The NIH Roadmap

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

Roadmap Participants were asked:

What are today’s scientific challenges?

What are the roadblocks to progress?

What do we need to do to overcome roadblocks?

What can’t be accomplished by any single Institute – but is the responsibility of NIH as a whole?

Bench Bedside Practice

The Problem

NIH Roadmap: three themes emerged New Pathways to Discovery

Research Teams of the Future

Re-engineering the Clinical Research Enterprise

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?

Bench Bedside Practice

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

New Pathways to Discovery

Biological Pathways and NetworksUnderstand 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.

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.

Molecular LibrariesChemical 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

Bench Bedside Practice

Building Blocks and PathwaysMolecular LibrariesBioinformaticsComputational BiologyNanomedicine

Interdisciplinary Research Teams;Pioneer Award

Public-PrivatePartnerships

Research Teams of the Future

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

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

NIH Director’s 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

Bench Bedside Practice

Building Blocks and PathwaysMolecular LibrariesBioinformaticsComputational BiologyNanomedicine

TranslationalResearchInitiatives

Integrated Research NetworksClinical Research Informatics NIH Clinical Research AssociatesClinical outcomesClinical Research PolicyTraining

Interdisciplinary Research

Pioneer Award Public-Private Partnerships

Re-engineering Clinical Research

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”

Translational Research Centers Provide sophisticated advice help scientists bring

a new product from the bench to clinical use, including:

Laboratory studies to understand a therapy’s mechanisms of actionPre-clinical drug synthesis, toxicity testingSophisticated manufacturing capacityExpert advice to ensure that drug-development

regulations are observed

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

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

National Clinical Research Associates

Goal: Create a diverse national group of trained and certified community health care providers Providers will enroll and follow their own patients Means to accelerate translation of results into practice

Steps: Determine feasibility: Barriers? Communities? Incentives needed? Inventory training methods, best practices Develop core competencies, certification Future: training programs

Multidisciplinary K12 Career Development Program

Goal: Promote development of investigators from a variety of disciplines (MD, DDS,PhD, RN, MPH..) To be trained in multidisciplinary team settings

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

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

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

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

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

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

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

ConsultationParticipation, consultation, collaboration, and funding

are needed from patients, health care providers, foundations, industry, academia, Federal partners

…all stakeholders

www.nihroadmap.nih.gov

The NIH Roadmap:A Work in Progress