When Dr. Elias Zerhouni undertook his post as Director of the National Institutes of Health (NIH) in 2002, he introduced the concept of a “roadmap” for medical research that would identify
significant opportunities and challenges that should be addressed
by NIH as a whole—not just by individual Institutes or
Centers. Convening a series of meetings involving more than 300
nationally recognized leaders in industry, government, academia
and the public the work on creating the Roadmap commenced.
These meetings provided the framework for the NIH Roadmap
and thereby, the future of medical research.
The Roadmap’s initial vision and priorities were divided
into three broad themes, New Pathways to Discovery,
Research Teams of the Future and Re-engineering
the Clinical Research Enterprise, each with its own
set of initiatives. (See page 13 for a breakdown of the
themes and the initiatives.)
A good deal of progress has been made in the intervening
years, including the creation of the National
Technology Centers for Networks and Pathways,
the Molecular Libraries Screening Center Network
(MLSCN), the NIH Director’s Pioneer Awards and Clinical
Research Networks/NECTAR, among others.
The Roadmap initiatives have generated funding opportunities
for a variety of organizations, including small
businesses, individuals, not-for-profits and academic
institutions. Below are listed some relevant Roadmap
solicitations and notices, with more information and
funding sources to be found at: http://nihroadmap.nih.
Molecular Libraries Screening Center Network
Notice of Opportunity for Fast Track Entry of
Assay Development Projects into the Roadmap
Molecular Libraries Screening Center Network
Solicitation of Compounds for High Throughput
Screening (HTS) in the Molecular Libraries
Screening Centers Network (MLSCN)
NIH Director’s Pioneer Awards
NIH Director’s Pioneer Award Program (DP1)
Clinical and Translational Science Awards
Notice of Intent to Publish a Limited Solicitation
for Pilot Projects in Informatics for Clinical
and Translational Science Awards
One initiative of the Roadmap, found under the theme
of Research Teams of the Future, may be of special
interest to small businesses—the Public-Private Partnership
Program. While this program does not provide
funding, it will, among other tasks, serve as the point
of contact for entities wishing to partner with the NIH.
The program is to provide contact information, as well
as advice concerning the initiation, establishment and
implementation of new partnerships, complementing the ongoing effort of the Institutes and Centers in their
work with the private sector. For more information on
the Public-Private Partnership program, visit: http://nihroadmap.nih.gov/publicprivate/.
With the first wave of projects successfully underway,
the NIH spent the second half of 2006 soliciting the
scientific community, patient advocates and the public
at large, for ideas to form new initiatives that would
address challenges in biomedical research. Following
considerable scientific discussion, the decision was to
focus on four broad areas: Major Roadmap Initiatives,
Pilot Studies, Coordination Areas and Strategic Planning
Areas, with requests for applications (RFAs) relating
to these initiatives and studies to be released on a
variable timeline in late 2007 through mid-2008.
Initiatives and Studies
In May 2007, four specific topics were chosen by the
Institutes and Centers’ Directors to move forward as
Major Roadmap Initiatives. The first two, Microbiome
and Epigenetics Programs, are to be implemented as
five year programs. The remaining two programs, Protein
Capture Tools and Phenotyping Services/Tools, are
being implemented in stages, with second phase funding
contingent on outcomes of the first phase research.
Only one Pilot Program was chosen for support at the
May meeting—the Genetic Connectivity Map (CMAP).
(See topics with descriptions and potential funding opportunities
Coordination and Strategic Planning
As for the working groups attached to Coordination
Areas and Strategic Planning Areas, their activities are
still ongoing. The Coordination Area groups are assessing
current efforts in the areas of Regenerative Medicine,
Pharmacogenomics and Bioinformatics.
The Strategic Planning Area groups are focusing their
activities on topics in Training/Careers, Health Disparities
and Science of Science Administration. For more
information concerning the Coordination Areas and
Strategic Planning, visit: http://nihroadmap.nih.gov/grants/sbir-sttr.asp.
What does this mean for Small Business?
It is essential to note that the Roadmap is not set in
stone and that it will change as knowledge and technology
move forward. Creating sweeping change in an
agency as large as the NIH and in an area as critical as
human health cannot be done overnight, but can certainly
create unique opportunities for innovative small
businesses. The Roadmap is an effective tool for small
firms in the health care market, allowing them to follow
what areas/challenges the NIH considers crucial
to the future of health care. This can ultimately lead
to funding, either through NIH initiatives directly or
through contracting/partnering with other businesses.
Areas that are deemed important to NIH and hold promise
for the future will likely receive funding in one way
or another. A statement that, given the 8.2 percent increase in spending provided to the NIH by the 109th Congress
from FY2007 to FY2008, seems to hold true.
Major Roadmap Initiatives
Five Year Programs
Microbiome- The proposed Human Microbiome Project is focused
on characterizing the microbial content of sites in the
human body and then examining whether or not changes in
the microbiome can be related to disease.
Funding for the initial research for this project was $8.2 million
for FY2007. The funding was awarded to sequencing centers
at The Baylor College of Medicine, The Broad Institute, The J.
Craig Venter Institute and Washington University.
While there is no solicitation currently listed for this initiative,
NIH intends to offer one in FY2008, which will be found
Epigenetics- Epigenetics is the study of stable genetic modifications
that result in changes in gene expression and function
without a corresponding alteration in DNA sequence. These
epigenetic changes have been associated with diseases. A
catalog, the epigenome, was created, noting the epigenetic
modifications that are known to occur in the genome, but further
progress requires the development of better methods to
detect the modifications and a clearer understanding of factors
causing the changes.
Stage One Programs
As of this publication’s printing, there are no solicitations currently
listed for these initiatives.
Protein Capture Tools / Proteome Tools- This initiative would
support developing high-quality probes, specific to every protein
in the human body, as well as those in requested animal
models, and making them available to the scientific community.
The information would allow the characterization of protein
function in both health and in disease and would provide
a way to monitor the markers of a disease.
Phenotyping Services and Tools- The goal of programs in
this area would be the development of resources to catalog
human phenotypes, ultimately characterizing complex diseases
Genetic Connectivity Map (CMAP)- CMAP is an effort to discover
and demonstrate the linkages between diseases, drug
candidates and genetic manipulation.
This effort is being led by the Broad Institute with more information,
including data from the study and a web-based
tool for scientists to perform their own analyses, available
at their site www.broad.mit.edu/cmap/.
An article concerning the CMAP and its uses was recently
published in the Sept. 28, 2007 issue of Science magazine.
Stay in Touch and Informed
With so many areas to cover, it is important to network
and stay connected to potential funding sources at the
NIH. Each Institute and Center has a liaison for the Roadmap.
The relevant contact for each can be found on the
NIH Roadmap site, visit http://nihroadmap.nih.gov/ricl.asp.
The NIH also provides a tool to assist those seeking funding.
It is the electronic publication called the NIH Guide. The
Guide, published weekly, provides highlights of all NIH funding
opportunities and notices. To learn more and to register
to receive the publication, visit: http://grants.nih.gov/grants/
And though it would not meet the criteria of Phase III
commercialization funding, the SBIR/STTR program also
has Roadmap-relevant opportunities. These can be found
by visiting: http://nihroadmap.nih.gov/grants/sbir-sttr.asp.
Ultimately, the NIH Roadmap is creating opportunities for
small businesses and the future of medical research certainly
looks bright, when looking in the right directions. ♦
Main Areas of Focus for the NIH Roadmap
New Pathways to Discovery
This area of the Roadmap addresses the need
to advance understanding of complex biological
systems and the accessibility of technologies,
databases and resources. With the completion
of the human genome sequence and recent
discoveries in molecular/cell biology a better
medical research “toolbox” is in the works.
Building Blocks, Biological Pathways and Networks
Molecular Libraries and Molecular Imaging
Bioinformatics and Computational Biology
Research Teams of the Future
This area of the Roadmap encourages scientists
of varying disciplines to collaborate and expand
their approach to the scientific enterprise.
This should stimulate new ways of combining
skills and disciplines, in both the physical and
biological sciences. The intent is to accelerate
scientific findings from bench to bedside.
Re-Engineering the Clinical Research Enterprise
The NIH is focused on the transition of basic research
discoveries into drugs, treatments and
methods for prevention and the simplification of
the regulatory process. NIH intends to promote
better integration of existing clinical research networks,
improve the training of clinical researchers
and encourage the development of technologies
to advance the assessment of clinical outcomes.
Clinical Research Networks/NECTAR
Clinical Research Policy Analysis and Coordination
Clinical Research Workforce Training
Dynamic Assessment of Patient-reported
Chronic Disease Outcomes