The main event of the fifth day I of the All-Russian Pharmaceutical Run was an event on the topic of orphan drugs in the Vladimir region. Round table “The market of orphan drugs . Development Prospects in Russian Realities ”was held on the territory of the Generium Biotechnopark in the Vladimir Region.
The event opened with a press approach, where its participants talked about the achievements of the Vladimir region. “This is not only science and medicine. There is space here. The space in which others come to live, work, develop, and then give world achievements that we can be proud of, ”commented the First Deputy Governor of Vladimir Region Maxim Brusentsov .
The achievements of the company and the region were described by the General Director of JSC Generium Dmitry Kudlay : “Today we are in the TOP-5 world manufacturers of orphan drugs in the assortment of the existing portfolio. As of today, the Vladimir region, represented by the GENERIUM, has completely solved the issue of production of Russian drugs in the framework of 7 nosologies, having started a new format of 12 nosologies. According to the most expensive development in the world for the treatment of nocturnal hemoglobinuria and hemolytic-uremic syndrome, until now there has been a monopoly of one American manufacturer. We dealt with this issue in our country a month ago. ”
“It is important that the executive branch and the leadership of the region do not just support this project. There is a huge understanding that what is happening here is a real opportunity to reduce gigantic costs in federal and regional budgets. After all, the lion’s share of the budget in all regions from the health side is the cost of orphan drugs, ”said Dmitry Chagin, Chairman of the Board of the Association of Pharmaceutical Manufacturers of the Eurasian Economic Union, who moderated the event.
As part of the round table, Dmitry Kudlay presented a large report on the activities of Generium, the leader in the production of orphan drugs in Russia. The expert spoke about the main trends in the field of orphan drugs: “The orphan topic is one of the most difficult in pharmaceuticals. The scientific search itself is very limited in scale, and there are also few centers of competence that deal with this. ” The report touched on the priorities of national drug policy, biomedical cell products, the principles of classifying diseases as orphan in different countries. Special attention was paid to ways of introducing innovations, determining the needs of patients and personalized medicine in terms of orphan diseases. He also said that according to forecasts, orphan drugs will make up 55% of the total cost of the European pipeline up to 2022, and noted the need for benefits from the state to address rare diseases.
The expert presented the portfolio of JSC Generium, in which 37% are rare diseases in the therapeutic areas of clinical trials. The CEO of the company also spoke about the special space for life and work created for the employees of JSC Generium.
Maxim Brusentsov spoke about the position of regional authorities and the implementation of national projects: “The common goal of national goals is to increase the Russian economy by 2.5 times for the period 2024. This is a difficult task, but if you do not set ambitious goals, then we will not come anywhere. ” The expert shared his opinion on the development of clusters in the Vladimir region: “Our location is already called the pharmaceutical cluster. I hope that we will create it, add about 10 manufacturers and achieve the synergy effect. ”
Denis Belyakov , executive director of the Union of Patients and Patient Organizations for Rare Diseases IDBOOOI, raised the main problems of orphan diseases from the point of view of the patient community: “According to international data, 7% of the world’s population have one or another rare disease. And even in America and Europe, such a patient receives a diagnosis within 6-7 years. We think this term is higher. ” The report noted that in the presence of about 7,000 various rare diseases, there are only about 400 therapies.
Among the main issues of the discussed direction, the speaker noted the insufficient level of awareness of the medical community, the psychological isolation of the patient, the inability to receive medical and social assistance, the possible lack of pathogenetic treatment, the weak functioning of the laws, the insufficient number of specialized centers and the neglect of the attitude of large funds due to the complexity of therapy.
Ilya Wesker , director of interaction with government bodies of Takeda Russia, shared his opinion on the position of the world community in relations of orphan diseases: “The topic of orphan diseases and the availability of appropriate drugs has been vividly discussed over the past few years.” The expert spoke about three areas from which the policy of most countries in this area proceeds: issues of early diagnosis, the availability of national registries and the development of innovative financing approaches.
Sergey Simeniv , the head of the contract research organization X7 Research, spoke about the practice of conducting home visits as part of clinical trials: “Orphan diseases became the precursors of this service. Often the word “patient focus” sounds, and this is one example of its implementation. ” According to the expert, the appearance of this event solves such problems as the high cost of inpatient monitoring, the difficulty of finding patients in a certain region according to some nosologies, and the inability of patients to often be present in the center.
After the event, participants of the Pharmaceutical Run took part in an excursion around the Biotechnopark of JSC Generium.