Edition of 09 October 2006

The E-Rare project is now well and truly on track and already looks set to have a real impact on European rare disease (RD) research. This FP6 (the EU Framework Programme for Research and Technological development) coordination action embarked on a 4-year contractual period in June this year.

E-Rare is a project gathering public bodies funding or managing research programmes. It is financially supported through the ERA-Net scheme, a principal means for FP6 to support the networking of research programmes carried out at national or regional level (including their mutual opening, and the development and implementation of shared activities, such as trans-national calls or even joint programmes). E-Rare has a total budget of about 2 million €.

The fundamental precept of the E-Rare project is the firm belief in the ”snowball effect” to be gained from trans-national research on the European level, with the broadening of technological and logistical possibilities for the development of research activity and project expansion through a growing number of nations in partnership.

E-Rare's main objective is to maximize the efficiency and impact of research on rare diseases by providing the environment necessary to bring together clinicians and scientists and gather research infrastructures, patient cohorts and related biological material on a European scale.

One of the first activities scheduled in the work agenda will consist of a survey of national and/or regional research programmes on RDs aiming at identifying common gaps and overlaps in current national initiatives. This survey, together with the common definition of strategic priorities will serve as the basis for the further implementation of joint trans-national actions whilst encouraging a multi-disciplinary approach.

The E-Rare consortium is currently made up of nine full partners, coming from 8 different countries - Belgium, France, Germany, Italy, Israel, the Netherlands, Spain and Turkey as well as two affiliated partners from the Russian Federation and the Region of Lombardy in Italy.

Interview

…..with Pascale Borensztein & Igor Beitia Ortiz de Zárate, respectively Secretary General & Project Manager of the GIS Maladies Rares, the French Institute for Rare Diseases, responsible for the coordination and financial administration of the E-Rare project.


Orphanews: What are the particular advantages and challenges of a European project such as E-Rare?
PB: This is the first time that so many players from the European RD field have come together on the political research level to exchange and discuss best practices, common strategic issues and joint activities. The RD subject is a difficult one but taking part in the construction of a real RD European movement is an exciting challenge! Of course, we don’t know the precise outcome of this venture, but we’re going for it !

Read the full E-Rare interview with the French Institute for Rare Diseases

In this article published in the September issue of the WHO bulletin, Pieter Stolk et al. call for the creation of an Orphan Medicines Model List of drugs for rare diseases, to complement the WHO List of Essential Medicines (EML) compiled in 1977 to provide advice to Member states in their choice of medicines. The fear is that the WHO could lose touch with orphan drugs through too strict an application of their own EML criteria which currently excludes orphan drugs.

By establishing an Orphan Medicines Model List, argues Stolk, the WHO could explicitly reintegrate this type of medicine into its policy sphere. In addition, developing countries could find this list helpful for the improvement of access to the orphan drugs listed and the stimulation of relevant policies. Stolk concludes that this Orphan Medicines Model List could ultimately lead to the WHO’s implementation of an extensive Orphan Medicines Programme.

Kuzelova M, Kubackova K, Palagyi M, Smid M.

Hope for patients with rare diseases - "orphan" drugs (Review in Slovak)
Cas Lek Cesk. 2006;145(4):296-300.