610
expertise globally. caBIG will help redefi ne how research is conducted, care is pro-
vided and patients and participants interact with the biomedical research enterprise.
Participation in this network – based on universal standards for information security
and ethical use – means that all stakeholders must adhere to strict security measures
for accessing, utilizing and transmitting patient data.
In its funding agreements and its own internal research programs, the NIH is imple-
menting policies to facilitate the exchanges of these research tools and related resources
for personalized medicine. NIH’s Research Tools Policy defi nes research tools very
broadly, recognizing that the tools may serve as a product in addition to being a research
tool. These tools may include cell lines, model organisms, MAbs, reagents, growth
factors, databases and computer software. All of these have important uses in develop-
ment of personalized medicine. Future genomic advances would require a greater col-
laboration between the NIH, the universities and the industry. This is a new paradigm
in the pharmaceutical industry with relation to intellectual property (IP) similar to the
situation in case of SNP Consortium. If pharmacogenomic- based tests and associated
therapeutics are sold as a package, there may be an opportunity for IP sharing between
the upstream and downstream partners in drug discovery and development.
NIH Collaboration with the FDA
NIH works closely with the FDA for the development of personalized medicine. The
NIH will implement this strategy through such efforts as the Therapeutics for Rare
and Neglected Diseases (TRND) program. With an open environment, permitting the
involvement of all the world’s top experts on a given disease, the TRND program
will enable certain promising compounds to be taken through the preclinical devel-
opment phase – a time-consuming, high-risk phase that pharmaceutical fi rms call
“the valley of death.” Besides accelerating the development of drugs to treat rare and
neglected diseases, the TRND program may also help to identify molecularly distinct
subtypes of some common diseases, which may lead to new therapeutic possibilities,
either through the development of targeted drugs or the salvaging of abandoned or
failed drugs by identifying subgroups of patients likely to benefi t from them.
In 2010, the NIH and the FDA announced a new collaboration on regulatory and
translational science to accelerate the translation of research into medical products
and therapies; this effort includes a joint funding opportunity for regulatory science.
Working with academic experts, companies, doctors, patients, and the public, they
intend to help make personalized medicine a reality. An example of this collabora-
tion is an effort to identify new investigational agents to which certain tumors, iden-
tifi ed by their genetic signatures, are responsive (Hamburg and Collins 2010 ).
NIH and Genetic Testing Registry
NIH will address the fact that there is no single public source of comprehensive
information about the >2,000 genetic tests that are available through clinical labo-
ratories. In 2010, the NIH, with advice from the FDA and DHHS, started the Genetic
20 Development of Personalized Medicine