Major Trends in Biomedical Research
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Transcript Major Trends in Biomedical Research
RE-ENGINEERING THE CLINICAL
RESEARCH ENTERPRISE
Co-Chairs
Working Groups and Co-Chairs
Stephen Katz, NIAMS
Stephen E. Straus, NCCAM
Subgroups
Harmonization of Clinical Research Regulatory Processes
Amy Patterson, OSP
Integration of Clinical Research Networks, including NECTAR
Larry Friedman, NHLBI
Stephen Katz, NIAMS
Enhance Clinical Research Workforce Training
Duane Alexander, NICHD
Rob Star, NIDDK
Enabling Technologies for Improved Assessment of Clinical Outcomes
Deborah Ader, NIAMS
Larry Fine, OBSSR
Stephen Katz, NIAMS
Regional Translational Research Centers
Stephen E. Straus, NCCAM
Steve Zalcman, NIMH
Translational Research Service Cores
Josephine Briggs, NIDDK
Stephen E. Straus, NCCAM
Re-engineering the Clinical
Research Enterprise
Stephen Katz, M.D., Ph.D
Amy Patterson, M.D.
Stephen E. Straus, M.D.
Re-engineering Clinical Research
Interdisciplinary
Research
Pioneer Award
Bench
Public-Private Partnerships
Bedside
Practice
Building Blocks
and Pathways
• Translational • Integrated Research Networks
Molecular Libraries
Research
• Clinical Research Informatics
Bioinformatics and
Cores
• National Clinical Research Associates
Computational
• Regional
• Patient-reported clinical outcomes
Biology
Centers
Structural Biology
Nanomedicine Cross cutting: Harmonization, Training
Integration of Clinical Research
Networks
Link existing networks
so clinical studies and
trials can be conducted
more effectively
Ensure that patients,
physicians and scientists
form true “Communities
of Research”
Typical NIH Network
Academic Health Center Sites
& Data Coordinating Center
Interoperable Networks
Share Sites and Data
Integration of Clinical
Research Networks
Create interoperable ‘Network of Networks’
National Electronic Clinical Trials/Research Network
(NECTAR)
Common data standards, informatics
Software application tools for Protocol preparation, IRB
management, adverse event reports
Use existing networks to rapidly address questions
beyond their traditional scope
BAA-RM-04-23: Feasibility of Integrating and Expanding Clinical Research Networks
Inventory and Evaluation of Clinical Research Networks: NIH Task Order 169
NIH Career Development Programs
College
Professional
Grad School
•NIH CR Training Program;
•Advanced Degree Program?
Post-Doc
Prof. Advancement
Trans-NIH K12 CR Career Dev;
National CR Associates Program
Trans-NIH Multidisciplinary K12
Career Development Program (RFA)
Promote development of investigators from a
variety of disciplines (M.D., Ph.D., R.N.-Ph.D.,
D.P.H., Pharm.D., D.C., …)
To be trained in multidisciplinary team settings
Goal:
Features:
Up to 5 years of training
Core didactic courses, Project-specific training
Mentored research experience in team settings
Faculty and mentor support to protect their time
Tuition support
Annual meetings
National Clinical Research Associates
Goal:
Steps:
Diverse national group of trained and certified
community healthcare providers
Will enroll and follow their own patients
Accelerate translation of results into practice
Determine feasibility: Barriers? Communities?
Incentives needed?
Inventory training methods, best practices
Develop core competencies, certification
Future: Training programs
Dynamic Assessment of PatientReported Chronic Disease Outcomes
Background:
Assessment of chronic diseases relies heavily on subjective reports
of symptoms and health related quality of life items
PROMIS – Patient-Reported Outcomes Measurement
Information System:
A collaborative network of investigators charged to develop and
implement a publicly available system of a large item bank and a
Computerized Adaptive Tests for more sensitive and efficient testing
of major chronic disease symptoms and functioning.
RFA-RM-04-011: Dynamic Assessment of Patient-Reported Chronic Disease
Outcomes
Re-engineering Clinical Research
Interdisciplinary
Research
Pioneer Award
Bench
Public-Private Partnerships
Bedside
Practice
Building Blocks
and Pathways
• Translational • Integrated Research Networks
Molecular Libraries
• Clinical Research Informatics
Research
Bioinformatics and
Cores
• National Clinical Research Associates
Computational
• Regional
• Patient-reported clinical outcomes
Biology
Centers
Structural Biology
Nanomedicine Cross cutting: Harmonization, Training
Clinical Research Policy
Analysis and Coordination
“Harmonization Initiative”
Clinical Research:
Navigating the Roadway
Clinical research impeded
by multiple and variable
requirements to address
fundamentally the same
oversight concerns
Variability among and within
agencies
Creates uncertainty
about how to comply
Hampers efficiency and
effectiveness
Clinical Research Policy
Analysis and Coordination Initiative
AIM:
Promote clear, effective, and
coordinated policies and regulations
for the conduct and oversight of
clinical research
Priority Issues
1.
Diverse adverse event reporting requirements
2.
Confusion regarding roles and responsibilities of
Data Safety and Monitoring Boards, IRBs, and other
review mechanisms
3.
Variable requirements for auditing and monitoring of
clinical trials
4.
Absence of uniform standards for electronic
submission of safety and clinical research
information
Priority Issues
5.
Confusion regarding applicability of privacy
requirements and HIPAA to clinical research
6.
Variability in interpretation of the human subjects
regulations
7.
Central vs. local IRB review
8.
Best practices in informed consent
9.
Investigator financial disclosure and conflicts of
interest
Methods
Establish formal NIH program to:
Work in concert with sibling agencies to catalyze the
federal-wide coordination of policies pertaining to
clinical research
Consult with key stakeholders including IRB, patient,
industry, and research communities
Develop tools and resources to facilitate
understanding of and compliance with clinical
research policies and requirements
Harmonized Adverse Event
Reporting Requirements
Gene Therapy Example
Prior to January 2002:
Principal Investigators had to report to NIH all serious
adverse events immediately
At variance with FDA requirements (21 CFR 312)
Current harmonized requirement:
Possibly associated, unexpected events reported within 15
days - or within 7 days if fatal or life threatening
Scope, timeframe, and definitions for safety reporting are
same for NIH and FDA
Analysis and Communication of
CR Information
Prevailing paradigm
Paper based
Varied information and diverse formats and vocabularies
Inefficient transmission and limited comparison of data sets
Model approach
Electronic data capture, analysis and reporting
Uniform data elements and format accepted by multiple agencies
Rapid transmission and ready comparison of data sets
Objectives
Facilitate investigator compliance
Enhance agency oversight
Optimize data sharing and analysis
Outcomes
Clear, effective, and coordinated rules for clinical
research
Maximally effective human subjects protections
More comprehensive analysis and sharing of
research data
Enhanced quality and productivity of the
research enterprise
Translational Research
Definition:
Research at the bench-bedside interface designed to
define disease pathobiology and to explore innovative
approaches to treatment and prevention
A Problem:
Academic investigators too often lack the resources and
expertise to study diseases or evaluate novel drugs and
biologics in early phase (I-II) human studies using stateof-the-art tools
Facilitating Translational Research
Interdisciplinary
Research
Pioneer Award
Bench
Public-Private Partnerships
Bedside
Practice
Building Blocks
and Pathways
• Translational • Integrated Research Networks
Molecular Libraries
Research
• Clinical Research Informatics
Bioinformatics and
Cores
• National Clinical Research Associates
Computational
• Regional
• Patient-reported clinical outcomes
Biology
Centers
Structural Biology
Nanomedicine Cross cutting: Harmonization, Training
Translational Research:
Goals for the Initiatives
Stimulate research at the bench-bedside interface
Implement mechanisms to prepare novel products
for pre-clinical and clinical studies
Ensure adequate support for regulatory oversight,
data management, and specimen analyses
Increase the sensitivity and impact of early phase
clinical studies
Invest sufficiently in a modest number of programs
to facilitate research at many institutions
Translational Research Core Services
Extensive consultations with leaders in academia and
industry
Pilot program will support early pre-clinical development
of small molecules for therapeutic applications
Modeled on NCI RAID program – Provides investigators
access to contractor facilities for key services
Requires extensive decision-making expertise, process
controls, and regulatory oversight
Funded ~25% by Roadmap; ~75% by sponsoring IC
Current status: Pilot initiative for implementation in
FY2005
Regional Translational
Research Centers
Current options for resources:
Regulatory, statistical, bioethics & data management support
Support bench-to-bedside and pilot projects, translational
research fellows program
Cores in some sites for pharmacology, immunology, genetics,
microarray, bioinformatics and other highly technical services
Could complement NCRR’s GCRC program, and support
phase I studies of products made by the Core Services program
Next Steps:
National meeting of academic opinion leaders in summer 2004
Goal is to call for and fund planning grants in FY2005, and rounds of
new centers starting in FY2006
Re-engineering Clinical Research
Interdisciplinary
Research
Pioneer Award
Bench
Public-Private Partnerships
Bedside
Practice
Building Blocks
and Pathways
• Translational • Integrated Research Networks
Molecular Libraries
• Clinical Research Informatics
Research
Bioinformatics and
Cores
• National Clinical Research Associates
Computational
• Regional
• Patient-reported clinical outcomes
Biology
Centers
Structural Biology
Nanomedicine Cross cutting: Harmonization, Training
Re-engineering Clinical Research:
A Work in Progress
Re-engineering Clinical Research:
Progress To Date (as of February 2004)
Multidisciplinary Clinical Research Career
Development (K12) programs: RFA-RM-04-006
Dynamic Assessment of Patient-Reported
Chronic Disease Outcomes: RFA-RM-04-011
Inventory and Evaluation of Clinical Research
Networks: NIH task order 169
Feasibility of Integrating and Expanding
Clinical Research Networks: BAA-RM-04-23
Re-engineering Clinical Research:
Future ‘05 Initiatives
Translational Research
Translational Research Core Services pilot
program
Planning grants for Regional Translational
Research Centers
Clinical Research
Feasibility of the National Clinical Research
Associates program
NIH
Ideas
People
Resources
Leadership