Open Source Medicines

Cayman-officialThe process of drug development has a high rate of attrition. Only 20% of the compounds that enter human clinical trials reach final approval – and for each of these candidates, there are nearly one hundred substances already discarded by pharmaceutical developers based on preclinical animal and in vitro tests.  Authors continue to write articles on the R&D productivity decline and note that R&D costs are on the rise. So what happens to all of these failed drugs?

Almost any career medicinal chemist can describe in detail the memory of staring at the contents of a few refrigerator shelves containing perhaps fifty flasks and jars of compounds in various stages of purity and crystallinity. One remembers it because of the simultaneous recognition that this small collection represents tens of millions of dollars of investment in human time and energy. And also, that your superiors have told you to dispose of it, like so much moldy food.

Sadly, most clinical candidate drugs are first hoarded, then destroyed. It is the aggregate cost of these spectacular fails that so inflates the expense of those few drugs that actually make it to market.

A recent proposal by Francis Collins (Director of the NIH) to at least study these failed drugs is a step in the right direction.  It takes only a little more imagination, and one key regulatory change, to build this idea into something revolutionary.

There are dozens and dozens of terms and requirements built into the application process for an Investigational New Drug. Imagine if there were one more – that the applicant must send to the NIH a 100 gram sample of the pure drug candidate compound.* The Institute for Translational Medicine would then plate these experimental compounds out as compound libraries, and provide them to researchers worldwide. Think of it as the equivalent of open sourcing for software. Instead of being cloaked in secrecy, any new compounds being tested in humans anywhere would be simultaneously available for widespread screening for activity against diseases and conditions that the initial developers never even contemplated. This screening would also enable the discovery of side effects and other unanticipated actions, protecting public health and minimizing human exposures.

Big Pharma may be lukewarm to the idea of allowing the entire world access to their clinical candidates. But in order to receive permission to give experimental drugs to human beings, part of the price for that should be to allow all humans everywhere a chance to examine for themselves exactly what those compounds do. The benefits to mankind will be substantial.

*100 g for small molecules – 1 gram for biological such as antibodies and proteins.

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