New hope for patients with rare genetic diseases

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New hope for patients with rare genetic diseases

http://www.medicalnewstoday.com/medicalnews.php?newsid=29536

23 Aug 2005

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. "

Citation: Fischer A, Borensztein P, Roussel C (2005) The European

Rare Diseases Therapeutic Initiative. PLoS Med 2(9): e243.

Public Library of Science

http://www.plos.org