Linking Drug Discovery to Successful Products

Stephen L. Eck MD, PhD

The current model of Translational Medicine, seeks to establish a concordance between laboratory findings and initial clinical findings.  While this has been a useful approach in developing targeted therapeutics with novel mechanisms of action, it is insufficient to meet the current needs in drug development.  New medicines must offer patients, physicians and payers exceptional value to justify their anticipated cost relative to the expanding pool of quality generic medicines.  Establishing the value of a new medicine will require attention to two areas previously unaddressed in Translational Medicine: The early identification of who might benefit from a new medicine (area of unmet medical need); and who will respond to the new medicine based on mechanism of action and variations in patient physiology.  In the first area, those engaged in translational research will need to partner with experts in health outcomes, and pharmacoeconomics to determine the performance characteristics of a new medicine that would justify the expense and risk of clinical development.  In the later area a significant effort will need to be expended, beginning very early in clinical development, to distinguish the responding population within the area of unmet need from the non-responding population.  In doing so, the value of a new medicine (e.g., response) will be not be diluted by the segment of the population that is molecularly in capable of responding to the drug. Candidate gene association studies embedded in early phase development provide the opportunity to differentiate responders from non-responders.  In some instances this will lead to the introduction of a companion diagnostic agent into clinical practice, as was the case with abacavir and maraviroc as well as several cancer therapies.  Several examples will be provided to illustrate the practical application of these principles.

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