Gene-ius

Aug 01, 2011


Rich Whitworth
There have been several new stories and reports lately that paint a rather gloomy picture of the state of pharma R&D. From reports on high clinical trial failure rates (see "News") to the CEO of Eli Lilly needing to defend his position on maintaining R&D expenditure to investors, there is a message emerging, and pharma companies, or rather their investors, are beginning to question the future of R&D, and its current inability to stave off losses from patent expiries and generic competition. (I listened with interest to a news story on the radio this morning about common painkillers, such as aspirin and paracetamol, being more efficacious for dementia than antipsychotics — talk about generic competition.)

There was one beacon of light that left me feeling inspired this month, though, unfortunately, it too stems from something of an R&D failure... The Global Genes Fund aims to raise financial support for research into rare diseases, as well as increase awareness. It has a wonderful and simple message: "Every child, everywhere, regardless of disease, deserves treatments, therapies and support." It is common knowledge that the traditional pharma model does not serve orphan diseases well; pharma companies are, of course, more interested in the provision of drugs for the majority to justify R&D costs. But awareness of orphan diseases is steadily increasing, thanks to this project and other campaigns such as Rare Disease Day ( http://rarediseaseday.org/).

The fund is part of the larger US-based Global Genes Project ( http://globalgenesproject.org/), and the aim is that, in the near future, potential donors will be able to peruse research projects and make individual donations online. The idea of connecting the public directly to research activities in this way is interesting and could add incentive to tackling rare diseases. It will no doubt also leverage the vast potential of social networking and "crowdsourcing". I look forward to seeing how it could change the future landscape of R&D.

Best wishes,

Rich Whitworth, Editor