Pharmacoeconomics: theory and practice
№2, 2021, Vol.9
The article describes the principles of the international classification of rare diseases, proposed by the organization Orphanet. The supranosological nature of rare diseases predetermined the task of developing their new classification, which is more convenient for coding in the framework of their treatment than ICD-11, but coordinated with it. For these purposes, the international organi- zation Orphanet, formed in France in 1997, together with WHO, is developing an additional classification of rare diseases with the assignment of each noso- logical unit its own unique code based on the collection of information and the formation of its own database of their nomenclature. The Orphanet nomencla- ture includes the following elements: a unique Orpha disease code, a common or most common disease name, synonymous disease names, keywords often used to search for a given disease, if any, and the disease definition itself.
This article analyzes the results of a sociological survey, which con- firmed the relevance of the issues of accessibility of providing medicines to various categories of citizens. The purpose of this study was to study the attitude of citizens to the existing system, as well as to identify the strengths and weaknesses of preferential security. The main research methods were: the method of studying and generalizing experience; comparative analysis; sociological survey; statistical. According to the presented data, the distribution of answers to questions is determined in % depending on the total number of respondents, including in the dynamics for the analyzed period (2019-2021). This study served as a tool for studying the key problems in obtaining preferential medicines (waiting times for a doctor to issue a prescription, the absence of a prescription in a pharmacy, the refusal of a doctor to issue medicines, etc.). Thus, the results of the conducted sociological survey showed that the LAW system plays a significant role in the structure of healthcare. At the same time, first of all, it is necessary to carry out additional work with various age groups of the popu- lation in the framework of providing information about the possibilities of the additional preferential security system.
Aim: to conduct a comparative assessment of the efficacy and safety of ERT drugs in MPS II type patients and analyze drug provision organization for pa- tients with this nosology. Methods: to conduct a comparative assessment of the efficacy and safety of ERT drugs, clinical trials data and real clinical practice data, including data from the Hunter outcomes survey register, were used. To assess the drug pro- vision organization were analyzed the current legal framework of the Russian Federation and public procurement data. Results and discussions: provision of pathogenetic therapy to the patients with orphan diseases is attributed to a number of administrative, clinical and eco- nomic constraints. The best example of the management of provision of this patients in Russia is a so-called Federal Program of High-cost nosologies (HCN) that has clear and transparent state regulation and the patient register that is essential for budget planning. The example of mucopolysaccharidosis type II (MPS II) that was included in HCN in 2019 shows noticeable increase in pa- tients’ access to therapy when transferring from regional budgets. Two medical products – idursulfase and idursulfase beta – are available in Russia for life-time pathogenetic treatment of this nosology. These products are produced using different cell lines; they have different INNs and are not interchangeable. Both drugs have registration clinical research, however, unique real world evidence is available for idursulfase only and show the survival rate of the patients with MPS II collected during 15 years of maintaining the international patient register cov- ering more than 1,000 patients from 129 countries, including Russia. According to the analysis, mortality risk in the patients treated with idursulfase is lower by 54% than in those who received no treatment (HR 0.46, 95% CI: 0.29; 0.72). This evidence can be used by health care decision makers to prioritize value of this medicinal product from both clinical and pharmacoeconomic perspectives. Predictability of therapy outcomes and higher prescription frequency according to current standards of care justifies idursulfase as a first choice treatment.