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Drug Discovery, Delivery and Pharmacogenomics

Broadly speaking, drug discovery is an industry characterized as one where the greatest opportunities exist when there is strong alignment between curiosity-driven activities and market-driven objectives.

There will be a growing impetus to bring an entrepreneurial ethic and greater manageability to the research mission at both university and corporate labs so as to reduce the 12-15 year cycle time and the roughly $800 million cost to bring new drugs to the market. Drug discovery and development includes the following areas:

target discovery and identification;
lead discovery and identification;
drug metabolism and toxicity;
drug process and development; and
application of genomics, proteomics, pharmacogenomics and bioinformatics in accelerating drug discovery and development.


Of particular value are technologies that facilitate drug delivery. Many of the newer, biotechnology-derived drugs face delivery challenges not seen in the prior generation of small-molecule synthetic pharmaceutical drugs. Novel delivery methods are required to effectively deliver therapeutic doses to their targets. These delivery systems can also benefit existing drugs, facilitating administration and improving safety and efficacy. Novel delivery methods also represent an inexpensive way to enter competitive markets. Recombinant insulin, for example, is established as the preferred treatment for diabetes. Biotechnology companies are pursuing gene therapy and aerosol delivery systems to improve on current shortcomings and gain market share without the expense of new drug development.

Pharmacogenomics examines the inherited variations in genes that dictate drug response and explores the ways these variations can be used to predict whether a patient will have a good, bad, or no response to a drug. In essence, pharmacogenomics is leading the drive for personalized medicine. Among the diagnostic tools used for pharmacogenomics are discrete genetic variations called SNPs. These variations can be used as a diagnostic tool for predicting a person’s drug response. DNA microarrays are an evolving technology that make it possible to identify SNPs quickly and are becoming increasing affordable. The implications for drug discovery and development are profound.

Pharmacogenomics makes it possible for pharmaceutical companies to exclude patients from clinical trials for whom testing indicates a drug would be harmful or ineffective. This increases the chance that a drug is shown useful to a particular population group, and the likelihood that the drug will make it into the marketplace. This would also decrease the cost and time of clinical trials. Finally, the doctor’s confidence in prescribing the drug, and the patient’s confidence in taking the drug increases, encouraging the development of other drugs in this manner and thereby stimulating more drug discovery activities. Genentech’s anti-breast cancer drug Herceptin is an example of a drug that is tied to a diagnostic test, which effectively identifies patients who are most likely to benefit from the drug.

 
 

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