Research Navigators (project managers) • Clinical Research Management Database

23. Johns Hopkins ICTR Progams • Basic Discovery Translational Forum • Clinical Sciences Translational Forum • Accelerated Translational Incubator Program (ATIP) support pilots • Research Participant Recruitment and Retention Program • Community Research Networks Office • Secondary Translation or Knowledge Transfer Office

24. 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:

25. Translational Cores • Genetics Core • Proteomics/Biomarker Core • Drug and Vaccine Development Core