New hope for patients with rare genetic diseases

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 month’s 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.

The idea behind ERDITI is that participating drug companies give these “abandoned” compounds to academic researchers who then investigate whether the compounds might be effective for treating rare diseases. Companies benefit from this relationship with academia in many ways, say the authors–for example, by demonstrating corporate social responsibility. The research on treatments for rare diseases may also lead to the discovery of drugs for the treatment of more common diseases, which could be highly profitable to industry.

The ERDITI partnership between industry and academia, say the authors, is governed by a charter of collaboration which includes “a standard agreement that describes the objectives of the research and defines the framework for the transfer and use of compounds. It also defines the rights and obligations of each party in terms of protection of information, intellectual property, and industrial property.”