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1
Introduction1
The number of new drug approvals has remained reasonably steady for
the past 50 years at around 20 to 30 per year (Munos, 2009), while at the
same time the total spending on health-related research and development
has tripled since 1990 (Scannell et al., 2012). There are many suspected
causes for this trend, including increased regulatory barriers, the rising
costs of scientific inquiry, a decrease in research and development efficiency,
the downstream effects of patent expirations on investment, and the lack
of production models that have successfully incorporated new technology
(Paul et al., 2010; Scannell et al., 2012). Regardless, this trajectory is not
economically sustainable for the businesses involved, and, in response,
many companies are turning toward collaborative models of drug develop-
ment, whether with other industrial firms, academia, or government (IOM,
2011). Introducing greater efficiency and knowledge into these new models
and aligning incentives among participants may help to reverse the trends
highlighted above, while producing more effective drugs in the process.
New technologies have the potential to open up avenues of develop-
ment and to identify new drug targets to pursue. Specifically, improved
validation of genedisease associations through genomics research has the
potential to revolutionize drug production and lower development costs.
Genetic information has helped developers by increasing their understand-
1 The planning committee's role was limited to planning the workshop, and the workshop
summary has been prepared by the workshop rapporteurs as a factual summary of what
occurred at the workshop. Statements and opinions are those of individual presenters and
participants and should not be construed as reflecting any group consensus.
1
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2 GENOME-BASED THERAPEUTICS
BOX 1-1
Workshop Objectives
The workshop New Paradigms in Drug Discovery: How Genomic Data Are
Being Used to Revolutionize the Drug Discovery and Development Process had
three broad objectives:
1.To examine the impact of and investment in genetic and genomic technolo-
gies and data in drug discovery and development.
2.To examine and discuss the challenges for incorporating genomic tech-
nologies into drug development and to explore solutions to remedy those
challenges.
3.To discuss and explore how innovative, novel, and global partnerships
between academia, industry, foundations, and government can enable the
use of genomic information for more efficient and effective drug discovery
and development programs.
ing of the mechanisms of disease as well as individual patients' reactions
to their medications. Warfarin, Gleevec®, XALKORI®, KalydecoTM, and
Zelboraf® are all examples of pharmaceuticals that utilize genetic informa-
tion to inform dosing or whose activity and effectiveness is determined by
inherent genetic properties of the patient or their tumor (i.e., a targeted
therapeutic). However, even with these successes there remains skepticism
over how useful genomic information will be to the larger drug develop-
ment process (Pollack, 2010; Wade, 2010). There is a need to identify the
success factors for the various models that are being developed, whether
they are industry-led, academia-led, or collaborations between the two.
The Roundtable on Translating Genomic-Based Research for Health
held a workshop on March 21, 2012, titled New Paradigms in Drug Dis-
covery: How Genomic Data Are Being Used to Revolutionize the Drug
Discovery and Development Process. The purpose of the workshop was
to examine the general approaches being used to apply genomic-based
research results to the discovery and development of new drugs, the suc-
cesses achieved so far, and the challenges ahead.2
STRUCTURE OF THE REPORT
Box 1-1 provides the overall objectives of the workshop. Chapter 2
discusses the current environment for personalized medicine approaches to
2 The full statement of task can be found in Appendix C.
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INTRODUCTION 3
drug discovery and development. Chapter 3 examines three case studies as
illustrations of both the promise and the challenges of genomic-based drug
discovery and development. Chapter 4 looks at the application of emerging
technologies, such as next-generation sequencing, to this field. Chapter 5
examines several specific evolving paradigms in genomic-based drug devel-
opment, approval, and prescribing. Finally, Chapter 6 features comments
and discussions drawn from throughout the workshop on overcoming the
challenges and achieving the promise of genomic-based drug discovery and
development.
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