ELEMENT patient or donor blood and other tissue samples


As many drugs needs to be developed, NCTR
(national center for toxilogical research) has reorganized that works as a cross
functional teams on NCTR research projects. The reorganization formed three new
branches within the Division of systems biology and has better positioned NCTR
to support the larger personalized medicine efforts of the agency.

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The three new branches the division of
systems biology are:

(1) Biomarkers and alternative methods

(2) Innovative safety and technologies

(3) Personalized medicine


The program aim is to provide a framework
for scientic development and regulatory acceptance of biomarkers for use in
drug development and and integrating the qualified biomarkers in this
regulatory review process and encourage for the new identification and emerging

CDER developed the process for qualifying
biomarkers for use in drug development. Once it has been qualified, a biomarker
can be used by drug developers in the context of use in investigations and
marketing submissions without requesting the relevant agency.


Microarrays and next generation sequencing
represent core technologies in pharmacogenomics, toxic genomics, and
personalized medicine. Implemented, organized and run by NCTR scientists, the
FDA-led MAQC/SEQC project seeks to advance translational and regulatory
sciences by assessing technical performance and practical utility of emerging
molecular technologies for clinical application and safety evaluation. This
collaborative effort wil help to ensure that the field of personalized medicine
will benfit from high quality diagnostic tests.


Human leukocyte antigen system is a large
number of genes and protein products that are related to immune system function.
It is a process of testing the patient or donor blood and other tissue samples
for the HLA antigens, and to determine the compatibility between the HLA

Molecular –based typing methods are used
to improve transplant outcomes for the precise HLA matching and especially
critical for bone marrow transplant, where the poor matches result in
catastrophic health consequences. For the ambiguous results,” gold standard”
DNA-based HLA typing methods, and also due to the variability and complexity of
the HLA genes. CBER scientists, along with others by using a high-resolution
HLA typing methods to achieve the results without ambiguities.


CDRH have made major advances in the
underlying of heart disease and used new methods for them using the analysis of
the electrocardiogram to predict which patient benefit from the cardiac
resynchronization therapy. This diagnose the electrical conduction problems and
to quantify the scar tissue in the heart, but the women benefit it more than
the men due to the certain criteria. This explains why the efficacy and safety
of medical products differs in patients and can be used to design more
efficient clinical trials.


It is a clinical trial to select the
patients for whom therapy is most likely to provide a benefit. FDA is working
to address the issues like missing data’s, multiple endpoints, patient enrichment,
and adaptive designs due to the development of targeted therapeutics. FDA is
most probably used for clinical trials for oncology drug development. Generally,
the cancer is heterogeneous in which each with their own specific genetic
makeup and thus respond differently to therapies with different people. The
I-SPY 2 trial, a highly collaborative initiative developed under a unique
public-private partnership and involving the participation of more than 20
centers are recruiting and treating the patient.


In the early stage, multiple drugs were
removed because of the increased fatal abnormal heart rhythm called “torsade de
poites”. This increases a measurement on the electrocardiogram called the “QT
interval”. However not all drugs cause torsade de pointes. Some effective drugs
are prevented in the market. Thus, CDER and CDHR is assessing new device based
algorithms and biomarkers that distinguishes benign (not harmful) from
malignant(harmful)drug-induced QT prolongation.

Recombinant protein therapeutics, which
are the fastest growing segment in the pharmaceutical industry are mainly used
for the complex medical conditions.

At 2010, 38% of drugs are approved for
efficacy, safety and pharmacokinetics.

At 2011, personalized medicines have been
increased to 12, which is 51%.

In 2013, it has been decreased from 117%
to 67% due to the design of more drugs to the small populations. Such
approaches can dramatically shorten overall drug development and review times.

most significant challenge in personalized medicine:

Limited understanding of
the intrinsic biology of diseases.

Common conditions
involving multiple gene.

An outdated disease
classification system.

Lack of infrastructure.

Investment uncertainties.

Access to personalized



The FDA is committed to
work improving the personalized medicine by advancing the science and tools
that will provide clarity and guidance to industry in order to provide more
drugs to the market.