The Uncensored Idea

I seem to be getting stuck on a single theme these days. However, I was recently in a meeting of presidents of U.S. and European universities and the topic of open-source research and education again came up in several presentations, including, of course, my own. After the third paper mentioned open-source applications, I decided I should raise with the readers of Change an idea I discussed in the hallway with one of the meeting participants. So here it is, in pretty much uncensored form.

One of the problems in drug development is that the costs associated with bringing a drug to market—especially costs incurred in Phase I, II and III testing—are high. Thus, pharmaceutical companies increasingly are focusing primarily on drugs that have a very large target population and/or that can be priced at a level which will allow them to recoup their development costs. Drugs that target diseases affecting small numbers of the population are not likely to interest the major pharmaceutical companies—and ditto for drugs that target diseases affecting the poor of the world.

Why not create a large open-source network, to both develop and test drugs, that could be used, for example, to treat HIV/AIDS patients in the poor nations of the world? Could we create a new compound through the interaction of hundreds of pharmaceutical scientists, who would assign the license for the compound to a worldwide open-source network? Similarly, an even larger network of clinical investigators could test the drug in various patient populations, funded by research agencies like the NIH, foundations and private philanthropy. The open-source consortium would hold the license for the intellectual property, create a database of shared information, and so forth.

The goal would be to create effective drugs against AIDS (or malaria and other infectious diseases) at a price that could be afforded by developing nations. The consortium would grant a royalty-free license to a pharmaceutical manufacturing house that would agree, as part of the license, to make and distribute the drug for a fraction of the cost that a normal proprietary new drug would command.

Some of these steps are already in process. For example, the NIH has funded studies that examine the efficacy of lower-cost alternative therapies for AIDS. The Gates Foundation has funded consortia of multiple research organizations for vaccine development. Expanding the concept to a completely open-source network could multiply the effectiveness of these current funding sources several times over. Once the infrastructure for data collection, analysis and storage is created, the costs of conducting incremental studies and adding to the database becomes much lower than might otherwise be the case.

Imagine assembling a network of 2,000 clinical testing sites in hospitals around the world. If each studied a new drug in 50 patients, one could rapidly and inexpensively acquire data from 100,000 patients. The NIH or a large foundation might fund the consortium and its infrastructure, with each of the clinical sites taking ownership for the trial on its patient population, absorbing the costs locally.

An advantage of this open-source approach is that once a new drug is approved, postmarket surveillance data could be collected and analyzed as well, leading to the early identification of new side effects and additional data on the efficacy of the drug when used more broadly.