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.”
European Researchers, Drug Companies Join Forces Against Rare Diseases. JAMA. 2005;294(16):2014-2015. doi:10.1001/jama.294.16.2014