The Expert Council of the Circle of Kindness Foundation has approved the inclusion of new nosologies in the list of diseases whose treatment it sponsors. One of the new items on the list is chronic hepatitis C, despite the fact that this is not an orphan disease.
This is a curable disease caused by the hepatitis C virus, which leads to liver cells inflammation, their death and replacement by connective tissue. Without treatment, it leads to liver cancer and cirrhosis, with onset in 20 to 30 years after one was infected as a child. According to the head of the Foundation, Alexander Tkachenko, the inclusion of the disease in the list of nosologies of the Circle of Kindness “will allow us to actually get rid of chronic viral hepatitis C in children.”
For the treatment of such patients, the Foundation will purchase drugs registered in Russia: Gilead’s Epclusa (INN velpatasvir+sofosbuvir) and AbbVie’s Maviret INN glecaprevir+pibrentasvir).
Other additions to the list include PIK3CA-mutation related overgrowth spectrum (PROS), a group of genetic disorders that leads to overgrowth of various body parts. In the absence of treatment, patients develop serious complications such as hemorrhages and embolism, vascular malformations, neurological and skeletal disorders, and chronic pain development.
The Foundation will now treat children with congenital bone marrow failure syndromes, hemoglobinopathy (beta-thalassemia and others), acquired idiopathic aplastic anemia, glycosaminoglycan metabolism disorder, and osteopetrosis. According to Aleksandr Rumyantsev, chief freelance pediatric hematologist of the Ministry of Health of Russia, “the peculiarity of the group of patients with these diseases is that, unlike those with primary immunodeficiency, they do not have to be administered the drug all their lives: the treatment is discontinued when their own immunoglobulin production is restored.”
In March, after several unsuccessful attempts, the Center for Medical Support held successful auctions for the supply of Translarna (INN ataluren) and immunoglobulin for the treatment of the wards of the Circle of Kindness. The only company that applied for participation in the competitive selection procedure was Pharmimex.
