“No private actor is interested” in financing research, due to lack of “commercial prospects”

While waiting for the fourth plan to combat rare diseases which will be unveiled in the spring, the Minister of Health and Labor, Catherine Vautrin, must announce additional funds on Thursday.

More than 7,000 rare diseases have been identified to date, more than 80% of which are of genetic origin. Cystic fibrosis, Ménière’s disease, Huntington’s disease, Charcot’s disease, Crohn’s disease, Duchenne muscular dystrophy… Although they affect a few patients each time, they still affect three million French people in total.

There are treatments for only 5% of them, and the big pharmaceutical companies are losing interest. To compensate, patient associations hope that the State will announce Thursday February 29, on the occasion of International Rare Disease Day, substantial funding for public research.

Because finding and developing a drug against a rare or ultra-rare disease costs tens, sometimes hundreds of millions of euros. Few patients, few opportunities… It’s difficult to amortize the cost for pharmaceutical laboratories, recognizes Laurence Thiennot-Herment, president of the AFM Téléthon. “The vast majority are ultra-rare diseases for which there are no commercial prospects.”

The latest plans have not kept their promises

Crigler-Najjar disease, a genetic liver disease, is one example. Until now, the research has been financed by Telethon funds and a treatment, experimental for the moment, has just been found. “We have had the first trials and now we know that 24 million are missing to treat children, to have the medicinesays Hélène Berrué-Gaillard, president of the Alliance Rare Maladies collective. But there have already been 30 or 40 million that have already been put on the table.”.

The problem is “that there is no private actor who is interested”, deplores Laurence Thiennot-Herment. The State must therefore invest. “In these ultra-rare pathologies, we have lots of scientists who have, in their drawers, drug candidates and they lack the means, an envelope to see the adventure through to the end”, regrets the president of the AFM Téléthon. The associations believe that, on the financial side, the latest national plans to fight rare diseases have not kept their promises.