It is a positive commitment, but France must set an example by perpetuating and amplifying a truly ambitious national policy.
February 28 marked the 15th International Rare Diseases Day. On this occasion, thousands of initiatives – national, European and international – took place in more than 100 countries to bring visibility to the immense needs of the 300 million people living with a rare disease around the world.
These initiatives took place in a context of greater global commitment to rare diseases. On December 16, 2021, the United Nations General Assembly adopted a resolution, also supported by France, aimed at "Meeting the challenges of people living with a rare disease and their families". All 193 UN Member States have formally committed to implementing policies to meet the needs and rights of everyone in the world living with a rare disease and their family.
At the European level, respecting these commitments requires new political dynamics.
As part of the French Presidency of the European Union, a Conference was held on Feb. 28 in Paris, dedicated to “Care and innovation pathways for a European rare disease policy”.
This conference is an important political step for the launch of the construction of an ambitious European plan against rare diseases. The future of the 30 million people living with a rare disease in Europe depends on the actions taken today by policy makers. 95% of rare diseases have no specific treatment and 85% of them affect less than one in a million people. For all these diseases, coordinated action at European level, with concrete goals, will bring better added value from all member countries so that every person living with a rare disease in Europe can benefit from scientific, technological, and therapeutic progress. This is an emergency, and everything must be done to accelerate this mobilization.
For more than 20 years, the members of the the Rare Diseases Platform have strongly contributed to the emergence and implementation of the three French Rare Diseases Plans. As such, they welcome France's political initiative to mobilize the European Union.
However, this can only be effective if each country involves its health system in the fight against rare diseases.
France must be exemplary by conducting a global and ambitious policy on its territory.
Indeed, in our pioneer country, there is still much to do:
- accelerate diagnosis of these diseases and eliminate misdiagnoses of which hundreds of thousands of French people are victims;
- ensure that health pathways allow access to information and to the best medical and paramedical expertise and support for health autonomy;
- stimulate innovative research and guarantee the therapeutic and industrial developments necessary to multiply innovative treatments, including for ultra-rare diseases;
- guarantee patient access to existing treatments as quickly as possible. It is unacceptable that some existing treatments are not available for French patients or that for lack of neonatal screening, children are victims of major loss of chance.
The third National Rare Diseases Plan will end in 2022. The actors of the Rare Diseases Platform ask the French Government to:
- decide now to organize the evaluation of NRDP 3, continuing its funding in 2023;
- launch with all rare disease stakeholders the co-construction process of a new, ambitious National Rare Disease Plan, endowed with substantial financial resources, precise objectives and effective management;
- use its current leadership in Europe to ensure that the momentum given today is followed by the construction of a European plan to fight rare diseases that will complement and strengthen national efforts.
- In addition, they ask all the candidates in the presidential election to make a clear commitment to ensure that the challenges of rare diseases are at the heart of their health policies. However rare their illness, no citizen should be forgotten.
We are therefore counting on the continuation of France's political commitment to rare diseases made today at the European conference.
About the Rare Diseases Platform
Created in July 2001, the Rare Diseases Platform, which brings together 6 associations (AFM-Téléthon, Fondation maladies rares, Orphanet, Maladies Rares Info Services, Alliance maladies rares and EURORDIS - Rare Diseases Europe) is a unique place in Europe. It promotes synergies between patient associations, health professionals and public actors, to improve the management of rare diseases and develop therapeutic research.