A toe in the water

toes by Ingorrr
The NIH Takes the Plunge:
[Via In the Pipeline]

The NIH has announced that they’re going to start up a preclinical drug discovery effort to address rare diseases. I find this interesting for several reasons. For one thing, it’s worth a try for conditions where no company has seen…

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Very interesting. The NIH doing development is somewhat novel and, for them to do it well, will require a different sort of researcher than normally found in academia. This is because most universities are organized around departments dealing with a specific discipline (such as chemistry, genomics, etc.) but diseases often require expertise across disciplines. As the first comment suggests, this is not something that most universities will be good at.

However, a lot of non-profit research institutions are organized around a disease, not a discipline. Their structure is more amenable to these sorts of approaches. Not that everyone sits down and sings Kumbaya. We are talking about people with large egos but the opportunity for collaboration is much more a part of the institution and its mission.

As someone from industry who has dealt with this issues, what I find more intriguing about this announcement is not that they are doing development but that the focus will be on rare and neglected diseases. This is a niche where few large drug companies venture simply because the economics are not a good fit. Any therapeutic for these diseases will not bring in a lot of money. If it was likely to do so, these would not be neglected diseases. Researchers would have gone hard after them a while ago.

These diseases are unlikely to make a lot of money and thus recoup the costs of development. And many of them affect large numbers of poor people throughout the world. The Institute of Medicine just came out with a report discussing the need for the US to take even a bigger lead in attacking these neglected diseases. Perhaps this is one way.

Corporations have to make money. That is why they exist. Neglected and rare diseases are a niche where research organizations that are not focussed on a profit could make substantial progress if motivated. Having new ways to access capital to perform this work would be vital. I’ve discussed one, the L3C, in my article at Xconomy. This approach from the NIH might be another, if done well.

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