Our fights

For more than 60 years it has been fighting against fatal diagnoses and in favor of rights and treatments, the Association has spearheaded many breakthroughs in medical research and support for patient’s health autonomy. Nonetheless, 20 years after the arrival of patients’ rights laws and the introduction of national plans, and while innovation therapies for rare diseases are being created, much remains to be done to truly change the lives of the people affected. 

Medical research as a national priority. 

Fact: although French researchers regularly demonstrate their excellence, especially in biotherapies, French research suffers from chronic under-investment and a lack of efficient organization. As such it is urgent that investments be made to increase effectiveness and bridge the health gap compared with our European neighbors, especially Germany and Italy. Furthermore, the bureaucratic organization of French public research into multiple entities is truly an obstacle to the development and recognition of our innovations in France.  

The Association advocates for:  

• simplifying the organization of French research and of recognition;
• increasing investments in medical research to catch up to the most efficient countries;  
• implementing a true strategy of innovation, combining “free” non-programmed research and a significant portion of financing according to goals, with an increased emphasis on results. 

Fact: the gene therapy revolution is underway for rare diseases as well as common diseases, with an increase in recent years of marketing authorizations and trials. Although proofs of concept are very often derived from French research - public or private not-for-profit - today it is other countries that are investing massively to solve the scientific and technical challenges necessary for the large-scale production of these innovative treatments at an affordable cost. Essential to the project are the installation of manufacturing sites, thousands of highly skilled jobs and a guarantee of supply for these new treatments. France’s weakness in the development of our inventions is damaging in two ways: we receive only a miniscule part of the value created by our research, and drugs are placed on the national market (when placed at all) at very high prices. 

The Association advocates for:  

• Creating investment funds that are sufficiently capitalized and able to be used at all stages of development of drug candidates. These funds must be accompanied by strong knowledge in the field, with the consultation of scientific, medical and financial experts, and must go hand in hand with an attitude of engagement and a culture of innovation; 
• Creating a multidisciplinary R&D center devoted to innovation in bioprocesses for therapies derived from knowledge of the genome, in order to improve production yields by a factor of 100. 

Fact: patients and their families affected by serious diseases, already dealing with complex decisions, are facing an incomprehensible administrative burden and timelines that are often incompatible with their needs and with the progression of their disease. In an environment where the simplification measures taken in recent years have had only a very slight impact on experiences, support for autonomy in health and independent living aiming to bolster and strengthen patients’ self-determination is vital. 

The Association advocates for:  

• establishing the principle of declaration instead of systematic justification; 
• coordinating public funding and providing the patient with a sole contact; 
• guaranteeing the right to support for health autonomy for people affected by serious diseases, and recognizing the initiatives, such as that of AFM-Telethon, that contribute. 

Fact: the law of 2005 led to major progress for the rights of people with disabilities, but the fight must continue in order to entrench some of these rights. 

The Association advocates for:  

• accelerating accessibility for people’s travel and housing, and more broadly, helping them achieve their life plan; 
• ensuring access to assistants who are trained to meet the needs and adapt to the individual home life of each patient; 
• ensuring the right to full financing without remaining copays of disability systems, such as wheelchairs or technical aids suited to their needs; 
• ensuring the right to financial resources that ensure not just survival but a life of dignity. 

95% of rare diseases have no treatment. A national rare diseases strategy must be reinstated, with innovation for ultra-rare diseases. 

Fact: public hospitals are an essential factor in the care of millions of French people affected by rare diseases. Cancelled consultations, long wait times, bed closures: the financial resources of the national health care system are not adequate to guarantee access to care and rare disease expertise. National rare disease entities are part of an exemplary recent public health structure, but due to lack of resources they are currently struggling.
Within rare diseases, ultra-rare diseases represent a majority in terms of numbers (several thousand) but affect only a few dozen patients in France. While the proliferation of scientific breakthroughs, especially gene therapy, is a source of immense hope, there is no economic model for ultra-rare diseases, and the usual marketing authorization mechanisms are not suitable. 

The Association advocates for:  

• providing the necessary resources to public hospitals and to care facilities for people affected by rare diseases; 
• instigating a new multi-year rare diseases National plan and having a clear and cross-functional national strategy with the funding essential to its rollout; 
• implementing neonatal screening in keeping with the expectations of families and comparable to that of our European neighbors; 
• creating an organization/initiative involving public, private not-for-profit and private for-profit players to finance the development and production of treatments for rare diseases.