The French National Plan for Rare Diseases

On 20 November 2004, the French Government announced the creation of a National Plan for Rare Diseases, the result of a wide consultation process involving rare disease experts, health professionals, patient organisation representatives (mostly from the French Rare Disease Alliance and Eurordis), and the Ministries for Health and for Research. This plan, welcomed by the rare disease community in France, is based upon ten strategic lines to be implemented during 2005-2008.

One year later, has the plan lived up to its expectations? 'Certainly!' says Alexandra Fourcade, project director for the Plan at the Ministry for Health. '67 centres of reference have been designated, which means that we will achieve our goal of 100 centres by the end of 2006 after the next call for tender. These 67 centres of reference cover the 18 rare disease categories that were defined. What now remains to be done is the coordination of these centres of reference with the “centres of competence” across the country' (i.e. expert centres, which for the main part still remain to be identified). The purpose of the centres of reference is to structure the diagnosis and care approach to be delivered by the centres of competence. Centres of reference are also supposed to develop clinical and research expertise. 'ALS (Amyotrophic Lateral Sclerosis) is a good example,' says Alexandra Fourcade. 'It has 2 centres of reference (with one coordinator) and 15 centres of competence, and they all work in coordination with each other. We need to do the same for all centres and for all disease categories.'

Françoise Antonini, Executive Director of the French Alliance for Rare Diseases (Alliance Maladies Rares) agrees that a lot of progress has been made, but she also stresses a few tricky points. 'Travel costs for patients travelling to centres of reference are theoretically reimbursed, but reality shows that reimbursement has yet to be implemented in many parts of France. Also, if travel costs are reimbursed, accommodation costs aren't! This is a real issue for people living far away from a centre and needing many examinations, making it impossible to do the trip in a single day.' 'However,' says Alexandra Fourcade, 'centres of reference should define national diagnosis and reimbursement policies and procedures for rare diseases to ensure homogeneity of reimbursement across France. These procedures will be disseminated to local health agencies, and the use of off-label drugs will be included in the procedures after the necessary regulatory changes. The Ministry for Health has also asked the French public health agency (CNAM) to create a national rare disease cell to ensure 100% reimbursement'.

The research aspect of the plan has been very successful. The PHRC (Clinical Research Hospital Programme) call for tender resulted in 24 projects funded for rare diseases, amounting to 6.65 million euros over three years. The ANR (National Research Agency) call for tender, issued by the GIS Maladies Rares, resulted in 35 projects amounting to 11.27 million euros!

EU countries, such as Germany and Spain, have been following the French Plan very closely. FEDER, the Spanish Federation for Rare Diseases, organised its National Conference on Rare Diseases in Madrid in November 2005, and put forward a similar plan. Centres of reference, research, and social and health actions were discussed. Rosa Sanchez de Vega, from the Spanish Aniridia Association, is very happy with the outcomes of the conference. 'The General Director of Quality and High-Inspection of the Spanish Ministry for Health committed to the creation of a network of centres of reference for specific pathologies or categories of pathologies.'

The Steering Committee that monitors the French Plan met on 8 November 2005. It will meet again in June 2006, and regularly thereafter, to check the progress of the Plan. It will focus on centres of reference, protocols, and coordination of healthcare and social aspects. 'We are paying close attention to the effective implementation of all the measures announced along the ten strategic lines. We want to be able to report new, concrete results in line with the positive outcomes of the Plan so far! We'd like the plan to set a precedent and be a benchmark for other EU Member States,' says Yann Le Cam, Eurordis' representative on the Steering Committee.

For more information:
Read the French National Plan for Rare Diseases >
National Plans for Rare Diseases: France >

This article was previously published in the February 2006 issue of our newsletter.

Author: Jerome Parisse-Brassens
Photos : Alexandra Fourcade © Alexandra Fourcade; patients with rare diseases © Claude Bilgoraj; Rosa Sanchez de Vega © Eurordis
Graphics: © Ministère de la Santé et des Solidarités