1. National Center for Research Resources NATIONAL INSTITUTES OF HEALTH T ranslating research from basic discovery to improved patient care The United States National Institutes of Health Clinical and Translational Science Award Program: An Overview JCSMR First International Conference on Translational Medicine November 1, 2010 Anthony Hayward, MD PhD Director, Division for Clinical Research, National Center for Research Resources, NIH, USA

2. NCRR Accelerating Research from Basic Discovery to Improved Patient Care is a shared goal Improved patient care Discovery and Mechanism Technologies, Tools, and Resources Clinical Studies Community Engagement

3. NCRR CTSAs – Creating a Home for Clinical and Translational Science

4. NCRR Pre-existing NIH awards that were combined:  General Clinical Research Centers  Mentored Career Development awards  Curriculum Development awards  Predoctoral Training awards Postdoctoral training awards Successful applicants received about 40% additional funding to support larger scope

5. NCRR Building a National CTSA Consortium 55 CTSA sites in 28 states and the District of Columbia = CTSA States = 2006–2009 CTSA Sites = 2010 CTSA Sites MT WY ID WA OR NV UT CA AZ ND SD NE CO NM TX OK KS AR LA MO IA MN WI IL IN KY TN MS AL GA FL SC VA WV MI NY PA MD DE NJ CT RI MA ME VT NH AK HI NC OH PR

6. NCRR NIH funding to successful CTSA applicants

7. NCRR CTSA – Assembling Local Participants Yerkes National Primate Research Center Kaiser Permanente Georgia Atlanta VA Medical Center Complex Carbohydrate Research Center at U Georgia Georgia Bio Georgia Research Alliance Emory University Morehouse School of Medicine CDC Georgia Tech Children’s Healthcare of Atlanta Atlanta Clinical and Translational Science Institute (Atlanta-CTSI) at Emory University

8. NCRR An Extravert CTSA with Focus on Impact

9. NCRR CTSA – Creating Regional Partnership Opportunities = CTSA Institutions = East Coast Consortium = Midwest Consortium = West Coast Consortium Weill Cornell Medical College Columbia University Rockefeller University Albert Einstein College of Medicine University of Rochester Yale University University of Wisconsin Mayo Clinic College of Medicine University of Washington Oregon Health & Science University University of California, Davis University of California, San Francisco Stanford University The Scripps Research Institute Washington University in St. Louis University of Iowa University of Chicago

10. NCRR Assembling a Consortium: Developmental Milestones  2006First 12 agree to work together and ask NIH Clinical Center to join them  20069 Key Function Committees established  2008First 24 start on Strategic Plan  2009Strategic Goal Committees in place. Number of Key Function Committees expands to 14  2010Increased attention to administration, Consortium Management Committee  2011March meeting to review products & goals

11. NCRR Top Down or Bottom up?  CTSA Consortium Steering 80  Child Health Oversight158  Biostatistics, Epidemiology, RD141  Ethics226  Clinical Research Management216  Communications110  Community Engagement105  Comparative Effectiveness 105  Education, Career Development176  Evaluation132  Informatics278  Public-Private Partnerships101  Regulatory187  Translational123

12. NCRR The CTSA consortium will enhance the efficiency and quality of clinical and translational research to improve the health of the nation Each Strategic Goal Committee comprises 3 PIs and 3 administrators Strategic Goals for the CTSA Program  Build national clinical and translational research capacity  Train and foster career development of clinical and translational scientists  Enhance consortium-wide collaboration efforts  Bring new communities into research programs  Encourage & promote bench to bedside (T1) translational research

13. NCRR Build national clinical and translational research capability Reducing protocol start-up times Measure and compare review times at all CTSAs Measure and compare contracting times Provide speedy and coordinated response to national needs (e.g., vaccine trials, H1N1 epidemic) Reducing enrollment times Data from individual studies and clinicaltrials.gov Bring new communities into research 13

14. NCRR CTSA Training & Career Development Pathways Curriculum development component funded through CTSAs:  Pre-doctoral Trainee component Supports 355 trainees Year-out research experience for medical students 5 year support for pre-doctoral students 3 year support for post-doctoral students  Post-doctoral component Supports 403 scholars Mostly “professional” doctorates, MD, DDS, DNP Mentored career development for post-doctoral students with option for MS, MPH and PhD higher degrees

15. NCRR Educational Impact of CTSA Program (Based on 2010 Annual Progress Reports from 46 CTSAs)

16. NCRR Training Clinical and Translational Scientists Core Competencies in Clinical and Translational Research  Identify major clinical/public health problems and relevant research questions  Critique the literature regarding the status of a health problem  Design a study protocol for clinical and translational research  Study methods, design and implementation  Laboratory, clinical and population research methods  Statistical methods and analysis  Bioinformatics Additional information available at http://www.ctsaweb.org/corecompetencies http://www.ctsaweb.org/corecompetencies

17. NCRR CTSpedia  Wiki structure  Identifies and shares resources across the national consortium and community researchers world- wide ResearchMatch  National recruitment Registry  Centralized, disease-neutral Web portal to connect researchers to research volunteers  Includes more than 40 CTSAs REDCap  Web-based tools  Supports data capture and dissemination for clinical and translational research www.CTSpedia.org www.ResearchMatch.org www.project-redcap.org Web Resources for Research Tools to Enhance Collaborations

18. NCRR ResearchMatch.org – Connecting Volunteers with Researchers  National Web portal for research volunteers Registration for volunteers launched November 2009 Researcher matching opened March 2010  More than 50 CTSA affiliated institutions participating  Over 100 studies registered  68 researchers have connected with 3,975 volunteers by email  Of those contacted 20% responded yes they wish to be contacted by the researcher

19. NCRR Activities in Community Engagement  Engage Communities and Enhance Trust CTSA community advisory boards link Academic Health Center and communities Community engagement cores build capacity  Facilitate Recruitment and Retention Efforts  Share and Disseminate Best Practices Consultant services Publish “Principles of Community Engagement” booklet Define Community engagement core competencies

20. NCRR Comparative Effectiveness Research  Develop capacity and methods for translation of research results into practice across the health care system  Conduct a capacity and needs assessment of comparative effectiveness research and related resources throughout the CTSAs to help articulate how the consortium can act as a portal to facilitate such research  Expand the workforce of clinical researchers competent to design, implement and analyze comparative effectiveness research.

21. NCRR  Promote HPV immunization of junior high school girls in Chicago  CTSA engaged underserved Chicago teenagers and parents  Community Connections (CTSA Community Advisory Research Board and church members) assisted in: Understanding community perspectives Determining awareness of potential risks and challenges in community perceptions of HPV Building community support  CTSA faculty bridged between investigators, community leaders, and residents and the discussion of ethical issues Promoting Adolescent Health in Chicago Schools

22. NCRR Promoting translational research All CTSAs have a pilot project program Launched Pharmaceutical Assets Portal and IP Portal Partner with other trans-NIH activities: Molecular Libraries Probe Production Center Network Therapeutics for Rare and Neglected Diseases (TRND) Screening by National Center for Translational Technologies Next Translational Research meeting in March 2011

23. NCRR A Pilot Project on Panic Anxiety Project Development Team Pilot Project Grant – translational acceleration program Pilot project grant – clinical translational research Research findings suggest new medication target Helping to Improve treatments for panic attacks  Panic anxiety model in rats finds increased orexin gene expression in neurons. An RNAi protects rats from symptoms  2 nd pilot project grant funds clinical imaging in panic patients  Collaboration with U of Lund, Sweden finds higher levels of orexin in CSF of patients with panic anxiety than controls  Orexin antagonists may offer potential new treatment strategy  Grant support from NIMH and NCRR CTSA. Publication: Johnson PL, Truitt W, Fitz SD, Minick PE, Dietrich A, Sanghani S, Träskman- Bendz L, Goddard AW, Brundin L, Shekhar A. A key role for orexin in panic anxiety. Nat Med. 2010;16:111-5.

24. NCRR Gravity Neutral Orthotic (GNO) Device for people with severe physical disability  Columbia CTSA pilot funded neurologist Petra Kaufmann* built a device to help children with spinal muscular atrophy (SMA)  Partnered with Elisa Konofagou, Ph.D., Assistant Professor of Biomedical Engineering and Radiology  Outcome: Patent IR 2380: Limb motion capture and rehabilitative assist device * = Currently Associate Director for Clinical Research at the National Institute of Neurological Disorders and Stroke, NIH

25. NCRR Wireless Technology to Monitor Patients with Chronic Congestive Heart Failure Scripps CTSA Pioneers Wireless Device  Scripps Translational Science Institute with Qualcomm develop wireless device that monitors heart and breathing rates, fluid status, posture and activity data in patients with congestive heart failure  CTSA program connected Scripps to Qualcomm and other technology firms  A1,200-patient randomized clinical trial to evaluate this wireless device (size of large adhesive bandage) is in progress Dr. Eric Topol (left), director of the Scripps CTSA and Gary West, a philanthropist

26. NCRR A Pharmaceutical Assets Portal  A portal at UC Davis CTSA matches investigators with “shelved” pharmaceutical compounds that can be repurposed for other indications – such as rare diseases  Has generated strong interest among both researchers and pharma community Pfizer initiated agreement with UC Davis to further develop Portal site UC Davis is also in talks with other industry groups who also wish to list their available compounds http://ctsapharmaportal.org

27. NCRR CTSA Academic-Industry Partnerships  University of California San Francisco  Stanford University  University of Pennsylvania  Yale University – Pfizer  Northwestern University  Columbia University – Merck  Washington University – Schering & Pfizer Many CTSA sites have partnerships with industry:

28. NCRR Evaluation  Meeting the expectations of: Congress Other NIH Institutes and Centers  With data from Annual Reports: Stories of Discovery Which Institute’s grants receive CTSA support Publications Trainees and their subsequent funding Patents

29. NCRR Whose research gets CTSA support?

30. NCRR Whose research gets CTSA support?

31. NCRR How many researchers get CTSA support?

32. NCRR Histogram: number of publications in 2009

33. NCRR Challenges  Maintaining cohesion Between KFCs Between KFCs and PIs  Maintaining communication  Sustaining tools and resources that originated through supplements  Ensuring accurate reporting

34. NCRR Future  Please follow our progress at: www.ctsaweb.org