[Is the health care system ready to apply gene therapy preparations?]

The gene therapy is totally new approach to treatment of patient. It has significant therapeutic potential for wide range of diseases, including those caused by genetic disorders. The in vivo therapy is one of types of gene therapy meaning that impact on gene apparatus of somatic cells occurs straight within organism of patient. Among significant advantages of of this kind of therapy is potential opportunity of treatment of diseases which previously had no effective therapy or only supportive symptomatic therapy was administered. The article describes international approaches to classification of gene therapy preparations. The review of in vivo gene therapy preparations currently registered in different countries. Also, the features of their development, production and registration are analyzed. For many in vivo gene therapy preparations, due to small number of patients, clinical evidence base is limited, especially in terms of long-term clinical effect that complicates process of registration and economic evaluation for subsequent their inclusion in the system of state funding. It should also be noted that gene preparations currently registered abroad have extremely high cost that limits their widespread implementation into clinical practice. The production of gene therapy preparations requires a number of additional measures targeted to supporting product quality that complicates process of their production and registration. Due to absence of necessity in large production capacities, gene therapy preparations are produced centrally that results in their logistic complexities. It is actual to analyze international experience of distribution of in vivo gene therapy preparations in order to optimize approaches to regulation, assessment and financing of gene technologies in the Russian Federation.