Clinical and Translational Research:Expectations and Goals

Daniel E. Ford, M.D., M.P.H.Director, Institute for Clinical and Translational ResearchVice Dean for Clinical Investigation

Clinical Research at Johns Hopkins

• Comprehensive in Scope• Cancer Center• Research in children• Neurosciences• Drug abuse

• Investigator Initiated• Large Discovery Pipeline• Expertise in Moving Discoveries to Population • Fortunate to Have Large Number of Trainees

Interested in Research

Research at ClOffJohns Hopkins Medicine

By Calendar Year Last updated 4/5/2010      

New Applications   2007 2008 2009

  NHSR 35 48 99

  New Exempt 443 364 311

  New Expedited 561 688 830

  New Convened 418 520 569

         

  Total New 1457 1620 1809

         

Applications   2007 2008 2009

  Active eIRB Applications 1552 2198 2890

  Active Paper Applications 1051 859 554

  Active SOM IRB 2603 3057 3444

         

  WIRB Active Applications 405 297 200

  CIRB Active Applications 32 58 63

  Total Active 3040 3412 3707

         

Randomized Clinical Trials   2007 2008 2009

  Active Randomized Clinical Trials 724 815 854

  New Randomized Clinical Trials 247 376 405

Commercial Applications   2007 2008200

9

  Active Commercial 550 543 487

  New Commercial 262 322 270

Commercial Clinical Trials   2007 2008

2009

  Active Commercial Clinical Trials 385 385 360

  New Commercial Clinical Trials 181 202 243

Using Drugs/Devices   2007 2008200

9

  Active Applications Using Drugs 869 1024 1091

  New Applications Using Drugs 282 346 386

         

  Active Applications using Devices 288 327 245

  New Applications using Devices 104 118 102

IDE/INDs Applications   2007 2008200

9

Investigator/Sponsor Held Active Applications using INDs 383 496 523

  New Applications using INDs 157 198 156

  Active INV Held INDs 74 118 162

  New INV Held INDs 13 55 82

         

  Active Applications using IDEs 12 29 44

  New Applications using IDEs 9 21 20

  Active INV Held IDEs 7 13 14

  New INV Held IDEs 3 3 2

Sponsored Project SourcesJohns Hopkins SOM – Prorated Awards

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100

200

300

400

500

600

700

Fiscal Years

$ (M

illi

on

s)

Commercial 32.8 40.6 39.7 43.9 49.8 50 53.6 69.4 62.9 52.6

Other 38.7 45.2 50.4 56.7 60.6 66.9 70.4 103 97.9 107

Federal 212 209 209 244 299 329 399 451 461 456

97 98 99 0 1 2 3 4 5 6

Translational Medicine

• T1 – “the transfer of new understandings of disease mechanisms gained in the laboratory into the development of new methods for diagnosis, therapy, and prevention and their first testing in humans.”

• T2 – “the translation of results from clinical studies into everyday clinical practice and health decision making.”

• IOM Clinical Research Roundtable

Translational Research Activities

• T1 activities included: 1) the translation of basic discovery into mechanistic studies in cell lines or animals, 2) the translation of mechanistic studies into initial human testing, and 3) the translation of initial human testing into proof of efficacy.

• The T2 activities included 1) the translation of proof of efficacy into proof of effectiveness in a usual care setting, and 2) research aimed at enhancing the adoption of best practices in the community

Conclusions

• 69% responded they are translational researchers (9% other survey but different definition)

• PhD and fellows report less translational activity

• Barriers to translational research are as expected but substantial

84

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11

5

0 10 20 30 40 50 60 70 80 90

Translational research is not part of my research agenda or

is not central to the goals of my research program.

I don’t have the resources (funding, space, equipment,

technology) for making my research more translational.

I have not yet identified an appropriate translational

research question to pursue.

I am not sure what the next translational step would be for

my research.

I haven’t identified or secured the colleagues or

collaborators I need to make my work more translational.

I don’t have the guidance or mentorship I need to make my

research more translational.

I don’t have the training or expertise I need to make my work

more translational.

I have methodological obstacles to making my research

more translational.

It is premature for me to consider translational research.

Number of Respondents (n=205)

It is premature for me to consider translational research.I have methodological obstacles to making my research more translational.I don’t have the training or expertise I need to make my research more translational.I don’t have the guidance or mentorship I need to make my research more translational. I haven’t identified or secured the colleagues or collaborators I need to make my work more translational.I am not sure what the next translational step is

I have not yet identified an appropriate translational research question to pursue.

I don’t have the resources (funding, space, equipment, technology) for making my research more translational.Translational research is not part of my research agenda or is not central to the goals of my research program.

Barriers to Translational Research (n=205)

Translational Pathway

Basic Discovery

MechanisticStudies

Initial Human Testing

Proof of Efficacy

Proof of Effectiveness

Diffusion toAll HealthCare Settings

21

Institute for Clinical and Translational ResearchInfluence on Scientific Discovery

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Enhancing Formulation of Creation of Development Conduct Analysis of PublicationObservation Scientific Question Research Team Study Protocol of Study Study Data Impact Study

Advisory Boards

Protocol Review

Translational Science Forums

Science Cores

Innovation Groups

Biostatistical Support

Regulatory Support

ATIP

Research Ethics

Clinical Research Units

Clinical Research Participant Core

Data/Safety Monitoring

Community Engagement Program

Biomedical Informatics

Navigator Program

Research Education and Training

Impetus for the CTSA Program

Implementing biomedical discoveries Implementing biomedical discoveries made in the last 10 years demands an made in the last 10 years demands an evolution of clinical science.evolution of clinical science.

New prevention strategies and treatments New prevention strategies and treatments must be developed, tested, and brought must be developed, tested, and brought into medical practice more rapidly.into medical practice more rapidly.

CTSA awards will lower barriers between CTSA awards will lower barriers between disciplines, and encourage creative, disciplines, and encourage creative, innovative approaches to solve complex innovative approaches to solve complex medical problems.medical problems.

These clinical and translational science These clinical and translational science awards will catalyze change -- breaking awards will catalyze change -- breaking silos, breaking barriers, and breaking silos, breaking barriers, and breaking conventions.conventions.

Johns Hopkins and Translational Medicine

• Need to support researchers at all career levels• Progress in translational medicine requires development of teams of

researchers that appreciate all phases • Clinical and translational researchers will continue to develop more

technical and specialized approaches. Handoffs require active coordination (project coordinators).

• Do to increasing regulations, researchers will need to rely more on systematized solutions at institutional or national level

• Academic organizations need to partner better with other organizations to increase efficiency (industry, payers, patient groups)

• Need to balance resources/support for early and late translation• Academic centers need better management structure to

systematically monitor research process and barriers

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Building a National CTSA Consortium

Since 2006Since 2007

Participating Institutions

CTSAWeb.org

Consortium Governance & Organization

Governance Manual available at http://ctsaweb.org/Docs/CTSA_Governance_Manual.pdf

Institute for Clinical and Translational Research

• Goals – Academic Home for Clinical and Translational

Researchers– Expand the Working Relationships of Clinical

Investigators with Basic Scientists and Population-oriented Scientists

– Coordinate Translational Research Activities to Increase Innovation and Speed of Translation

Institute for Clinical and Translational Research

• Goals – Support Training of Clinical and Translational

Research Faculty and Staff– Provide Centralized Support for Research Where

Efficient– Measure and Track Efficiency of Human Subjects

Research to Address Barriers

• Director – Daniel Ford, MD, MPH Vice Dean for Clinical Investigation

Deputy Directors of the Institute for Clinical and Translational Research

• Dr. Charles Balch• Dr. Stephen Desiderio• Dr. Charles Flexner• Dr. Elizabeth Jaffee• Dr. Pete Miller• Dr. Pamela Ouyang• Dr. Jeff Rothstein• Dr. Pamela Zeitlin

Trial Design

Advanced Degree-Granting

Programs

Participant& CommunityInvolvement

RegulatorySupport

Biostatistics

ClinicalResources

BiomedicalInformatics

Research participantRecruitment office

Johns HopkinsJohns Hopkins ICTRICTR

NIH & other government

agencies

Healthcare organizations

IndustryIndustry

Johns Hopkins Institute for Clinical and Translational Research

Johns Hopkins ICTR Progams

• Translational research training– K junior faculty/fellows– Medical students– Research staff and community physicians

• Clinical Research Units – 1200 inpatient research bed days– Over 10,000 outpatient research visits– Pediatric and adult beds including Bayview

Johns Hopkins ICTR Progams

• Biostatistical Consultation

• Innovative Methodology Workgroups

• Clinical Research Ethics Consultations

• Data and Safety Monitoring Services

• Translational Research Navigators (project managers)

• Clinical Research Management Database

Johns Hopkins ICTR Progams

• Basic Discovery Translational Forum• Clinical Sciences Translational Forum• Accelerated Translational Incubator

Program (ATIP) support pilots• Research Participant Recruitment and

Retention Program• Community Research Networks Office• Secondary Translation or Knowledge

Transfer Office

Clinical Research Management System

BudgetingSponsor Billing

Investigators Research Nurse

Data Manager

Insurance Clearance

Budgeting by Dept. Admin.

ProgramManager

Patient/Protocol Registry

Pharmacy CoreFacilities

Labs SoMLeadership

Billing Compliance

IRB

EligibilityScreening

EPR/Labs Integration

IRB Integration

ProtocolSchema / Patient

Calendar

BillingCompliance

External Recruiting

Website

JHED/Site Minder Integration

Data Warehouse

Library Research

Forms

Work InProgress

CurrentlyIn Use

PossibleFuture

FunctionalityKey:

Translational Cores

• Genetics Core

• Proteomics/Biomarker Core

• Drug and Vaccine Development Core