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Medical News and Perspectives
October 26, 2005

European Researchers, Drug Companies Join Forces Against Rare Diseases

JAMA. 2005;294(16):2014-2015. doi:10.1001/jama.294.16.2014

European academic research institutions and pharmaceutical companies have teamed up to encourage the development of treatments for rare diseases. The goal of the European Rare Diseases Therapeutic Initiative (ERDITI): to promote the evaluation of compounds that have been abandoned by drug companies.

Although development of drugs for rare diseases benefits from orphan drug legislation in Europe and the United States, most companies cannot justify the cost of traditional pharmaceutical research, including screening of compounds and preclinical development. “The thousands of compounds that have been developed by pharmaceutical companies for more common diseases but that were abandoned or failed to achieve registration . . . represent a treasure worth exploiting,” explained Alain Fischer, MD, of Necker University Hospital in Paris and colleagues in an article recently published online in PLoS Medicine (doi:10.1371/plosmedicine0020243). “The availability of such compounds could allow us to shortcut the traditional route of pharmaceutical development and evaluate swiftly—at minimal costs—drug candidates for the treatment of rare diseases.”