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?

The Problem Bench Bedside Practice 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 Bench Bedside Practice Building Blocks and Pathways Molecular Libraries Bioinformatics Computational Biology Nanomedicine

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.

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

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

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 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 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

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

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 action Pre-clinical drug synthesis, toxicity testing Sophisticated manufacturing capacity Expert 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

Consultation www.nihroadmap.nih.gov 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