Anthony Hayward, MD PhD Director, Division for Clinical Research,

1. The United States National Institutes of Health Clinical and Translational Science Award Program: An OverviewJCSMR First International Conference on Translational MedicineNovember 1, 2010 Anthony Hayward, MD PhDDirector, Division for Clinical Research, National Center for Research Resources, NIH, USA

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

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

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

6. NIH funding to successful CTSA applicants

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

8. An Extravert CTSA with Focus on Impact

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

10. Assembling a Consortium: Developmental Milestones • 2006 First 12 agree to work together and ask NIH Clinical Center to join them • 2006 9 Key Function Committees established • 2008 First 24 start on Strategic Plan • 2009 Strategic Goal Committees in place. Number of Key Function Committees expands to 14 • 2010 Increased attention to administration, Consortium Management Committee • 2011 March meeting to review products & goals

11. Top Down or Bottom up? • CTSA Consortium Steering 80 • Child Health Oversight 158 • Biostatistics, Epidemiology, RD 141 • Ethics 226 • Clinical Research Management 216 • Communications 110 • Community Engagement 105 • Comparative Effectiveness 105 • Education, Career Development 176 • Evaluation 132 • Informatics 278 • Public-Private Partnerships 101 • Regulatory 187 • Translational 123

12. Strategic Goals for the CTSA Program The CTSA consortium will enhance the efficiency and quality of clinical and translational research to improve the health of the nation • 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 Each Strategic Goal Committee comprises 3 PIs and 3 administrators

13. 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

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

16. Training Clinical and Translational ScientistsCore 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 athttp://www.ctsaweb.org/corecompetencies

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

18. 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. 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. 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. Promoting Adolescent Health in Chicago Schools • 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

22. 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. A Pilot Project on Panic Anxiety • Panic anxiety model in rats finds increased orexin gene expression in neurons. An RNAi protects rats from symptoms • 2nd 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. 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. 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. 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. CTSA Academic-Industry Partnerships Many CTSA sites have partnerships with industry: • University of California San Francisco • Stanford University • University of Pennsylvania • Yale University – Pfizer • Northwestern University • Columbia University – Merck • Washington University – Schering & Pfizer

28. 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. Whose research gets CTSA support?

30. Whose research gets CTSA support?

31. How many researchers get CTSA support?

32. Histogram: number of publications in 2009

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

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