The academician spoke about the rapid development of technologies for the treatment of orphan diseases

Technologies for the treatment and diagnosis of orphan (rare) diseases are rapidly developing in the Russian Federation. This was stated by a State Duma deputy, President of the Federal State Budgetary Institution "NMIC of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev," Academician of the Russian Academy of Sciences Alexander Rumyantsev told the Izvestia Press center on March 25.

"Orphan technologies have been rapidly developing in the Russian Federation. Over the past four years, 17 diseases in adults and more than 100 nosological forms in children have been effectively brought under federal control with full funding for technology and drug provision. These are very important processes. Moreover, they were supplemented with neonatal screening for almost 40 diseases," he said.

At the same time, the expert recalled that neonatal screening of newborns is carried out for the third year. Over the course of two years, more than 600 patients were diagnosed at the level of genetic diagnoses. Some of them came under the control of the financing of the Circle of Goodness Foundation.

"It is important for doctors to evaluate the results of the extensive work carried out by the state in terms of the effectiveness of technologies or individual drugs in patients with orphan or rare diseases. There is no other way for such an assessment than conducting clinical multicenter studies, which is very important in order to obtain a specific result. For many diseases, new drugs are emerging that require replacing old ones, and in order to assess their effectiveness, comparative studies are needed," the professor emphasized.

Treatment — light: patients with COPD without disability ask to be given drugs

With a low degree of obstructive pulmonary disease, they are not allowed free therapy.

Earlier, on March 11, it was reported that a backup mechanism for providing medicines to patients with rare genetic diseases would appear in Russia. Such amendments to the law "On the Basics of Health Protection" were developed by the Ministry of Health. The changes were approved by the government commission on legislative activity, sources in the government told Izvestia.