Drug Security Forum

Orphan Diseases and Medicines: International Cooperation as a Tool to Improve Effectiveness and Access to Treatment

14 Jun , 10:00–11:30
‘Ensuring Drug Security’ Russian Pharmaceutical Forum
Pavilion G, conference hall G4

Patients with rare diseases around the world face significant challenges in ensuring early and accurate diagnosis and access to life-saving treatment. Orphan diseases place a heavy burden on the healthcare system due to the limited availability of pathogenic therapies and their high cost. The development of new drugs is a knowledge-intensive and costly process. The feasibility of investment in it by pharmaceutical companies depends on the size of the potential market. In addition, the limited number of patients makes it difficult to conduct full-scale clinical trials in one country. Cross-country collaboration at all stages of making treatment of patients with orphan diseasesavailable, from development, clinical trials, collection, and analysis of real-world clinical practice data to consolidation of demand for procurement, is of particular importance in this context. What strategies for ensuring the availability of orphan drugs are being implemented in the Russian Federation and in foreign countries? What approaches are most appropriate for molecular genetic research for orphan diseases? What solutions can contribute to the creation of rare disease patient registries? What obstacles do pharmaceutical companies encounter when introducing orphan drugs to the Russian and foreign markets? What approaches exist in Russian and international practice to increase the effectiveness of financing innovative orphan drugs? Should the experience of the Circle of Kindness Foundation be extended to the provision of drugs for adult patients? How to ensure the availability of unregistered orphan drugs? What platforms are needed to build a cross-country dialogue on drug development and improve the regulatory environment for market access?

Sergey Kutsev, Director of the Federal State Budgetary Scientific Institution “Medical Genetic Research Center named after A.I. Academician N.P. Bochkov"; Chief Freelance Specialist in Medical Genetics of the Ministry of Health of Russia

Hesa Sabah Al Doseri, Chief, Health Facilities Regulation, National Health Regulatory Authority of the Kingdom of Bahrain (NHRA)
Yury Zhulev, President, Russian Hemophilia Society
Elena Maksimkina, Director, "Federal Center for Planning and Regulation of Medical Supply Circulation" of the Ministry of Health of the Russian Federation
Inga Nizharadze, General Director, Skopinfarm
Vitaliy Omelyanovskiy, General Director, Center for Healthcare Quality Assessment and Control of the Ministry of Health of the Russian Federation
Aleksandr Rumyantsev, Scientific Director, Dmitry Rogachev National Research Centre (online)
Aleksandr Tkachenko, Archipiest; Chairman of the Committee of Charity and Social Work of the Civic Chamber of the Russian Federation; Chairman of the Board, Circle of Kindness Foundation
Oleg Ergashev, Vice-Governor of Saint Petersburg

Front row participants
Darya Kryuchko, Head of the Department of Translational Medicine and Innovative Technologies, Federal Medical and Biological Agency (FMBA of Russia)
Oleg Lapochkin, Committee on Health Care and Health-Saving Technologies, Business Center for Economic Development of the CIS