A new form of partnership between academic institutions and pharmaceutical companies, the European Rare Diseases Therapeutic Initiative (ERDITI), is encouraging the development of treatments for rare diseases by reviving interest in compounds that have been ’left on the shelf’ and looking at alternative uses for those currently in development.
ERDITI was set up in 2004 under the sponsorship of the European Science Foundation and is coordinated by the French Institute for Rare Diseases Research in Paris. The ERDITI partnership is based on a charter which defines a streamlined facilitated process of collaboration between academic teams and pharmaceutical companies to develop drugs for rare diseases. The charter comprises three parts : a working procedure, a standard material transfer agreement and a standard intellectual property rights agreement.
Despite five years of European orphan drug regulation (and similar legislation in the USA, Japan, Australia and Singapore) to promote development of therapies for rare diseases, many pharmaceutical companies are not prepared to commit to the costs involved in the compound screening and preclinical development necessary to develop a drug for the extremely limited markets represented by rare disease patients. However, these same companies have a potential treasure trove of compounds developed for more common diseases but that were abandoned or failed to achieve registration. The principle behind ERDITI is to open up this treasure trove to academic researchers interested in developing the compounds into a potential therapy for a specific rare disease. “The availability of such compounds could allow us to shortcut the traditional route of pharmaceutical development and evaluate swiftly and at minimal costs drug candidates for the treatment of rare diseases”, says Claire Roussel, ERDITI’s coordinator.
The heterogeneity of rare diseases and their sheer number – over 7000 identified to date – means that most are poorly characterised and lack diagnostic tools and appropriate treatment. Through the ERDITI partnership, researchers working on the characterisation of a particular rare disease present a proposal for evaluating a compound’s potential therapeutic interest through in vitro and animal models to ERDITI’s scientific advisory board. The board, made up of experts from both the public and private sectors, assesses the proposal, and, if accepted the ERDITI coordinators then consult with the pharmaceutical partners on the availability of the selected compounds.
“The industry partners involved in the ERDITI project are convinced of the usefulness of such a partnership. But for each given proposal, the scientific committee must be assured of the pertinence of the scientific hypothesis, that the study will be well run and of quality and that it will answer the question being asked” says Anne Bousseau, the Sanofi-Aventis representative on ERDITI’s scientific board.
If a suitable compound is identified in a drug company’s compound library they sign an agreement with the academic team in which they agree to provide ’reasonable quantities’ of the drug for preclinical studies. The company has no obligation to produce further quantities of the compound but it must allow the academic team to synthesise the compound as required for the research.
The academic team, in turn, agrees to share its research data with the drug company. The results applicable to rare diseases remain the sole property of the academic partner in terms of publication and patent application. Any results that have applications outside rare diseases become the property of the drug company.
At this point the company can decide to exploit the results of the preclinical studies in which case it negotiates a licence agreement with the research team. Alternatively it may decide not to continue development of the compound into a potential orphan drug in which case it grants licence rights for the development of the compound by a third party.
So far four major drug companies - Sanofi-Aventis, GlaxoSmithKline, Roche, and Servier – and ten European academic institutions are partners in ERDITI but researchers from any European research institute can apply. The coordinators plan to open ERDITI to other pharmaceutical companies or biotechnology societies as soon as they have shown that ERDITI is a feasible initiative. ’The ERDITI project has been developed at an optimal moment for researchers as the potential of recent advances in human genomics and pathophysiological knowledge of rare diseases now open the way to therapy development’, says Pascale Borensztein, general secretary of the French Institute of rare disease research. ’We hope that more researchers will see the potential of this partnership approach in the future’, she adds.
One of the future challenges for ERDITI is funding. No direct funding is provided to researchers, and pharmaceutical partners are not obliged to fund either development or clinical trials. The European researchers interested in establishing an ERDITI partnership must currently find funding for their projects from the usual sources of academic financing. However, the coordinators remain optimistic that companies will be interested in investing because of the incentives provided by orphan drug regulation once their compound has been designated. In the future they hope that new forms of financing can be envisaged on a European scale to support the development of these new drugs.
For further information contact the ERDITI coordinator: Claire Roussel
http://www.erditi.org
