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 a way to fund research, and there are quite a few of them. Treating rare diseases can be quite profitable in the industrialized world (ask Genzyme, among other companies), but if the conditions are localized in poorer areas no one’s likely to take a crack at them. So my first reaction is “Good, and the best of luck to you”. The NIH has been getting closer to doing preclinical drug discovery in recent years, so this is a logical next step.
The second thought I have is that this will be an interesting experience for the researchers involved. There’s nothing quite like drug discovery, and if they do it right, everyone will come away with an appreciation of just how complicated a process it is. The only way to make it simple and reasonable is to cut corners. I notice that the press release says:
Typically, drug development begins when academic researchers studying the underlying cause of a disease discover a new molecular target or a chemical that may have a therapeutic effect. Too often, the process gets stuck at the point of discovery because few academic researchers can conduct all the types of studies needed to develop a new drug. If a pharmaceutical company with the resources to further the research does get involved, substantial preclinical work begins with efforts to optimize the chemistry of the potential drug. This involves an iterative series of chemical modifications and tests in progressively more complex systems — from cell cultures to animal tests — to refine the potential medicine for use in people. Only if these stages are successful can a potential treatment move to clinical trials in patients.
Unfortunately, the success rate in this preclinical process is low, with 80 to 90 percent of projects failing in the preclinical phase and never making it to clinical trials. And the costs are high: it takes two to four years of work and $10 million, on average, to move a potential medicine though this preclinical process. Drug developers colloquially call this the “Valley of Death.”
. . .If a compound does survive this preclinical stage, TRND will work to find a company willing to test the therapy in patients. There are several stages to the clinical trials process that can take several years before the safety and efficacy of a new drug is determined. FDA will only approve a drug for general use after it passes these trials. The clinical trials process is also expensive, but the failure rate is lower at this stage.
Well, a tiny bit lower. I think that the general clinic-to-market failure rate is still somewhere around 90%, but it varies by therapeutic area. And that 80 to 90% failure rate that they quote for preclinical is a bit lowballed, I’d say, because you’d want to subtract that things that get recommended to the clinic (but really should never have been). But overall, this is a reasonably clear-eyed look at the difficulties involved. If they can get some things to the point that a company or foundation is willing to take on the (now somewhat reduced) risks, that’ll be great.
The last thought I have (for now) is that I feel like writing a bunch of people and asking them why the NIH is doing this, since they’ve been telling me for years that this is what the NIH already does, anyway. The “Big Pharma does nothing but rip off NIH” meme hasn’t surfaced for a little while, but it’s always out there.