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''There's Gold in Them Thar Hills'' Govind Rao

PDA J Pharm Sci and Tech 2014, 68 102 Access the most recent version at doi:10.5731/pdajpst.2014.00982

Downloaded from journal.pda.org on February 20, 2015

EDITORIAL

“There’s Gold in Them Thar Hills” Dahlonega, Georgia was the site of the first gold rush in the US. The quote above is attributed to its mayor, when he was trying to persuade miners to hang around and not leave for new discoveries reported in California. Where am I going with this? None of us would mind striking “gold” in our line of work—that is, finding the next blockbuster drug. Let us consider the parallels with looking for gold. Prospectors look in “them thar” sites where gold is likely to be located and start taking samples. Modern tools allow for precise geolocation. With sophisticated tools for analysis, estimates for the amount of gold and the economics of recovery are assessed prior to deciding if mining is worth it. The key aspect is sampling— every site is individually assessed. In contrast, the drug discovery approach takes the “sample”, in this case the test subject’s response to the investigational new drug, and uses a variety of statistical approaches to see if the effect is real or not. In effect, it is analogous to a mining company grinding up all its samples together from a prospective site and

doi: 10.5731/pdajpst.2014.00982

102

coming up with an average gold composition. Does this approach for assessing clinical significance make sense? Many drugs that have performed well end up failing efficacy end points in Phase III trials. Are we losing many potential useful drugs due to our clinical trial process, which subjects the data to the tyranny of the mean? Or should we start using the power of computing to focus more on each individual’s unique response to the drug candidate? And if this means that you have to deal with an n ⫽ 1, then what new analytical paradigms do we need? Computational technology now exists to sift through big datasets and find the outliers and unexpected metadata correlations. Can we use a similar approach in clinical “prospecting”? Could we go back through old clinical trial files and see what we may have missed with the old approach? Could such meta-analysis even provide information on other disease conditions that existing drugs can target? Lots of questions to ponder! Govind Rao UMBC, 1000 Hilltop Circle, Baltimore, MD 21250

PDA Journal of Pharmaceutical Science and Technology

Downloaded from journal.pda.org on February 20, 2015

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"There's gold in them thar hills".

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