Because of the very high costs in developing drugs for rare genetic diseases, and the low return on investment, the pharmaceutical industry has rarely developed specific treatments for many of these diseases. But a new public-private partnership, called the European Rare Diseases Therapeutic Initiative (ERDITI), could provide an important new mechanism for developing new drugs.
In an article in this months open access international medical journal PLoS Medicine, Alain Fischer, Head of the Department of Pediatric Immunology at the Necker University Hospital, Paris, France, and colleagues describe how ERDITI (www.erditi.org) is bringing drug companies and academic researchers together to find new treatments for rare diseases.
"The thousands of compounds that have been developed by pharmaceutical companies for more common diseases but that were abandoned or failed to achieve registration for several reasons (such as biopharmaceutical properties, toxicity, lack of efficacy, or strategic reasons) represent a treasure worth exploiting," say the authors.
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