The NIH Roadmap

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The NIH Roadmap
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Imperatives for NIH

• Accelerate pace of discoveries in life sciences
• More rapid translation from laboratories to
  patients and back
• Develop novel approaches orders of magnitude more
  effective than current ones
• Develop new strategies NIH Roadmap

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Roadmap Participants were asked

• 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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The Problem
Bench
Bedside
Practice
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NIH Roadmap three themes emerged

• New Pathways to Discovery
• Research Teams of the Future
• Re-engineering the Clinical Research Enterprise

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Criteria for Roadmap Initiatives

• Is it transforming -- will it change how or
  what biomedical research is conducted in the next
  decades?
• Would its outcome enhance the ability of all ICs
  to achieve their missions?
• Can the NIH afford to NOT to do it?
• Will it be compelling to our stakeholders,
  especially the public?
• Is it something that no other entity can or will
  do?

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New Pathways to Discovery
Bench
Bedside
Practice

• Building Blocks
• and Pathways
• Molecular Libraries
• Bioinformatics
• Computational
• Biology
• Nanomedicine

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Biological Pathways and Networks
Understand in quantitative terms how gene
regulatory networks, signal transduction
pathways, and metabolic pathways are integrated
to orchestrate normal development Understand
responses to internal and external stimuli in
highly complex organisms Understand how pathways
and networks are perturbed in disease.
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Bioinformatics and Computational Biology
Deploy a rigorous biomedical computing
environment to analyze, model, understand and
predict dynamic and complex biomedical systems.
Be able to integrate data and knowledge at all
levels of organization.
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Molecular Libraries

• Chemical Diversity
• -Publicly available database
• -Biologically relevant chemical space optimally
  populated with naturally occurring and
  synthetic compounds
• Screening
• -Small molecule activators and inactivators
  available to researchers for individual
  functions of every gene product in human
  genome
• Drug development as needed

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Research Teams of the Future
Interdisciplinary Research Teams Pioneer Award
Public-Private Partnerships
Bench
Bedside
Practice
Building Blocks and Pathways Molecular
Libraries Bioinformatics Computational
Biology Nanomedicine
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Challenges to Interdisciplinary Research

• The current system of academic advancement favors
  the independent investigator
• Most institutions house scientists in discrete
  departments
• Interdisciplinary research teams take time to
  assemble and require unique resources

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Interdisciplinary Curriculum Development Awards

• Support the development of innovative courses and
  curricula designed to train interdisciplinary
  scientists
• Curricula can be designed for undergraduate,
  pre-doctoral or postdoctoral students, or
  combinations of these.
• Focus on programs that encourage the integration
  of quantitative, physical, behavioral, or social
  sciences with the traditional biomedical sciences

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NIH Directors Pioneer Award

• New program to support individuals with untested
  ideas that are potentially groundbreaking
• Encourages innovation, risk-taking
• Totally new application and peer review process
• Provides 500 K/year for 5 years
• Expected to be highly competitive

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Re-engineering Clinical Research
Interdisciplinary Research Pioneer Award
Public-Private Partnerships
Bench
Bedside
Practice
Building Blocks and Pathways Molecular
Libraries Bioinformatics Computational
Biology Nanomedicine
Integrated Research Networks Clinical Research
Informatics NIH Clinical Research
Associates Clinical outcomes Clinical Research
Policy Training
Translational Research Initiatives
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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, physicians, and scientists
  form true Communities of Research

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Translational Research Centers

• Provide sophisticated advice help scientists
  bring a new product from the bench to clinical
  use, including
• Laboratory studies to understand a therapys
  mechanisms of action
• Pre-clinical drug synthesis, toxicity testing
• Sophisticated manufacturing capacity
• Expert advice to ensure that drug-development
  regulations are observed

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Patient Reported Disease Outcomes

• Establish a collaborative of investigators to
  improve measurement of patient-reported outcomes.
  
• Focus on the collection of self-report data from
  a diverse population of individuals, including
  racial and ethnic minorities, having a variety of
  chronic diseases.
• Compare the performance of specific items,
  instruments, and models across diverse clinical
  populations
• Develop common metrics by which scores on new and
  existing instruments can be standardized and/or
  linked

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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

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National Clinical Research Associates
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Multidisciplinary K12 Career Development Program
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Current Polices, Procedures, Regulations Major
Traffic Jam

• The clinical research community faces redundant
  and sometimes variable requirements to address
  fundamentally the same oversight concerns (e.g.,
  AERs, COIs)
• Variability exists not only among various
  Federal agencies, but also within the NIH and it
  hampers efficiency and effectiveness

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Toward a Solution NIH Role

• NIH promotes the responsible conduct of high
  quality clinical research
• Opportunity to convene other federal agencies and
  to facilitate dialogue and change

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Roadmap Clinical Research Policy Coordination
Initiative

• Improve coordination and simplify requirements
  for clinical research in ways that enhance public
  trust
• Adverse event reporting
• Human subjects protections, including DSMB-IRB
  interactions and consent procedures
• Auditing and monitoring clinical trials
• HIPAA, privacy, conflict of interest policies
• Investigator registration, financial disclosure
• Standards for electronic data submission/reporting

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Policy Coordination Implementation Plan

• Need for sustained attention
• Identify and prioritize issues
• Maintain open communication with all stakeholders
  investigators, patients, families,
  institutions, communities, policy-makers
• Work collaboratively with sibling Agencies
• Develop tools for investigators to facilitate
  compliance
• Education and outreach

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Roadmap Implementation Groups

• Molecular Libraries and Imaging
• Building Blocks, Biological Pathways and Networks
• Structural Biology
• Bioinformatics and Computational Biology
• Nanomedicine
• Interdisciplinary Research
• High-risk Research
• Public-Private Partnerships
• Re-engineering the Clinical Research Enterprise

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Roadmap Implementation

• All Institutes and Centers committed to invest
  jointly in a pool of resources to support current
  and future Roadmap initiatives
• 128 M in FY 2004 (DDF funds and 0.34 each ICs
  budget)
• Over 2 B by FY 2009

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Consultation

• Participation, consultation, collaboration, and
  funding are needed from patients, health care
  providers, foundations, industry, academia,
  Federal partners all stakeholders
• www.nihroadmap.nih.gov

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The NIH RoadmapA Work in Progress