NIH Roadmap for Medical Research

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NIH Roadmap for Medical Research

• ACCELERATING MEDICAL DISCOVERY TO IMPROVE HEALTH

www.nihroadmap.nih.gov
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Evolving Public Health Challenges

• Acute to Chronic Diseases
• Aging Population
• Health Disparities
• Emerging Diseases
• Biodefense

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U.S Health Expenditures
(Percentages of GDP)
18
Actual Projected
16
Percent
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12
10
1985
1987
1989
1991
1993
1995
1997
1999
2001
2003
2005
2007
2009
2011
Year
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Science-Driven Opportunities

• Human Genome Project
• Functional Genomics
• Proteomics
• Integrative Biology of Disease
• Revolutionary Research Methodologies
• Information Sciences

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Need to Transform Medical Research in the 21th
Century
21th Century
20th Century
Intervene before symptoms appear and preserve
normal function for as long as possible
Treat disease when symptoms appear and normal
function is lost
Understanding of preclinical molecular events and
ability to detect patients at risk
We did not understand the molecular and cellular
events leading to disease
Orders of magnitude more effective
Expensive in financial and disability costs
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Imperatives for NIH

• Accelerate pace of discoveries in life sciences
• Enable deeper understanding of pathobiology of
  disease prior to irreversible damage
• Translate research more rapidly from laboratories
  to patients and back
• Explore novel biomedical strategies orders of
  magnitude more effective than current ones
• NIH Roadmap

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What is the NIH Roadmap?

• A framework of priorities the NIH as a whole must
  address in order to optimize its entire research
  portfolio.
• A set of initiatives that are central to
  extending the quality of healthy life for people
  in this country and around the world.
• A vision for a more efficient and productive
  system of biomedical and behavioral research.

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How was the NIH Roadmap developed?

• Extensive consultations with stakeholders,
  scientists, health-care providers
• Discussions addressed
• What are todays scientific challenges?
• What are the roadblocks to progress?
• What do we need to do to overcome roadblocks?
• What cant be accomplished by any single
  Institute but is the responsibility of NIH as a
  whole?

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Criteria for Selecting Roadmap Initiatives

• Is it transforming -- will it change how or
  what biomedical and behavioral research is
  conducted in the next decades?
• Would its outcome enhance the ability of all
  Institutes to achieve their missions?
• Can the NIH afford NOT to do it?
• Does it address key issues of interest to
  stakeholders, especially the public?
• Is it something that no other entity can or will
  do?

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NIH Roadmap Goals

• Accelerate basic research discoveries and speed
  translation of those discoveries into clinical
  practice
• Explicitly address roadblocks that slow the pace
  of medical research in improving the health of
  the American people

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Three Themes Emerged
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NIH Roadmap for Medical Research
Bench
Bedside
Practice
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Roadmap Funding(dollars in millions)
Directors Fund 35 59 83
Total NIH budget in the same period is gt 220B.
Roadmap initiative is about 0.8 of NIH budget.
Totals on table may not add due to rounding
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NIH Roadmap within NIH Budget
Non-Roadmap 99.2 (28,520 Million)
Non-Roadmap 99.2 (28,520 Million)

Roadmap 0.8 (237 Million)
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How does the NIH Roadmap benefit research funded
by NIH ICs?

• Speeding removal of major and fundamental
  roadblocks common to all diseases
• Institutes working together to solve issues
• This is a common trans-NIH pool of transforming
  investments open to all disease areas and all
  investigators for competition

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FY2004 Funded Projects and Activities

• Development of new tools and technologies
• Preparation of research workforce for integrative
  and team science
• Support for planning feasibility studies and
  clinical research policy analysis
• Testing of new approaches to research review and
  support

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New Pathways to Discovery
Initiatives within this theme address
technologies and approaches necessary to meet
contemporary research challenges.

• Molecular Libraries and Imaging Probes
• Building Blocks, Biological Pathways and Networks
• Structural Biology
• Bioinformatics and Computational Biology
• Nanomedicine

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Molecular Libraries and Imaging

• FY04
• Molecular Libraries Small Molecule Repository
• Establishment of the NIH Chemical Genomics Center
• Development of High Resolution Probes for
  Cellular Imaging P20s
• High-Throughput Molecular Screening Assay
  Development R03s

• FY05 RFAs
• Molecular Libraries Screening Centers Network (6
  or more pilot centers)
• Molecular Libraries Screening Instrumentation
• Innovation in Molecular Imaging Probes
• Predictive ADME/Toxicology
• Exploratory Centers for Cheminformatics Research
• Pilot-Scale High-Throughput Screening
• New Methodologies for Natural Products Chemistry

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National Centers for Biomedical Computing

• 4 Centers funded in FY04
• Physics-based Simulation of Biological Structures
• National Alliance for Medical Image Computing
• Computational Biology Center
• Informatics for Integrating Biology and the
  Bedside
• Programs in preparation for partnerships between
  individual investigators and National Centers
• National Centers RFA re-announced for FY05
  (applications were due 1/24/05)

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Nanomedicine

• Nanomedicine, an offshoot of nanotechnology,
  refers to highly specific medical intervention at
  the molecular scale for curing disease or
  repairing damaged tissues, such as bone, muscle,
  or nerve

• 20 Nanomedicine Center Concept Development Awards
  funded in FY04
• 3-4 Nanomedicine Development Centers to be funded
  in FY05
• 2-4 Nanomedicine Development Centers to be funded
  in FY06

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Nanomedicine Concept Award Examples

• Probing functions of membranes with nanomaterials
  to better understand membrane-disrupting agents
• Understanding properties of materials that render
  them biocompatible
• Understanding how molecular motors drive mitosis
• Applying integrated scanning nanoprobe sensing
  systems

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Research Teams of the Future
Initiatives within this theme provide mechanisms
for interdisciplinary research, high-risk
strategies and public-private partnerships.

• Explore new organizational models for scientific
  teams
• Interdisciplinary research
• Larger, coordinated, resource-sharing
• Preserve the investigator(s)-initiated strategy

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Interdisciplinary Research Exploratory Centers

• Increasing complexity of questions requires
  multidisciplinary and interdisciplinary research
• Combining aspects of individual disciplines to
  provide new approaches to solving problems
• 21 P20s funded in FY2004

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Interdisciplinary Research Exploratory Centers
Examples

• Behavioral Epidemiology
• Integrate theoretical, methodological and
  analytic aspects of economic sciences, population
  sciences, and behavioral sciences to develop a
  new approach to discovery and intervention in
  youth vulnerable to HIV/AIDS
• Antimicrobial Resistance
• Integrate new disciplines to address the fast
  growing problem of antimicrobial resistance using
  novel new strategies developed by
  interdisciplinary research teams using long-term
  strategies

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Interdisciplinary Training Programs - Examples

• Clinical Research Experience for Engineers
• Undergraduate engineers engage in clinical
  research projects with clinical and
  bioengineering mentorship
• Regenerative Sciences Training Program
• Biologists, engineers, and clinicians augment
  training via didactic and research experiences
  with focus on musculoskeletal systems
• Clinical Biodetective Training
• Trainees to develop novel methods for detection
  of disease states, markers of good health, and
  therapeutic markers

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Removing Barriers to Interdisciplinary Research

• Lack of recognition of co-PIs
• How can NIH recognize the intellectual
  contributions of all investigators?
• Fiscal constraints have limited collaborations
  among institutions
• NIH grant application instructions have
  eliminated the fiscal disincentive to
  establishing consortiums.
• grants.nih.gov/grants/guide/notice-files/NOT-OD-04
  -040.html

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NIH Directors Pioneer Award

• Nine awardees in FY04
• High-risk, high-impact ideas
• Examples of investigation areas mathematical
  modeling of neurons and neural networks cellular
  reprogramming understanding and manipulating
  metabolic cycles new approaches to AIDS
  vaccines nanoscale studies of how cell surfaces
  interact with environment
• Annual Cycle

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Re-engineering the Clinical Research Enterprise
Initiatives within this theme address the need
for creating better integrated clinical research
networks, preparing the clinical research
workforce and developing technologies and
policies.

• Developing new research partnerships
• Patient communities
• Community-based health care providers
• Academic researchers
• Lower barriers to innovation
• Building better integrated clinical research
  networks
• Academic centers linked to community-based
  providers

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Standard Model
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The Way it Should Work
Laboratory Research
Population-based Clinical Research
Clinical Trials
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Translational Research Roadmap Initiatives

• Regional Translational Research Centers
• Fund planning grants in FY05
• New centers (10) starting in FY06
• Translational Research Core Services
• Modeled on NCI Rapid Access to Intervention
  Development (RAID) Program
• Pilot initiative planned for FY05

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Integration of Clinical Research Networks

• Link existing networks so clinical studies and
  trials can be conducted more effectively
• Ensure that patients, health care providers, and
  scientists form true Communities of Research
• FY 2006 NECTAR Network

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National Clinical Research Associates

• GOALS
• Form a national group of certified community
  healthcare providers
• Trained in clinical research
• Linked to regional academic centers
• Enroll and follow their own patients
• Partner with scientists, patient groups and
  industry as appropriate
• Access to NIH-NECTAR information system at
  practice site
• Accelerate translation of results into practice

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Multidisciplinary Clinical Research Career
Development

• Features
• Formal training in the discipline of clinical
  research
• Apprenticeship in mentored multi-disciplinary
  team setting
• Shared research support facility and project
  support
• Flexible salary support for scholars and mentors
• Coordinated national program
• Status
• 7 programs funded in FY04 new RFA for FY05

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Patient Reported Outcomes Measurement Information
System (PROMIS)

• Funded 7 grants in FY2004 that aim to improve
  assessment of patient-reported clinical outcomes
• Develop measurements of symptoms (pain and
  fatigue) and aspects of health-related quality of
  life with focus on chronic diseases
• Develop a publicly available system of a large
  item bank and Computerized Adaptive Tests

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Clinical Research Policy Analysis and Coordination

• Clinical research is impeded by multiple and
  variable requirements to address similar
  oversight concerns
• Variability exists among and within agencies
• Creates uncertainty about how to comply
• Hampers efficiency and effectiveness

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FY 2006 Roadmap

• New Pathways to Discovery
• Predictive ADME-Tox.
• HTS Assay Tech.Dev.
• Imaging Probe Dev. Center
• Standards and Critical Reagents for Proteomics
• Nanomedicine Dev. Centers

• Research Teams
• Innovation in Interdisciplinary Tech. Summit
• NIH Directors Pioneer Award
• Clinical Research
• Regional Translational Research Centers
• National Clinical Research Associates program
• NECTAR

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Interdisciplinary Research New Initiatives
FY 2004
FY 2006
Meetings and Networks for Methodological
Development in Interdisciplinary Research
Interdisciplinary Technology And Methods Summit
Supplements for Methodological Development in
Behavioral and Social Sciences
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Re-engineering the Clinical Research Enterprise
New Initiatives
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More Information

• http//nihroadmap.nih.gov

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NIH Roadmap for Medical Research

• IDEAS, PEOPLE, RESOURCES, LEADERSHIP