1. Clinical and Translational Research at Vanderbilt
Gordon Bernard MD
Professor of Medicine
Assistant Vice-Chancellor for Clinical Research

2. Clinical and Translational Research to Improve Health
In vitro studies
In vivo/ animal models
Potential Clinical Application
Feasibility and safety
Efficacy and safety*
Effectiveness and safety*
Basic Science Content of Care Process of Care Structure/Barriers Outcomes
Efficient*
Equitable*
Timely*
Effective*
Safe*
Patient-Centered*
Behaviors:
Patient*
Provider*
System*
Patient Health:
Morbidity*
Mortality*
Satisfaction* Cost*
Access to Care*
Health Policy*
Insurance System*
Community Health*
Epidemiology: Surveillance of System & Outcomes*
Clinical and Translational Research to Improve Health
* Research results trigger assessment for change in care content/process/structure

3. S.E.2 GCRC: Pediatric Research
S.E.1 GCRC: Inpatient and Outpatient Utilization Diversity:
S.E.2 GCRC: Pediatric Research
(N= 69, most recent 12 months)
S.E.3 GCRC: Inpatient and Outpatient Clinical Research Portfolio By Category

4. CTSA – A roadmap initiative
“It is the responsibility of those of us involved in today’s biomedical research enterprise to translate the remarkable scientific innovations we are witnessing into health gains for the nation.”

5. Vanderbilt Institute for Clinical and Translational Research (VICTR)

6. NIH CTSA Awards: A Home for Clinical and Translational Science
Industry
Clinical
Research
Ethics
Trial Design
Biomedical
Informatics
Advanced
Degree-Granting
Programs
CTSA
HOME
Other
Institutions
Participant
& Community
Involvement
Clinical
Resources
Biostatistics
Regulatory
Support

7. Impediments to translation
Current Situation
Small proportion translated
Large number of ideas
T1 and T2 blocks are psychological, organizational,
procedural and physical

8. Organizational Chart for VICTR Administration

9. COMMUNITY!

10. 2. Biostatistics core support 4. Database of collaborators
1. Access to pilot funds
2. Biostatistics core support
3. Synthetic derivative
4. Database of collaborators
% very beneficial
N=575

11. Voucher System Designed to facilitate support for innovation.
Funds pilot type interactions
Scientific consultations (medical model)
Pilot assays
Statistical/informatics imput
Study design assistance
Vouchers
Small – web based request and approval
Medium – Administrative review
Large – GCRC Advisory Comm (GAC)
Automatic tracking of funds provided, completion, value.

12. Structure for Support of Study Development
Hypothesis Studio
Behavioral Research
Core
Surveillance Epidemiology Core
Quality Improvement Research Core
Clinical Epidemiology
Design Studio
Implement Studio
AnalysisStudio
Community Research
TranslationStudio

13. Grassroots Clinical Research: A case study

14. Building a strong clinical trials program:
- creates a national reputation that attracts patients
- allows us to compete as a center for the study of various patient populations for large NIH trials

15. Interstitial Pulmonary Fibrosis Program
Created by James Loyd
Directed by Lisa Lancaster
Evaluates patients for:
Healthcare options
Clinical trials
Lung transplantation
Large referral base
Closest IPF centers a distance away – Duke, Emory, UAB, U. Colorado

16. Growth of IPF Research Center
In 2006 became part
Of the NIH IPF Clinical
Trials Network

17. Vanderbilt DNA Databank

18. Genomics In Full Force Senator Bill Frist (1/05, NEJM):
“in the next decade, the practice of medicine will change dramatically through genetically based diagnostic tests and personalized, targeted pharmacologic treatments …”
Francis Collins (2/05):
Personalized Medicine: How the Human Genome Era Will Usher in a Health Care Revolution

19. Vanderbilt DNA Databank:
Background
Uses left-over clinical samples to create a centralized, cost-efficient genetic research resource
associate genetic information with disease susceptibility and variable drug responses across populations
improve patient safety and reduce adverse drug reactions
Patients have the option to opt-out of participation

20. Most Vanderbilt Patients Support DNA Databank project: Local Survey Results
General favorability apparent*
~80% think the project is very important
~90% think the hospital should be able to use leftover blood for medical research if personal information is removed
~4-5% Oppose this type of genetic research
Scientific aims generate support
Prefer direct notification methods; but a fine line with IRB
* Patient Survey To Assess Attitudes and Opinions (N=1,005)

21. Distinct Attitudinal Groups (Among Respondents Who Could Be Segmented)
Allow opt outs

23. The Synthetic Derivative Medical Information System
One-way Hash ID
Identified
Clinical data
(1.3M records)
“HIPAA” identifiers (name, address…)
removed
Techniques to prevent re-identification based on specific queries
Linked to genetic markers
Replaced record numbers
Random social security numbers
Shifted birthdates, adm dates, etc
Substituted names

24. Comparison to the worlds largest
existing DNA bank
Iceland database:
125,000 records
Genealogy
Homogenous population
Paper medical records
Commercialized
Vanderbilt database:
300,000 planned records*
No genealogy
Heterogeneous population
Electronic medical records
Academic/shared genotypes
*out of 1.5 million possible

25. What Could Be Done? Focus on common, but genetically complex diseases:
Short term
Long term
Confirmation
of role of known polymorphisms in causing disease
Hypothesis
Generation
Rapid assessment of areas suitable for further study
Discovery
of new meaningful SNP’s, haplotypes, targets/pathways
Prediction
Development of statistical models for decision making
Healthcare
Paradigm Shift
Personalized medicine
Focus on common, but genetically complex diseases:
Cancer, heart disease, diabetes, Alzheimer’s, macular
degeneration, arthritis, Parkinson’s, hypertension….
As well as the dugs used widely to treat those diseases

26. Personalized Medicine
September 5th, 2005
“Doctors might be able to screen patients’ DNA and predict not only whether they are likely to have a heart attack but which type of heart disease to expect and which drug or procedure might work.”

27. Artist’s conception of the
Vanderbilt Institute for Clinical and Translational Research
(VICTR)