Rare diseases: AFM-Telethon calls for the creation of a public fund to ensure access to treatment

To mark International Rare Disease Day on 28 February, AFM-Telethon is advocating for the creation of a public intervention and innovation fund in France for the treatment of ultra-rare diseases with no prospect of commercial profitability. The aim: to guarantee access to treatment for all.

95% of rare diseases have no treatment. And while innovative therapies have shown their effectiveness in the first few rare diseases, today the market is focused on those diseases with the most commercial prospects: the development of treatments for the rarest and most complex diseases is hampered by the lack of funding.

It is urgent to act to support access to treatments for all!

“As a pioneer in rare disease policy and early access to treatments, France must now create a financial mechanism to allow the development of and access to treatments for diseases without a commercial model. For thousands of patients affected by these often fatal diseases, there is no alternative plan!

To meet this public health challenge, AFM-Telethon proposes to create FITURARE, a public fund for intervention and innovation in the treatment of ultra-rare diseases with no prospect of commercial profitability. This fund would intervene by financing projects targeting specific therapeutic needs by immediately evaluating the therapeutic potential and the feasibility of making treatments available. Its long-term financing could come from the French health insurance system – it is indeed a question of treating patients suffering from serious diseases – but also from the pharmaceutical industry to compensate for market failure,” explains Laurence Tiennot-Herment in an article published in Le Monde on 28 February.


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