# Presentation: 789833

## The NIH Roadmap

## Acute to chronic conditions

-  **Evolving Public Health Challenges**

**Health Disparities**

**Emerging Diseases**

**Aging Population**

**Biodefense**

##  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?**

## The Problem

**Bedside**

**Practice**

**The Problem**

**Notes:**
System Broken

## 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?

## New Pathways to Discovery

**Bedside**

**Practice**

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

**New Pathways to Discovery**

## 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.

## The Biological Data of the Future

- Destructive
- Qualitative
- Uni-dimensional
- Low temporal resolution
- Low data  density
- Variable standards
- Non-cumulative

- Non-destructive
- Quantitative
- Multi-dimensional and spatially resolved
- High temporal resolution
- High data density
- Stricter standards
- Cumulative

## 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 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**** **

## Structural Biology:Life in Three Dimensions

- Focus on trans-membrane proteins

## Research Teams of the Future

**Bedside**

**Practice**

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

**Interdisciplinary **
**Research Teams;**
**Innovator Award**** **

**Public-Private**
**Partnerships**

**Research Teams of the Future**

## The Scientist of the Future

- Need to examine role and status of postdoctoral fellowships
- Need for multi-disciplinary and inter-disciplinary teams
- Need for larger, coordinated, resource sharing teams
- Need for new career pathways
- New approaches to training
- Need to examine the balance between Projects vs. People
- Need for pioneering high-risk research
- Need to preserve the investigator-initiated strategy

## 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

**Notes:**
a single PI is recognized; indirect costs from collaborating institutions become embedded in direct costs of  the “lead” institution indirect costs follow the PI.

## Interdisciplinary Recombination

**Computer and Information Sciences**

**Biological **
**Sciences**

**Physical****Sciences**

**Behavioral Sciences**

## Interdisciplinary Research Centers

**Interdisciplinary Research Centers**

- Lowers barriers that slow interdisciplinary studies
- Allows the interdisciplinary team to evolve

## NIH Director’s Innovator 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

## Re-engineering Clinical Research

**Bedside**

**Practice**

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

- Translational
- Research
- Initiatives

- Integrated Research Networks
- Clinical Research Informatics
- NIH Clinical Research Associates
- Clinical outcomes
- Harmonization
- Training

- Interdisciplinary
- Research
- Innovator Award

- Public-Private Partnerships

**Re-engineering Clinical Research**

## Integrated Network of NetworksTypical NIH Network

## Need for Interoperable Networks

## 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

**Clinical Research evolved haphazardly**
- Started as cottage industry and select centers
- Now has more complex requirements: regulation, technology, speed, efficiency
- Need for greater links to basic science
**Need transformation to move into the 21****st**** Century**
- Apprenticeship  discipline of clinical research
- Uniformity  harmonize rules, build infrastructure and create networks
- Mentoring  multidisciplinary teams
**Need for access to well characterized cohorts of patients and biological samples**

## Translational Research

- Translational Research Centers
-  Pre-clinical drug synthesis, toxicity 	testing
-  Enabling technologies for improved  	assessment of clinical outcomes

## 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

## Multidisciplinary Clinical Research Team

- Clinical
- Research
- Team

- Clinician

- Study Coordinator

- Statistician

- Tech Transfer;
- Regulatory Affairs

- Pharmacologist;
- Medicinal Chemist

- Public;
- Industry

- How to make each path viable?

- Principal Investigator

## 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
 |
|  |  |  |
|  |  |  |

## Trans-NIH Multidisciplinary K12 Career Development Program (RFA)

| Goal: |  |  Promote development of investigators from a      variety of disciplines (MD, PhD, RN, MPH, DC ...)
 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
 |
|  |  |  |
|  |  |  |

## Harmonization of Clinical  Research Regulatory Processes       

**Goal:**	Harmonize and simplify requirements for clinical 	research in ways that enhance public trust
- Adverse event reporting
- Human subjects protection
- DSMB-IRB interactions
- Consent procedures
- Auditing and monitoring clinical trials
- HIPAA, privacy, conflict of interest policies
- Investigator registration, financial disclosure
- Standards for electronic data submission/reporting

## 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
** **

## Common Pool Roadmap Fundingdollars in millions

|  | FY04 | FY05 | FY06 | FY07 | FY08 | FY09 | Total |
| --- | --- | --- | --- | --- | --- | --- | --- |
| Pathways to 
Discovery | 64 | 137 | 169 | 182 | 209 | 188 | 948
 |
| Research
Teams | 27 | 39 | 44 | 92 | 96 | 93 | 390 |
| Clinical
Research | 38 | 61 | 120 | 174 | 214 | 227 | 833 |
| Total | 128 | 237 | 332 | 448 | 520 | 507 | 2,172 |

## Current RFAs (as of December 3, 2003)

- _Supplements for Methodological Innovations in the Behavioral and Social Sciences_
- _[Dynamic Assessment of Patient-Reported Chronic Disease Outcomes](http://grants.nih.gov/grants/guide/rfa-files/RFA-RM-04-011.html)_
- _[Interdisciplinary Health Research Training: Behavior, Environment and Biology](http://grants.nih.gov/grants/guide/rfa-files/RFA-RM-04-010.html)_
- Short Programs for Interdisciplinary Research Training
- Curriculum Development Award in Interdisciplinary Research
- Centers for Innovation in Membrane Protein Production
- Multidisciplinary Clinical Research Career Development Programs
- Development of High Resolution Probes for Cellular Imaging
- Metabolomics Technology Development
- Exploratory Centers (P20) for Interdisciplinary Research
- National Technology Centers for Networks and Pathways
- National Centers for Biomedical Computing

## Consultation

- Participation, 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

## NIH

**Ideas**
**People**
**Resources**

**NIH**