Orphan medicines towards rare diseases treatment
Abstract
The review analyzed information on the prevalence, causes and development of rare diseases; problems of diagnosis and current approaches to the treatment of rare diseases have been considered; the experience of orphan medicines approval in some countries has been studied; problems in the development and production of orphan medicines, and clinical trials have been analyzed. One of the main problems of medicines use in the treatment of patients with rare diseases has been defined as the high cost of orphan medicines, which is associated with the development of biotechnological approaches and methods, as well as the difficulties of conducting clinical trials. The production of orphan medicines in some countries is supported by state incentives, that promote increasing of availability of treatment with medicines belonging to the specified group for patients with rare diseases.
Key words
Soldatov A.A. – doctor of medical sciences, chief expert of office of expertise allergens, cytokines and other immunomodulators of center for examination and control of medical immunobiological preparations
Avdeeva Zh.I. – doctor of medical sciences, professor, chief expert of office of expertise allergens, cytokines and other immunomodulators of center for examination and control of medical immunobiological preparations
Alpatova N.A. – candidate of biological sciences, chief expert of laboratory of immunology of test centre of quality expertise of medical immunobiological preparations
Olefir Yu.V. – doctor of medical sciences, General Director
Bondarev V.P. – doctor of medical sciences, professor, director of Centre of expertise of the medical immunobiological preparations quality
Lysikova S.L. – candidate of medical sciences. 1st category expert of laboratory of immunology of test centre
of quality expertise of medical immunobiological preparations
