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 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 Research at Johns Hopkins Medicine New Protocols 2007 2008 2009 2010 2011 New Exempt 443 364 311 353 218 New Expedited 561 688 830 913 923 New Convened 418 520 569 603 682 Total New 1422 1572 1710 1869 1972 Total Protocols 2007 2008 2009 2010 2011 Active Protocols 3040 3412 3707 3962 4509 Active PIs 944 1011 1081 1215 1214 Randomized Clinical Trials 2007 2008 2009 2010 2011 Active Randomized Clinical 724 815 854 1090 1283 Trials New Randomized Clinical Trials 247 376 405 422 468 Commercial Protocols 2007 2008 2009 2010 2011 Active Commercial 550 543 487 620 699 New Commercial 262 322 270 267 307 1
Research at Johns Hopkins Medicine (cont d) Commercial Clinical Trials 2007 2008 2009 2010 2011 Active Commercial Clinical Trials 385 385 360 431 516 New Commercial Clinical Trials 181 202 243 192 227 Protocols with Drugs/Devices 2007 2008 2009 2010 2011 Active Protocols Using Drugs 869 1024 1091 1111 1249 New Protocols Using Drugs 282 346 386 401 418 Active Protocols using Devices 288 327 245 380 486 New Protocols using Devices 104 118 102 165 159 Protocols with IND/IDEs 2007 2008 2009 2010 2011 Active Protocols using INDs 383 496 523 649 701 New Protocols using INDs 157 198 156 243 292 Active Faculty Held INDs 74 118 162 156 148 New Faculty Held INDs 13 55 82 38 39 Active Protocols using IDEs 12 29 44 43 46 New Protocols using IDEs 9 21 20 11 17 Active Faculty Held IDEs 7 13 14 13 14 New Faculty Held IDEs 3 3 2 2 2 Sponsored Human Subjects Research for Johns Hopkins University 2007-10 Translational Pathway Basic Discovery Mechanistic Studies 1 Initial Human Testing Proof of Efficacy 2 Proof of Effectiveness Diffusion to All Health Care Settings 2
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 Translational Research Activities T1 activities: 1) the translation of basic discovery into mechanistic studies in cell lines or animals; 2) the translation of mechanistic studies into initial human testing; 3) the translation of initial human testing into proof of efficacy. Translational Research Activities The T2 activities include: 1) the translation of proof of efficacy into proof of effectiveness in a usual care setting; 2) research aimed at enhancing the adoption of best practices in the community 3
Johns Hopkins Researchers and Translation 69% responded they are translational researchers PhD and fellows report less translational activity Barriers to translational research are as expected but substantial Barriers to Translational Research (n=205) It is premature It is for me to to consider consider translational translational research. research. I have methodological I have methodological obstacles to making my research my research more translational. more translational. I don t have the training or expertise I need to make I don t have the training or expertise I need to make my work my research more translational. more translational. I don t have the guidance or mentorship I need to make my I research don t have the more guidance translational. or mentorship I need to make my research more translational. I haven t identified or secured the colleagues or I haven t identified or secured the colleagues or collaborators I need to make my work more collaborators I need to make my work more translational. translational. I am not I sure am not what sure what the the next translational step would step be for is my research. I have not yet identified an appropriate translational I have not yet identified an appropriate translational research question to pursue. research question to pursue. 5 11 20 30 33 43 I don t have I don t the have resources the resources (funding, space, equipment, 44 equipment, technology) for making for making my research my more research translational. more translational. Translational research is not part of my research agenda or Translational research is not part of my research 84 is not central to the goals of my research program. agenda or 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) NIH Mission Statement NIH s mission is to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce the burdens of illness and disability. 4
Institute for Clinical and Translational Research Influence on Scientific Discovery Research Education and Training Observation and Formulation of Scientific Question Creation of Research Team Development Study Protocol Conduct of Study Analysis of Study Data Publication and Enhancing Impact of Study Protocol Review Translational Cores Innovation Methods Biostatistician Support Regulatory Support Institute Programs ATIP Research Ethics Clinical Research Units Office of Recruitment and Retention Data/Safety Monitoring Community Engagement Program Biomedical Informatics Navigator Program 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. ICTR: A Common Vision Led by the National Center for Research Resources, the CTSA program supports a national consortium of medical research institutions that work together and share a common vision to: Improve the way biomedical research is conducted across the country Reduce the time it takes for laboratory discoveries to become treatments for patients Engage communities in clinical research efforts Train a new generation of clinical and translational researchers www.ncrr.nih.gov/ctsa 5
Guiding Principles of the CTSA Program Team Science Connectivity Partnerships CTSA Philosophy Breaking Down Institutional Barriers Collaboration Consortium Website www.ctsaweb.org 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 6
Johns Hopkins and Translational Medicine 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 regulatory complexity, 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 Clinical and Translational Science Awards (CTSA) Deputy Directors of the Institute for Clinical and Translational Research Dr. Karen Bandeen-Roche Dr. Cheryl Dennison Himmelfarb Dr. Stephen Desiderio Dr. Charles Flexner Dr. Elizabeth Jaffee Dr. Edgar (Pete) Miller Dr. Pamela Ouyang Dr. Jeff Rothstein Dr. Darius Tandon Dr. Pamela Zeitlin 7
What do we expect of the new translational researchers? How to work in translational teams Sharing data and reuse of collected data Pushing the field in use of informatics at all levels: Engaging with patients Improving efficiency of research Ontology and organization of data Use of clinical data for research Computational biology Greater awareness of paths to ultimate translation including commercialization Advancing discovery and translation in the international context 8