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        • Top manager of NANOLEK speaks about the pharmacoeconomics of innovative drugs for the treatment of genetic and orphan diseases
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        Top manager of NANOLEK speaks about the pharmacoeconomics of innovative drugs for the treatment of genetic and orphan diseases

        15.12.2020




        December 10 and 11, 2020 saw an open, hybrid academic and research forum "The latest genetics. Current state of knowledge about human diseases, promising approaches in health care" (based on the example of orphan diseases).

        The event was held as part of the "All-Russian Pharmaceutical Rally" and is expected to become an annual event that brings together experts, manufacturers, representatives of science and patient organizations for exchange of ideas on the advances of orphan medicine, treatment of "rare" patients, development of a knowledge-intensive system for diagnosis and prevention of the spread of hereditary diseases, ways to regulate innovative technologies and organize medical care for people suffering from life-threatening, and rare (orphan) diseases.

        The event was attended by the industry experts: representatives of the Federation Council, State Duma, Ministry of Health of Russia, Eurasian Economic Commission, medical and scientific communities, educational institutions, pharmaceuticals manufacturers, patient organizations.

        On December 10, Maxim Stetsyuk, Executive Director of NANOLEK, delivered a speech as part of the panel discussion "The role of science in the development of innovative drugs and treatment of genetic and orphan diseases".

        The organizers brought a variety of experts together to discuss a key topic conсerning modern, effective drugs used for the treatment of rare diseases. The following experts were also invited to participate in the event: Alessandro Cirrinchione (General Director at Novartis Gene Therapies), Vadim Kukava (Executive Director of the Association of the World's Leading Pharmaceutical Companies "Innovative Pharma"), Dmitry Kudlay (General Director at Generium), etc. The discussion panel was moderated by Roman Ivanov, rector of the Sirius University of Science and Technology (Autonomous non-profit educational organization of higher education).

        The participants were able to discuss the challenges in science and healthcare covering genetic and orphan diseases, the prospects for the development of Russian science and public healthcare system in the field of early diagnosis, medical care for patients, current scientific Russian and global discoveries in the context of orphan diseases, main trends in changing approaches to the treatment of such diseases in the future, R&D solutions in the field of their treatment.

        Maxim Stetsyuk, Executive Director of NANOLEK: "The development strategy of NANOLEK is to produce specialized drugs and vaccines in line with the National Preventive Immunisation Schedule. We decided to set up a domestic production in Russia as part of the Pharma-2020 project to address the issue of availability of such drugs. This particularly applies to idursulfase beta used for the treatment of mucopolysaccharidosis type II (in Russia about 150 patients reportedly suffer from MPS, and annual expenditure on this disease is approx. 3 billion rubles.)

        We have registered this drug and have already gone through some stages of localization, and in 2019 this disease was included in the VZN list and in the regimen for treatment of Hunter syndrome.

        The use of this drug allows to lessen the financial burden on the federal budget since idursulfase beta is 20% cheaper as compared to the current enzyme replacement therapy, i.e. the savings amount to more than 700 million rubles per year.

        The resulting budget savings generated through a cheaper drug therapy allows to increase the number of patients receiving treatment for a specific disease, in this particular case, MPS II, and provide treatment for patients with other nosologies by way of reallocation of funds. Therefore, it is crucial to create a product within the country through cooperation with research centers. We can boast of having successful experience of cooperation with the M.P. Chumakov Center (Federal State Funded Research Institution "Federal Scientific Center for Research and Development of Immunobiological Drugs of the Russian Academy of Sciences"), and we also plan to expand cooperation with other research institutions."

        THE ALL-RUSSIAN PHARMACEUTICAL RALLY ("Pharmprobeg-2020") is a professional cross-industry association and an annual automotive press tour, which aims to draw public attention to the most pressing issues of national goals in health care and related industries as well as the development and support of the entire chain: from regulating authorities, manufacturers, distributors and the pharmacy segment to medical communities and patient organizations. Due to the unfavourable epidemiological situation caused by the outbreak of coronavirus infection (COVID-19), in order to prevent its spread in the Russian Federation, "Pharmprobeg-2020" moved to a virtual format and became a permanent online expert platform.



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        • Top manager of NANOLEK speaks about the pharmacoeconomics of innovative drugs for the treatment of genetic and orphan diseases
        • Top manager of NANOLEK speaks about the pharmacoeconomics of innovative drugs for the treatment of genetic and orphan diseases
        Top manager of NANOLEK speaks about the pharmacoeconomics of innovative drugs for the treatment of genetic and orphan diseases
        Top manager of NANOLEK speaks about the pharmacoeconomics of innovative drugs for the treatment of genetic and orphan diseases
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