evant pathophysiological hypotheses and conducting preclinical research analyses.” Roussel, a coauthor of the PLoS Medicine article, added that the ERDITI approach is timely because “we may expect in the [coming] years that the mechanisms underlying many rare genetic disorders will be unraveled.” In most cases, the potential therapeutic efficacy must be evaluated in animal models, after which the compounds will go through the usual development process. “In some cases, if compounds have been previously developed by pharmaceutical companies and thus are well known in terms of toxicity [and] biopharmaceutical properties, this traditional route could be perhaps ‘shortcutted,’” explained Roussel. PUBLIC MEETS PRIVATE The Dutch Steering Committee on Orphan Drugs in The Hague, the Netherlands, one of the 10 European public research institutions supporting the ERDITI so far, “is extremely happy with the initiative of bringing academic researchers closer to existing ‘molecules on the shelf’ of pharmaceutical companies that may be useful in developing treatment of rare diseases,” said Sonja van Weely, PhD, scientific secretary of the Dutch organization. Funding is a challenge for the ERDITI, though. Although access to compounds of interest is dissociated from funding, the ERDITI does not provide any direct funding to researchers and pharmaceutical partners have no obligation to fund either development or clinical trials. Therefore, European researchers must get funding for their projects from customary sources. However, Roussel said, “we hope that companies will be interested in investing because they will be in position to apply for orphan drug designation for the molecule and to benefit from all regulation incentives.” Four major drug companies— Aventis, GlaxoSmithKline, Roche, and Servier—support the ERDITI so far. “Once we have shown that ERDITI is a feasible initiative, we intend to open ERDITI to other pharmaceutical companies or biotechnology societies, and to broaden its scope to a more global partnership,” concluded Fischer and colleagues.