How to leverage the NCATs template across the industry.

NIH Director Francis Collins has made a centerpiece of his tenure the National Center for Advancing Translational Science (NCATs), an effort to "repurpose" compounds under patent by drug companies that have passed early stage clinical trials, and thus have at least a baselline record of safety in humans. The purpose of the initiative to to look for additional uses for the drugs, and then launch them through final confirmatory (Phase III) trials if they look like promising treatments for other diseases.

Here's some detail from the NCATs mission page:

The Center strives to develop innovations to reduce, remove or bypass costly and time-consuming bottlenecks in the translational research pipeline in an effort to speed the delivery of new drugs, diagnostics and medical devices to patients.

Good luck, Godspeed, and amen. Overcoming bottlenecks and advancing translational research tools that can improve the productivity of the drug development pipeline are sorely needed. And NIH is arguably best positioned to drive this effort forward, since it works with all the key players involved, and has a much bigger budget than the FDA - and the FDA is also plagued by conflict of interest concerns from Congress about its nascent role in facilitating new drug development.

Today, the NIH released more details about its efforts, and announced new partners:

The National Institutes of Health (NIH) today unveiled the details of its $20 million program for finding new uses for abandoned drugs--along with five more participating companies. The program's expansion brings to 58 the number of shelved compounds that academic researchers can test for new uses.

Discovering New Therapeutic Uses for Existing Molecules, announced in early May, is the first major initiative from NIH's new National Center for Advancing Translational Science (NCATS). The idea is to give academic researchers access to compounds that made it through safety testing but were dropped by companies for business reasons or because they didn't work on a specific disease. Initially, three companies--Pfizer, AstraZeneca, and Eli Lilly--offered to share 24 compounds.

Now Abbott Laboratories, Bristol-Myers Squibb, GlaxoSmithKline, Sanofi, and Janssen Pharmaceuticals have signed on, bringing the number of compounds to 58. NIH has posted a table of the compounds that links to one-page fact sheets about the drugs that include the mechanism of action and summary clinical results. NIH is also taking preapplications (due 14 August) for the program's 2- to 3-year grants.

Again, I hope that this work succeeds. But I'm also curious: What tools is the NIH using, or proposing to use, to identify new targets for old compounds that industry doesn't have already? Assuming that new targets are identified, how will the NIH address remaining safety and efficacy concerns - after all, companies will still have to run Phase II and Phase III trials for FDA approval, with an awful lot of unknowns still unknown about how the drugs will perform in the clinic. These concerns are all tractable, and the sooner they become so the better off we'll all be.

But here's the final problem: patents. Assuming that these drugs have already been through significant preclinical and early stage clinical testing, how much time is still left on the patent clock to take them through to FDA approval?

I'm betting that NIH's industry partners carefully chose products with significant patent time left on the clock, but it's still a big problem if your intent is to re-use not just a few dozen, but the hundreds of promising compounds out there with little or no patent time left that have never advanced far into clinical testing.

Beyond the NIH, Congress could give a big jolt to repurposing old compounds with basic safety data by offering market exclusivity for any drug (not FDA approved for another indication) that didn't have enough patent time left to justify commercial development. The MODDERN Cures legislation developed by the National Health Council would be a significant help in this effort.

It would lead many more small and large companies alike diving into their compound libraries looking for therapies to rescue, and incentize additional investments in new diagnostics to spur the process along. This would be a synergistic approach that would leverage the NCATs approach across the entire industry - and lead to many more potential successes.

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