National Center forResearch Resources NATIONAL INSTITUTES OF HEALTH Translating 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 MedicineNovember 1, 2010

Anthony Hayward, MD PhDDirector, Division for Clinical Research,

National Center for Research Resources, NIH, USA

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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

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CTSAs – Creating a Home for Clinical and Translational Science

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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

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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

ILIN

KY

TN

MS AL GA

FL

SC

VAWV

MINY

PA

MD

DE

NJCT

RIMA

ME

VTNH

AK

HI

NC

OH

PR

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NIH funding to successful CTSA applicants

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CTSA – Assembling Local Participants

Yerkes National PrimateResearch Center

Kaiser PermanenteGeorgia

Atlanta VAMedical Center

Complex Carbohydrate Research Center

at U GeorgiaGeorgia BioGeorgia

Research Alliance

Emory University

Morehouse School of Medicine

CDC

GeorgiaTech

Children’sHealthcareof Atlanta

Atlanta Clinical and Translational Science Institute (Atlanta-CTSI) at Emory University

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An Extravert CTSA with Focus on ImpactAn Extravert CTSA with Focus on Impact

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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

Universityof Iowa

University of Chicago

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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 Committeesexpands to 14

2010 Increased attention to administration, Consortium Management Committee

2011 March meeting to review products & goals

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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

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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

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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

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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

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

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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 at http://www.ctsaweb.org/corecompetencies

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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

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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

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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

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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.

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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

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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

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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.

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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

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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

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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

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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:

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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

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Whose research gets CTSA support?

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Whose research gets CTSA support?

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How many researchers get CTSA support?

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Histogram: number of publications in 2009

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Challenges

Maintaining cohesion

• Between KFCs

• Between KFCs and PIs

Maintaining communication Sustaining tools and resources that originated

through supplements

Ensuring accurate reporting

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Future

Please follow our progress at:

www.ctsaweb.org