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INTRODUCTION TO CLINICAL RESEARCH Lecture 1: Who, What, Why and Where of Clinical Research

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INTRODUCTION TO CLINICAL RESEARCH Lecture 1: Who, What, Why and Where of Clinical Research Richard J. Barohn, M.D. Professor and Chairman Department of Neurology – PowerPoint PPT presentation

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Title: INTRODUCTION TO CLINICAL RESEARCH Lecture 1: Who, What, Why and Where of Clinical Research


1
INTRODUCTION TO CLINICAL RESEARCHLecture 1Who,
What, Why and Where of Clinical Research
  • Richard J. Barohn, M.D.
  • Professor and Chairman
  • Department of Neurology
  • Director, General Clinical Research Center
  • University of Kansas Medical School
  • August 24, 2006

2
What is Clinical Research?
  • Types of clinical research
  • Retrospective looking back at data previously
    collected
  • - Case Reports
  • - Case Series
  • Prospective make plan for future data
    collection
  • - Observational
  • - Interventional
  • - Device/technique
  • - Drug

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Observational Research Studies
  • Can involve direct participant contact
  • - Measure a clinical end-point
  • Obtain tissue samples from participant to study
    in the laboratory
  • Can be data-mining of clinical databases
  • - Never see participant
  • - Example Medicare database

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Types of Clinical End-Point Measurements
  • Survival (mortality)
  • Physiologic parameter
  • Example quantitative strength, breathing (vital
    capacity)
  • Clinical scales
  • Example Mini Mental Status
  • Blood or other body tissue measurement
  • Example - Glucose Level
  • - Hormone Level

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Ann NY Acad Sci 1998
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Quantitative MG Score for disease activity
12
Interventional Trials
  • Open-label Trial
  • Everyone receives research intervention
  • Controlled Trial
  • Some receive research intervention, some do not
  • Control group - Ones that do not receive research
    intervention
  • Control group can be getting another intervention
  • Comparing new drug to older one
  • Or control group can get placebo (inactive
    intervention)
  • Randomized Trials
  • Participants randomly allocated to active
    intervention or control group
  • Blinded usually double blind
  • Doctor and participant do not know which group
    subject is in
  • Gold Standard Randomized Double-Blinded
    Controlled Trial

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ALS Treatment Trials at University of Kansas
Medical Center
  • Most proceeded by beneficial results in
    laboratory models
  • All multicenter with other ALS centers
  • COX-2 inhibitor (glutamate inhibitor) (MDA)
  • Celebrex trial just completed no benefit
  • Creatine
  • ? improves strength in athletes (NIH)
  • Minocycline oral (cell death inhibitor) (NIH)
  • Ceftriaxone intravenous (cell death inhibitor)
    (NIH)
  • CoQ10 (antioxidant) (NIH)
  • Arimoclomol (increases heat shock protein)
    (Biotech investigator initiated)
  • Celebrex/creatine vs mino/creatine (ALSA)

17
Drug Trials
  • Pre-Clinical Animal and In Vitro Lab Studies
  • Phase I Safety (normals disease)
  • Phase II Preliminary Efficacy Data with
  • additional safety
  • usually lt 100 patients
  • Phase III Pivotal efficacy trial
  • Large, often multicenter
  • Phase IV - Post-marketing

18
Who Does Clinical Research?
  • In Academic Health Centers
  • Faculty MD, DO, PhD are Principal Investigators
    and Co-Investigators in Schools of Medicine,
    Nursing, Allied Health
  • Trainees (students, residents, fellows) work
    under Faculty
  • Outside of Academic Health Centers
  • Private practice health care providers
  • Industry
  • Pharmaceutical companies, example Quintiles
  • Biomedical device companies

19
Who Does Clinical Research? Team Approach
  • Investigator
  • Clinical Research Coordinators
  • (Example nurse, respiratory therapist, RD)
  • Clinical Research Evaluator
  • Research Assistant
  • Biostatistician
  • Data Manager
  • Research Pharmacy
  • If laboratory based
  • Lab research personnel (students, post doctorate,
    technicians)

20
Who Pays for Clinical Research?
  • Federal Grants
  • Example National Institutes of Health
  • FDA-Orphan Drug Grant Program
  • Foundations
  • Example Muscular Dystrophy Association
  • Industry
  • Internal-funding at medical center
  • Example Research Institute of KUMC
  • Private Donations
  • Non-Funded Research

21
What Types of Grants Should a Junior Clinical
Investigator Apply For?
  • Local Institutional Support
  • KUMC Research Institute
  • Department
  • Disease-Related Foundations
  • Standard Research Grants
  • Career Development Grants
  • NIH (NIH.gov)
  • Apply to NIH for
  • K23 Clinical Research
  • K08 Lab/Translational
  • R03 Pilot Program
  • R21 Developmental/Exploratory
  • Institutional Awarded Training Grants
  • T32 Training
  • K12 Career Development
  • VA Career Development Awards
  • Merit Grants
  • GOAL RO-1 THE BIG ONE

22
Who Monitors Clinical Research?(for science,
safety, privacy)
  • Food Drug Administration (FDA)
  • PI applies to get IND for drug trial
  • Local Human Subjects Committee (Institutional
    Review Board)
  • Local HIPPA Compliance Program
  • General Clinical Research Center
  • Research Subject Advocate
  • Safety Monitors
  • Data Safety Monitoring Boards
  • Local KUMC DSMB
  • National NIH DSMB

23
Who Helps in Clinical Research at KUMC?
  • Research Institute Clinical Trials Office
  • Serve as centralized sponsor contact point for
    KUMC
  • Prepares consent with PI input
  • Prepares and negotiates trial budgets
  • Submits protocol/consent to HSC if there is a
    sponsor
  • Contact Laurie Kemble at 588-1242 or
    http//www2.kumc.edu/researchinstitute/
  • Human Subjects Committee
  • KUMCs Institutional Review Board
  • Committed to ethical, legal and safe conduct in
    all research involving human participants
  • Meets 2nd and 4th Tuesdays
  • Submissions by noon Friday, 7 business days prior
    to meeting
  • Submission of materials
  • Contact Karen Blackwell at 588-0942 or
    http//www.kumc.edu/hipaa/
  • Mentors

24
Mentoring Analogy to Driving Lessons
  • Focus on steering mechanism not the
  • drive train or brakes, as they are
  • not usually accessible to mentors.

25
Keys in Finding a Mentor
  • The mentor should
  • Be interested
  • Have experience
  • Be available
  • Limit number of mentees
  • Commit substantial time
  • Be vigilant but not intrusive

26
Where is Clinical Research Done?
  • In academic health centers
  • Departmental clinical space
  • Designated research space
  • Example GCRC
  • Hoglund Brian Imaging Center
  • Veterans Administration Medical Centers
  • Hospitals not affiliated with AHC
  • NIH in Bethesda intramural
  • Private health care provider offices
  • Industry offices

27
Why Do Individuals Want to Participate in
Research?
  • Advance science/improve knowledge
  • Obtain access to a potential Rx at the earliest
    possible time
  • Current Rx not working
  • It helps maintain hope - better than not doing
    anything
  • Desire to meet and speak with researchers
  • To help others
  • Reimbursement to subject/patient

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Why Do Individuals not Participate?
  • Painful unpleasant procedures
  • Fear of going off current medication
  • New treatment may not be better
  • Time commitment/travel
  • Side effects
  • Fear of the unknown
  • Fear of being assigned to placebo
  • They accept the disease is their fate

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Why Do Individuals not Participate ?(More
Reasons)
  • Efforts futile
  • Privacy concerns publication, etc.
  • Resentment of medical personnel
  • Distrust of science community
  • Dont want to be a guinea pig
  • Dont agree with focus of research
  • Long wait to get research results

30
What Do Participants Want/Expect from Involvement
in Clinical Research?
  • Be considered a partner in the research
  • Be treated with respect / taken seriously, kept
    informed and up-to-date
  • Do not want to be talked down to
  • Full disclosure of side effects
  • Option to stop involvement in study
  • Support from research team - availability

31
What Do Participants Want/Expect from Involvement
in Clinical Research? (continued)
  • After study, want to know results and if they
    were on drug or placebo
  • Realistic idea when results will be available
  • No cost to participant
  • Confidentiality
  • Enough time and comfortable area
  • To ask questions
  • In which to do the study

32
General Clinical Research Center Program
33
What is a General Clinical Research Center?
  • A NIH-supported multidisciplinary research unit
    which facilitates investigator-initiated clinical
    studies and trials conducted by full-time faculty
    of the AHC.
  • Provides clinical research infrastructure to
    investigators who receive funding from federal
    agencies, private foundations, other
    peer-reviewed sources.
  • Also, can include investigator initiated unfunded
    pilot studies and industry sponsored studies.
  • This is at no cost to the PIs for
    Investigator-Initiated Trials

34
General Clinical Research Center (GCRC) at KU
Medical CenterGoals established in 2002
  • Provide clinical investigators from the SOM, SON,
    and SOAH with a modern, state-of-the-art facility
    in which clinical research could be conducted
  • Enhance multidisciplinary research across
    departments and the three schools
  • Enable and train junior faculty and trainees to
    become more involved in clinical research
  • Apply for federal funding to support the GCRC

35
Typical GCRC Organizational Structure
Principal Investigator (PI) (Usually Dean
of School of Medicine)
GCRC Advisory Committee (30 Members) (GAC)
Program Director (Senior Faculty/Investigator)
Facilities
  • Outpatient Unit
  • Inpatient Unit
  • Scatter Beds
  • Core Lab
  • Metabolic Kitchen
  • Informatics Core

36
GCRC Advisory Committee (GAC) Members - June
2006Matthew Mayo, PhD Jared Grantham, MD
(Co-Chairs)
  • VOTING MEMBERS
  • Matthew Mayo, PhD
  • Jared Grantham, MD
  • Marge Bott, RN, PhD
  • Richard McCallum, MD
  • Stephen Williamson, MD
  • Richard Dubinsky, MD
  • Barbara Lukert, MD
  • Kevin Latinis, MD, PhD
  • Debra Sullivan, PhD, RD, LD
  • Patrick Moriarty, MD
  • Jo Ann Harris, MD
  • John Ferraro, PhD
  • Andrea Charbonneau, MD
  • Kathryn Ellerbeck, MD
  • Ossama Tawfik, PhD, MD
  • Barbara Quaney, PT, PhD
  • Kathleen Gustafson, PhD
  • NON-VOTING EX OFFICIO MEMBERS
  • Barbara Atkinson, MD Executive Dean SOM
  • Richard J. Barohn, MD Program Director
  • Curt Hagedorn, MD Associate Director
  • Jeff Burns, MD Assistant Director
  • Patricia Kluding, PT, PhD Assistant Director
  • Paul Terranova, PhD
  • Susan Schmitz, BA, CCRC Administrative Director
  • Judy Otey, RN, BSN Nurse Manager
  • Ed Ellerbeck, MD, MPH
  • Jon Jackson
  • Jo Denton, MSN
  • Laurie Kemble, BS, CRT

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What The GCRC Can Do
  • For GAC approved studies
  • Space to see patients
  • Biostat support study design
  • Data management
  • Nurse support
  • Administrative support
  • Specimen collection/storage
  • Common equipment
  • Some labs
  • No cost for above services to PIs on
    investigator initiated studies
  • Industry sponsored with charges for
    space/resources

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What The GCRC Cannot Do
  • Space/resources for industry studies
  • Permanent space for research coordinators
  • Overnight stay on GCRC unit

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A Visit to the GCRC
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KUMC - GCRC Time-line/Progress
  • GCRC Planning Committee formed 2002
  • Construction/Remodeling began June 1, 2004
  • Completed October 2004
  • 6, 000 square feet in Delp
  • Announce GAC will accept research applications
    from investigators, September 2004
  • Began doing studies on GCRC January 2005
  • Approval of 53 on-going studies by GAC as of June
    2006
  • GCRC NIH grant submitted June 1, 2006
  • 2 million (direct costs) per year for 3 years

42
To Find Out More Information About the GCRC
  • Contact Judy Otey, RN, BSN
  • GCRC Administrative Director
  • 913-588-0984 alt. 2460
  • jotey_at_kumc.edu
  • Nicole Ladesich, BS
  • GCRC Senior Coordinator
  • 913-588-0976 alt. 2294
  • nladesich_at_kumc.edu
  • Visit Website http//gcrc.kumc.edu

43
How to access the GCRC websitewww.gcrc.kumc.edu
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Current Challenges for Clinical Researchas
perceived by Dr. Zerhouni in May 2005 and NEJM
October 2005
  • Recruiting and retaining researchers
  • Explosion in clinical demands, reduced financial
    margins ? limit time, diluted value
  • Regulatory burden
  • Fragmented training
  • Complexity of training
  • No real HOME for clinical researchers
  • Limitations / barriers due to NIH funding
    mechanisms, review, program structures

45
Definitions per Dr. Zerhouni / NIH
  • Clinical Research covers all studies of diseases
    and trials of
  • treatments in human subjects
  • Translational Research describes the steps
    between a
  • fundamental discovery and its application in
    clinical
  • medicine. For example
  • Testing a new anti-cancer drug in humans for the
    first time
  • Identifying best practices in the diagnosis,
    prevention, or management of a disease and
    enhancing their adoption by the community

46
Current Clinical Science Infrastructure as
perceived by Dr. Zerhouni
Disease X Center
GCRC
Disease Y Center
Training Programs
K30 Curriculum
47
Dr. Zerhounis ModelSystems Biology Approach
to Creating a Home for Clinical and Translational
Sciences

Disease X Center
Upgraded Biostatistics
IRB
Upgraded Regulatory Advice
???
NECTAR NCRA
Degree Granting
Training Programs
Clinical Research Design Incubator
Disease Y Center
K30 Curriculum
Translational Cores
GCRC
Upgraded Informatics
???
NIH RAID
48
Institutional Clinical and Translational Science
Award (CTSA)RFA announcement Oct. 12, 2005
  • Purpose Forge a transformative and integrative
    academic home for clinical and translational
    science
  • The home must be a Center, Department, or
    Institute.
  • Encompass all components of clinical research
    (education, career development, clinical research
    infrastructure)
  • Promote multidisciplinary research teams
  • Create an incubator for innovative research tools
  • Catalyze the application of new knowledge to
    clinical practice
  • Degree granting capabilities in Clinical Research
  • Masters and/or PhD

49
KUMC Strengths and Weaknesses for CTSA Application
  • Weaknesses
  • No current GCRC funding
  • No T32
  • Current K12 applicability?
  • Small pool of clinical mentors, current clinical
    R01s
  • Limited culture for CR
  • Strengths
  • K30
  • GCRC infrastructure
  • Institutional support
  • Research Institute
  • SOM / SON / SOAH
  • Bioinformatics Center (Mayo)
  • K-BRIN (Hunt)
  • KU Lawrence (Georg and others)
  • Intro to CR Course

50
KUMC Strengths and Weaknesses for CTSA Application
  • Strengths
  • K30 (MSCR program)
  • GCRC infrastructure
  • Institutional support
  • Research Institute
  • SOM / SON / SOAH
  • Bioinformatics Center (Mayo)
  • K-BRIN (Hunt)
  • KU Lawrence
  • Life Span Institute
  • Drug Development
  • Introduction to CR Course
  • Weaknesses
  • No current GCRC funding
  • No Clinical T32
  • Small pool of clinical mentors, current clinical
    R01s
  • Limited culture for CR

51
CTSA Application Funding and Timeline
  • Letter of Intent February 27, 2006
  • Grant Application March 27, 2006
  • Planning grant 150,000 for 1 year
  • Full CTSA up to 6 million with pediatrics
  • 4 million without pediatrics
  • Existing K30, T32, and GCRC are in addition
  • 5 year award RFA offered annually
  • Goal 60 CTSAs to be awarded by 2012
  • Will replace all GCRCs and NCRR / Roadmap K12s

52
CTSA Planning Process at KUMCEstablished CTSA
Planning Committees and Sub-Committees in
October 2005
  • Planning Steering Committee
  • Chairs
  • Barbara Atkinson, MD
  • Richard J. Barohn, MD
  • Governance Planning Sub-Committee
  • Chairs
  • Richard J. Barohn, MD
  • Barbara Atkinson, MD
  • Grant Writing Planning Sub-Committee
  • Chairs
  • Richard J. Barohn, MD
  • Lauren Aaronson, PhD, RN
  • Education Planning Committee
  • Chair Ed Ellerbeck, MD, MPH
  • Clinical Research Resources Planning Committee
  • Regulatory Planning Committee
  • Chairs
  • Jim Voogt, PhD
  • John Finley, JD, MPH
  • Novel Methods Translational Technologies
    Planning Committee
  • Chairs
  • Curt Hagedorn, MD
  • Paul Terranova, PhD
  • Health Disparities Research Planning Committee
  • Chairs
  • Patricia Thomas, MD
  • Kirby Randolph, PhD
  • Community Participant Planning Committee
  • Chairs
  • Joshua Freeman, MD
  • Lauren Aaronson, PhD, RN

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