It’s a relief for thousands of patients with cystic fibrosis in France. Monday, June 28, the Minister of Health Olivier Véran announced that two new treatments, Kaftrio and Symkevi, will now be reimbursed by Health Insurance. This announcement comes after a negotiation initiated by the Economic Committee for Health Products (CEPS) with the American laboratory Vertex Pharmaceuticals on the price of these two pharmaceutical specialties, used in combination with the drug Kalydeco. “The negotiations resulted in an agreement allowing these treatments to be covered 100% by Health Insurance. The reimbursement of these drugs will intervene in the next few days. “, the ministry said in a press release.
Concretely, thanks to this agreement, these two new so-called “innovative” treatments. “Today, a child is born every three days with cystic fibrosis and the disease still disrupts the daily lives of many families and victims. The arrival of innovative treatments is thus a real hope. », Adds the ministry. These medicines (always in combination with the Kalydeco treatment), may be used in patients aged 12 years and over. These have been granted by the Haute Autorité de Santé an improvement in actual benefit (ASMR), level II and III respectively, in the management of cystic fibrosis: in May 2020 for Symkevi and in August 2020 for Kaftrio.
Relief for patient associations
Minister Olivier Véran met the associations “To overcome cystic fibrosis” and “Association Grégory Lemarchal” on the occasion of this agreement. According to him, it is “a tremendous source of hope for patients. This means very concretely that patients will benefit innovative and effective treatments, giving them the best chances of quality of life and increased life expectancy ”. The announcement was long awaited by these organizations who started a petition a few days ago to market the Kaftrio treatment as quickly as possible in France. “Kaftrio is vital for 5,000 patients suffering from cystic fibrosis. It is still not available in France although it has been authorized in Europe for more than 300 days. »Indicates the document.
Kaftrio treatment allows patients to regain better breathing capacity, therefore a general well-being. “It represents a gain of luck and for some patients the only hope of avoiding a lung transplant. This combination of molecules (ivacaftor / tezacaftor / elexacaftor), already marketed in the United States under the name Trikafta and known for its effectiveness, is therefore now indicated in Europe for the treatment of patients 12 years of age and over, carriers of only F508del mutation. “, notes the association Overcome cystic fibrosis. These treatments are aimed at correcting CFTR protein involved in the disease. According to Health Insurance, “They represent new perspectives insofar as the treatments available to date were symptomatic. ”
Cystic fibrosis is the rare genetic disease most common in the European population, whoconcerns 6000 patients in France. It is characterized by viscous secretions in several organs, mainly the lungs and the pancreas. It develops from birth, and evolves with episodes of aggravation. The digestive and respiratory functions are the main affected, and its disabling symptoms and the infectious and functional complications that result from it impact the life expectancy of patients. Today, thanks to advances in research, average life expectancy is between 40 and 50 years old, while she was only 5 years old in the 1960s.