In the past decade, many of the biological applications of genetic and genomic information to the problems of medicine development and delivery were not possible. These applications required the tools provided by the Human Genome Project. The next decade will see three new applications of genetics to medicine: (i) identification of disease-associated genes in target classes that are amenable to high-throughput drug screening; (ii) inexpensive high-throughput genotyping technologies and new statistical algorithms for rapidly defining multiple gene variants associated with a defined phenotype; and (iii) a pharmacogenetic system approved by regulatory authorities that facilitates delivery of safe and effective medicines. There are no reviews discussing these topics in this issue.
Time is a key variable that is frequently misunderstood. Identification of disease-specific drug targets is now possible, but the wave of new and approved medicines resulting from these findings will take years to pass through the drug discovery and regulatory processes. Expensive methods for whole-genome genotyping already exist in 2003. However, the collection of human DNA samples and the critical definition of clinical phenotypes that is necessary for high-quality association analyses have yet to be appreciated (and supported properly) as the rate-limiting steps in phenotype–genotype associations. A systematic method for regulators and industry to deal with adverse events is needed to maintain availability of effective medicines. This is particularly the case when dealing with the uncommon complications recognized only when a drug becomes available to large numbers of patients. Using current tools, it is possible to develop inexpensive tests that can predict an individual's susceptibility to an adverse event. Application and availability depend on a societal commitment to safety and new surveillance systems.
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