One of the most complicated issues of modern medicine is diagnosis, treatment and prevention of rare diseases. In the structure of rare diseases the vast majority accounts for genetically determined disorders. The introduction of genetic testing methods helped to identify a lot of hereditary diseases, that currently reach the number of 8,000 cases and according to estimations the frequency of their detection is about to increase. Medicinal products for diagnosis or treatment of rare diseases form the group of orphan drugs. Biologicals are the most effective medicinal products in the whole group, due to their ability to bind specifically with the «target receptor», which allows to use them as targeted drugs. Another advantage of biologicals, compared with preparations of chemical origin, is their low toxicity. Orphan drug provision is the most severe issue because the treatment of severe forms of rare diseases may require the prescription of an expensive drug. Manufacturing of orphan drugs may be unprofitable for the reason of the low prevalence of rare diseases. Therefore, the availability of orphan drugs for patients depends on the amount of state participation in manufacture stimulation. In various countries regulatory requirements for the recognition of a disease as a rare disease, and marketing authorization procedures for orphan drugs are developed with due regard to the social and economic characteristics of the country. The article presents the analysis of the regulatory requirements for various countries for the detection of rare diseases, and a common approach to marketing authorization procedure for the drugs used in treatment of rare diseases, exemplified by biological orphan preparations.

The present article describes principal problematic issues that refer to the standardization of allergen preparations. It is shown that at present domestic allergen preparations are being standardized by protein content in allergenic material, however, the mentioned characteristic does not reflect true allergic activity of a drug. There is a need in harmonization of domestic standardization technology for allergen preparations with the approaches of EMA (European Medicines Agency) and FDA (Food and Drug Administration), regulating their standardization in allergenic activity units, as well as the use of modern immunoassay methods for quantitative evaluation such as radioallergosorbent test, chemiluminescence analysis, immunoallergological test, enzyme-linked immunosorbent assay with inhibition phase etc.

Biotechnologie is a rapidly developing branch of modern science, which is increasingly used in various fields, especially in medicine. Increasingly developed and introduced into medical practice of new Biopharmaceuticals - medicines derived from modern biotechnology. The development of technology in the production of “similar” medicines is gaining momentum and leads to a reduction in the cost and availability of drugs. On biotech drugs now pinning major hopes on a more affordable means of combating the most dangerous non-communicable diseases of modernity, such as cancer, multiple sclerosis, Alzheimer’s disease, storage diseases, etc. In this review we will discuss what is the difference between the original medicinal substance from the generic, and what is the bioequivalent. To date, the biosimilars market is actively developing and has very good growth prospects. According to experts of the pharmaceutical industry in the coming years, the biologics will be at least 50% of all medicines.

The present article describes clinical signs of meningococcal disease. It justifies the necessity of its vaccine prevention. The article provides with the historical data on the evolution of inoculants from corpuscular inactivated vaccines to modern chemical polyvalent vaccines. It provides a glimpse of the efficacy of the use of corpuscular vaccines and considers the corresponding implications. The historical overview includes the use of mono-, di-, tetravalent polysaccharide vaccines, and the discussion on the role of bactericidal antibodies in the formation of immunity against meningococcal disease. The article also describes the experience of using polysaccharide vaccines in various countries and in different age groups in randomized clinical trials. It provides with the data analysis about the effect of vaccination on the level of meningococcal carriage and the incidence of meningococcal disease. The WHO’s position on the use of polysaccharide meningococcal vaccines in routine practice are also provided. In the conclusion the article presents the prospects for further use of polysaccharide vaccines to control the spread of meningococcal disease.

The problems of quality and safety products derived from human blood plasma and hyperimmune animal sera as well as recombinant blood products resolved strict government regulation of their production processes. The risk of implications is minimized by plasma fractionation and purification of a specific drugs from various impurities (immunoglobulin aggregates, protease, plasmin, plasminogen, prekallikrein activator, IgA and IgM etc.). Viral safety is achieved by multi-step manufacturing process that includes at least two independent methods (treatment with solvent/detergent + incubation at low pH or pasteurization, combined with polyethylene glycol processing). It was justified that for today the technological process of the development of plasma preparations and hyperimmune animal sera has reached its limit. Their further development is the most likely to refer to specific improvements. The improvements will relate to increasing the efficiency of manufacturing technologies and methods of clinical use (preparations for subcutaneous administration, combinations of different immunoglobulin preparations, etc.), viral safety, ways to eliminate component, that were previously not considered to be able to influence the outcome of clinical use (soluble molecules CD4, CD8, HLA, thrombin, trace amounts of blood clotting factors VIII, IX, X, XI, XII etc.). At the same time new genetic engineered preparations with well-characterized molecular composition and a high selectivity for target impact are expected to appear on the market because of these unsolved issues. These are recombinant blood factors with altered properties; cocktails of recombinant antibodies and Fab-fragments of IgG, highly affine for toxin epitopes, etc. Therefore, in the upcoming years it is necessary to create in Russia a new system for assessing the quality, efficacy and safety of blood products, taking into account the future course of their development.

Presents information on 1st series of branch standard sample to characterization the quality of the candidate of branch standard sample tested for performance evaluation: specific activity of authenticity, a description of sterility, presence of mycoplasma, loss on drying, pH, filling accuracy, the residual oxygen content in the vials. Attested feature of the new series of the standard sample is a specific activity, because the purpose of the of branch reference standard activity live Rubella vaccine is the suitability of the results of determining the specific activity of the Rubella virus in vaccines. Indicator specific activity of the candidate in the of branch reference standard was assessed by interlaboratory studies compared to current 1st standard sample of the enterprise and 1st International Reference Reagent For Rubella (Live) NIBSC-91/688. Certified value of the index is set «Specific activity of branch reference standard activity for rubella (4,63±0,50) lgCCE₅₀/0,5 ml. Shelf-life stated by using test accelerated the degradation is not less than at minus 20°C.

The paper contains the results of the development of mathematical models for O-antigen synthesis by atoxigenic cholera vibrio strains KM 262 biovar El Tor serovar Ogawa, KM 263 biovar El Tor serovar Inaba, and M 377 O139 serogroup based on the modeling of the synthesis of O-antigen, produced in the course of cultivation of the virulent Vibrio cholerae M 41 strain belonging to classical biovar, serovar Ogawa. The data obtained provide the possibility to successfully perform the task of up-scaling the designed pilot technology for cultivation of atoxigenic cholera vibrio strains to be utilized on commercial-scale bioreactors.

The presented work comprises information about developed by Republic Scientific and Practical Center for Epidemiology and Microbiology the diagnostic kit for detection the BK virus DNA in clinical samples by the real-time Polymerase Chain Reaction method. Accomplished studies showed high level its diagnostic sensitivity and specificity. Suitability of developed kit for laboratory BK virus diagnosis was demonstrated.

To estimate the influence of the plantain juice on neurological manifestations of the severe acute cyclophosphamide intoxication, rats have been gavaged with 4 ml/kg plantain juice 1 h before cyclophosphamide dosing (intraperitoneally, 600 mg/kg). Within 3 h after cyclophosphamide administration, rats’ blood ammonia, glutamine and urea was increased. These alterations have been attenuated with the prophylactic administration of the plantain juice. At the background of the administration of the plantain juice the hypodynamia was less pronounced, duration of life has increased. Therefore, experimentally has been demonstrated the potency of the plantain juice usage for prophylaxis of hyperammonaemic and neurotoxic action of cyclophosphamide.