ABSTRACT
In.the.past.decade,.the.cost.of.drug.discovery.and.development.has.risen.exponentially,.but.the.rate.of.introduction.of.new.drugs.has.actually.decreased..The.cost.of. developing.a.new.chemical.entity.(NCE).currently.ranges.from.$800.million.to.$1.1. billion.[1,.2]..These.new.economic.fundamentals.of.the.industry.are.not.sustainable,. and.the.problem.is.being.addressed.by.pharmaceutical.companies.[2]..Application. of. novel. genomics. technologies. has. offered. significant. promise. in. improving. the. efficiency.of. the.drug.discovery.and.development.process.. In. the.years. following. the.publication.of.the.human.genome.sequence,.genomics.technologies.have.revolutionized.drug.discovery.and.development..In.contrast.to.the.traditional.trial-anderror.approach,.an.innovative,.hypothesis-driven,.and.systematic.strategy.has.been. adopted..This.strategy.is.based.on.an.initial.target.selection.and.validation.followed. by.optimization.and.selection.of.compounds.that.would.modulate.the.activity.of.the. target.with.minimal.side.effects..Today’s.drug.development.process.is.characterized. by. a.very.high. failure. rate.of. experimental. compounds,. particularly. at. the. costly. late.stages.of.development.(Phases.IIb.and.III).[3]..In.particular,.the.major.causes.of. attrition.in.the.clinic.are.currently.lack.of.efficacy.and.safety,.both.accounting.for. approximately.30%.of.failures.[3]..Consequently,.approaches.using.genomics.technologies.and.designed.to.evaluate.or.predict.toxicity.and.efficacy.earlier.represent.a. solution.to.control.the.failure.rate.in.clinical.development.
