A recent paper showing some (very preliminary) promising results for the treatment of brain tumours with dichloroacetate (DCA) has stirred up a bit of debate about the funding of drug trials. The issue here is that DCA is a cheap, non-patentable drug (though the researchers are trying to patent its use as a cancer treatment). As a result, the research group studying its cancer application have had difficulty obtaining funding, since the likelihood of anyone getting the money back is negligible. The cost of getting a drug to the stage where agencies such as FDA approve its use is huge; a 2002 study estimated the cost at over $800 million, and it is likely higher now. No corporation is going to put up that sort of money (for something that hasn’t even been conclusively shown to work) without getting a return on the investment.
I’m not going to comment on whether this treatment is likely to be a good one or whether it might have applications beyond a specific type of brain tumour – cancer treatment is way outside my area of expertise (Update – there’s a post from this angle at Science-Based Medicine). The research group does seem to be legitimate; in fact, when their first in vitro study showed some promising effects a lot of quacks and snake-oil salesmen immediately started selling DCA to cancer patients, Dr. Michelakis, the lead researcher, came out pretty strongly against the quacks.
My interest is more in how we (as a society) can deal with these types of situation. Pharmaceutical companies are profit-driven enterprises. They make huge investments in drug development, and in return can make some pretty significant profits. I don’t think being a profit-driven corporation is inherently bad. However, this model really isn’t equipped for situations like this, where an inexpensive drug that can’t be patented has some potential for being a significant treatment. The pharmaceutical companies probably aren’t going to donate on the order of a billion dollars to research a drug that they won’t be able to make much money on – they aren’t charities. At the same time, there isn’t really anyone else who can come up with that sort of money on a regular basis (maybe on a one-time basis, but not for every promising cancer drug out there). As a result, DCA may never get regulatory approval, even if it does work.
I don’t think there are easy answers here. The big problem is that it costs a lot of money to run the necessary trials to demonstrate that a drug is safe and effective. I would not support decreasing these requirements; even with current testing, harmful effects can turn up later once a drug is in wider circulation, and reducing the requirements would make this problem worse. Improving in vitro test methods to the point where they could start to replace more of the animal and human trials might be a start, though for the foreseeable future I think we’ll still need animal and human studies to confirm the results of in vitro tests. Funding from government agencies and cancer charities for this sort of research would also help, but again there is only so much money to go around, and the funding agencies would need to be able to clearly distinguish the legitimate hopes from the long shots from the money-making scams.
I’d welcome any other ideas for how a non-patentable drug could be brought to the point of getting regulatory approval.