1. UT Southwestern Center for Translational Medicine Summary Statement Discussion November 14, 2013 Robert D. Toto, M.D. Associate Dean for Clinical and Translational Research 0

2. Center for Translational Medicine Mission Statement Enabling investigators to discover, translate, and disseminate new knowledge that will improve the prevention, detection, diagnosis, and effective treatment of disease by aligning and integrating our basic and clinical research communities to promote and facilitate Clinical and Translational Research at UT Southwestern.

3. Vision Integrate and centralize research resources to accelerate translation of new knowledge across the continuum of translational research (T1-T4). Create an academic practice in which each patient is considered a potential research participant, capitalizing on existing clinical IT infrastructure to establish a “real time” repository of potential research participants. Improve quality, safety, efficiency and speed of clinical and translational research, particularly for NIH supported research.

4. Strategy Better integrate academic research resources including the CTSA with hospital/clinic EHRs and improve existing informatics infrastructures to assist in high quality, safe, cost-effective translational research, education and patient care Encourage trans-disciplinary collaborations to accelerate translation of discovery into practice  Integrate clinical research into practice  Develop new technology and methodology  Fund transdisciplinary pilot studies  Improve training and mentoring programs Goal: Form a coordinated structure that leverages institutional strengths to generate an outstanding program of translational research

5. Center for Translational Medicine: Organization

6. Center for Translational: “Strategy and Vision” Aims 1.Enhance our Research Environment to Accelerate Translation of Discovery into Practice Across the Spectrum (T0-T4) of Translational Science.  Create new mechanisms to improve efficiency, quality, and affordability of CTR.  Provide new translational technologies to enhance CTR.  Promote a research culture among faculty, trainees, and healthcare personnel.  Develop an expanded bank of potential research subjects 2.Leverage Existing and New Resources to Build on Four Innovative Programs in Translation.  Target Identification and Validation  Discovery in Humans  Intervention in Humans  Population Science and Community Engagement 3. Share Knowledge and Discovery and Contribute to the Leadership of the National CTSA Consortium. 5

7. Research Environment Office of Clinical Research Facilitation Recruitment of Human Subjects  Promote research opportunities to patients  Affirmative “Opt-in” Informed consent  Innovation in EHR and Social Media communication  Central repository for study participants Basic training in principles of clinical research to health care providers, nursing and allied health professionals, and clerical staff. Necessary operational support through the Hospitals Clinical Research Coordination Committee (CRCC). Integration of research staff into our hospitals and clinics.

8. Programs in Translation 1.Target Identification and Validation: Organize, enhance, and increase core facilities to provide: state-of-the-art technologies to the research community assistance in the statistical analyses of large datasets development and validation of assays to measure biomarkers and clinically relevant metabolites in populations. 2.Discovery in Humans: Incorporate highly phenotyped human cohorts into the Center to facilitate discovery science Create a centralized infrastructure for human specimen procurement, processing, storage, and data management. create a centralized infrastructure for human specimen procurement. State-of-the-art imaging methods and physiological assays Re-engineer the GCRC and integrate into the University Hospital to better serve investigators and study participants 7

9. Programs in Translation 3.Intervention in Humans. Build capacity for early phase as well as later phase multisite clinical trials through strategic relationships with our partners and the Center’s OCRF. Use imaging, genomic, biomarker, and informatics technologies to identify study populations for targeted interventions. Leverage existing resources to form and support a Phase 1 clinical testing facility 4.Population Science and Community Engagement. Promote observational and interventional patient-centered outcomes and comparative effectiveness research Accelerate methods for EHR-enabled interventions to improve outcomes Expand highly successful community-based research programs initiated during the prior funding period.

10. Education New and Flexible pre- and post-doctoral training programs in clinical and translational research Enhance and Expand the Curriculum for the Master of Science in Clinical Science Degree. Increase Multidisciplinary Team Science Training and Interactions. Establish an Integrated “Mentored to Independent Investigator Transition” (K2R) Program. Expand Opportunities for Clinical and Translational Science Training for Predoctoral PhD and Medical Students

11. Summary Statement: Clip from the Overall Review Proposed UT Southwestern CTM is exceptionally strong and is likely to have a sustained powerful impact on clinical and translational sciences in the participating institutions and in the region. Significance is very high. The overall vision and strategy are ambitious, forward looking and far-reaching. Clear commitment to identify potential problems, hurdles and inefficiencies and definitive proposals to address them. Organizational structure, including the organization of the advisory committees, is very strong and thoughtful. Overall environment is superb, with new buildings and considerable space available for the CTSA and many of its components. New hires in translational research, along with Dr. Toto, who is vested with the appropriate authority, document the commitment of the University to the multiple initiatives described. Plans to engage and integrate investigators in some of the disciplines mentioned in the application, such as nursing, nutritional science, behavioral science and dentistry, are not adequately developed or articulated. T3 and T4 research in the CTM is minimally addressed, and investigators in these research domains are not clearly defined. However, these minor issues do not diminish the likely overall high impact of the proposed CTM. 10

12. Center for Translational Medicine Reviewers felt creation of a new Center as the integrated home of the CTSA would likely have a sustained impact on and improve the environment for CTR.

13. Integrated Home Leadership Reviewers were highly complimentary of the following:  Our organizational structure that places final decision- making regarding resource utilization, direction, and priority areas in the hands of the Steering Committee, which consists of key leaders of the CTM.  Our proposed “Evaluative Logic” approach to workflow.  Enhancements to the Pilot Program.  Our ClinDEN clinical data exchange network.  Our plan to integrate a research environment into our clinical facilities.  Our community health program.

14. Clinical and Translational Resources and Services We selected 20 specific resources to highlight in the application to provide evidence of rapid access to a broad group of resources and services that support the entire spectrum of CTR. Reviewers felt the resources were extremely strong and well-conceived and that grouping the resources around the four institutional strengths allows the specific resource elements to be organized in a manner that will be logical and accessible to CTR researchers.

15. Research Education, Training, and Career Development Resources noted as particular strengths  K2R program, which includes small group grant writing workshops, individual grant writing tutorials, mock study sections  Translational Medicine Grant support office  Mentors Connect program to address our acknowledged low success rate of past KL2 program graduates in competing for R01s.