Actuallity. Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disease caused by the production of antibodies to the platelet and megakaryocytes membrane structure, which causes to increase the platelet destruction, but also inadequate thrombocytopoiesis. e disease is characterized by a significant loss of ability to work and decreasing quality of life, higher mortality rate, as well as frequent hospitalizations, re­ecting to health care resources utilization. Therefore, burden of disease assessment is a point of high interest. Methodology. Two pharmacoeconomic models were developed for the "cost-minimization" and "budget impact" analysis on the basis of Microso Excel soware. e modeling hypothesis is comparable efficacy and safety of thrombopoietin receptor agonists (TPO), based on the absence of statistically significant differences from the results of an indirect comparison and the efficacy of romiplostim is assumed to be equal to the efficacy of eltrombopag. Direct medical costs were calculated based on the costs of drugs from the TPO receptor agonist group, the administration of drugs, «rescue therapy», bleeding treatment, and follow-up monitoring of patients with ITP during therapy. Calculations were based on registered limit transfer drug prices from the VED List and standards of the financial expenses per medical care volume unit of Russian Federation in 2018. e reduction in costs per patient per year is 796,967 rubles (33 %). Results. Clinical and economic analysis of drugs from the group of thrombopoietin receptor agonists in the therapy of chronic idiopathic thrombocytopenic purpura in adults has shown that eltrombopag in comparison with romiplostim in patients with chronic ITP at the background of previous treatment failure is more economically expedient being not less clinical effective. It is possible to reduce the budget impact of the healthcare system of the Russian Federation by 1,693 million rubles (8 %) for 5 years with the gradual increasing in eltrombopag share in the purchase of TPO receptor agonists. Conclusion. Eltrombopag with comparable efficacy and safety is more economically sound option of chronic ITP treatment as compared to romiplostim.

Overactive bladder (OAB) is a chronic disease that correlates with the age of the patient, and occurs in almost one in seven women and men. Pharmacotherapy is one of the main methods for correction of this nosology. Taking into account the absence of additional therapeutic option on the Vital and Essential Drugs List for patients with contraindications for the prescription of solifenacin and BTX-A, patients with intolerance to these drugs conduct, an analysis of the economic consequences of the use of mirabegron under the State Guarantees for Providing Free Medical Care for the OAB treatment. Approved recommendations on carrying out the budget impact analysis were used. Results of the study show, that the expansion of the mirabegron use in the treatment of patients with OAB in the target population leads to the reduction in the cost of medical therapy for this group of patients: with an increase in the share of mirabegron up to 5 %, costs will decrease by 7.8 %; with an increase in the mirabegron share up to 10 % costs will decrease by 12.5 %; when using in 15 % of the target population – by 22.3 %. Trend analysis of changes in the average weighted costs shows the following: an increase in the use of mirabegron leads to a significant reduction in total costs, the total benefit can reach 6 250 002 576 rubles per 3 years of the drug presence in the VED List, which allows attributing the OAB treatment with mirabegron to economically feasible and resource-saving medical technologies.

Actuality. Acute cerebral circulation disordersremain an important problem of modern health care. Available evidence suggests that both reperfusion and neuroprotection are important to ensure a favorable outcome of treatment. In the context of neuroprotection, considerable interest are peptideswith neuroprotective properties. A recent randomized controlled trial of the Cellex® showed positive results for treatment of severe patients with acute cerebral circulation disorders with less favorable outcomes and the highest direct medical costs, which makes the question of assessing the pharmacoeconomic properties of this drug relevant in the light of new data. Objective. To conduct a clinical and economic analysis of treatment outcomes for Cellex® and therapeutic alternatives used in patients with Acute cerebral circulation disorders and early rehabilitation period of the disease within the framework of the state guarantee programs of free medical care. Methodology. Pharmacoeconomic modeling, cost-eectiveness analysis, budget impact analysis. Results. Established that the use of Cellex® in the context of Russian practice in patients corresponding to the characteristics of the target population of this pharmacoeconomic study leads to reduce the budget burden by 26 % for 1 year, reaching 46 % at 4 years. e cost-eectiveness analysis indicates that Cellex® leads to greater eciency in the use of health resources. e sensitivity analysis conrms the stability of this result to a change in the market situation, expressed in an increase in the price of Cellex® preparation to 25 % inclusive. This indicates a high pharmacoeconomic feasibility of Cellex® in the context of therapy the ischemic form of acute cerebral circulation disorders in Russian Federation.

Aim. Conduct a comparative assessment of the pharmacoeconomic ecacy of using the combination Vezomni as compared to a combination of tamsulosin and solifenacin in the form of monopreparations in men with symptoms of the lower urinary tract against a benign prostatic hyperplasia. Methodology. The design of the study is a retrospective analysis of the literature data. Methods of pharmacoeconomic analysis – cost analysis, "budget impact" analysis, cost-effectiveness analysis, sensitivity analysis. Results. The use of Vezomni instead of a combination of monopreparations tamsulosin + solifenacin will reduce the burden on the budget of the state guarantee program by 11.68 % for 3 years. With a target population of 1,000 people, the savings will be 6.28 million rubles. The cost of treatment of symptoms of the lower urinary tract for 1 year will decrease by 2 340.76 rubles for 1 person. At the same cost, the use of a fixed combination will provide therapy with 13.23 % more patients than with monotherapy (1132: 1000 people, respectively). Сonclusion. The use of the combination drug Vezonmi is pharmacoeconomically justified and allows to lower the costs for the treatment of lower urinary tract symptoms that occur against the background of benign prostatic hyperplasia in comparison with the use of a monopreparations of tamsulosin and solifenacin.

A health economic study was conducted under the Russian economic conditions to evaluate the feasibility of the use of dexamethasone 0.7 mg as a sustained release intravitreal implant (hereinafter, DEX implant) in the treatment of diabetic macular edema. Cost-effectiveness analyses (CEA) were performed using a decision tree model with calculation of cost effectiveness ratios (CER), as well as incremental cost-effectiveness and cost-utility ratios (ICER and ICUR). Ranibizumab 10 mg/ml, solution for intravitreal administration (hereinafter, ranibizumab), and a­ibercept 40 mg/ml, solution for intravitreal administration (hereinafter, a­ibercept), were used as the comparator drugs; the modeling study covered a three-year period. A probabilistic sensitivity analysis (PSA) was carried out to evaluate the level of uncertainty of the obtained results. A budget impact analysis was also performed. Free scenarios were reviewed for each analysis: the main scenario for phakic and pseudophakic eyes with the weighted average effects of the study drugs; alternative scenario 1 for pseudophakic eyes with the weighted average effects; alternative scenario 2 for pseudophakic eyes with the maximum possible effects of the study drugs. A network meta-analysis of 10 randomized controlled studies (RCT) was performed to evaluate the weighted average effect. e maximum possible effects were determined based on specific studies, including observational studies. e results of the main scenario and alternative scenario 1 demonstrated that the comparator drugs were inferior to the DEX implant with regard to the economic feasibility, since they had higher CER; the ICER and ICUR for the comparator drugs exceeded the social willingness to pay threshold. e results of alternative scenario 2 demonstrated that the DEX implant was dominant with regard to ranibizumab, and it was superior to a­ibercept with regard to the CER, ICER, and ICUR. The budget impact analysis demonstrated that a 3-year use of the DEX implant will allow to save the funds equivalent to an additional treatment of 102–189 patients if it replaces ranibizumab (depending on the scenario) or 72–286 patients if it replaces a­ibercept (depending on the scenario). e sensitivity analysis demonstrated low level of uncertainty of study conclusions.

Relevance. The most effective treatment for focal dystonia (blepharospasm (BsP) and cervical dystonia (CD)) is use of botulinum toxin, including onabotulinumtoxinА (BoNTA-Ona), incobotulinumtoxin А (BoNTA-Inco), abobotulinumtoxinА (BoNTA-Abo). In 2016, it became possible to use ­exible-dose regimens of incobotulinumtoxin А, thus necessitating analysis of pharmacoeconomic properties of such regimens. Aim. To determine pharmacoeconomic properties of BsP and CD therapy with incobotulinumtoxin А relative to regimens based on onabotulinumtoxinА (BoNTA-Ona and abobotulinumtoxinА (BoNTA-Abo) accounting for properties of ­exible administration regimen of incobotulinumtoxin А. Methodology. Pharmacoeconomic analysis was performed from the perspective of Russian healthcare system within context of mandatory healthcare insurance, as well as from the perspective of overall social interests of Russian Federation, including impact upon GDP. Modelling horizon was 5 years. A complex Markov model was developed to estimate costs and outcomes. The parameter used for clinical efficacy assessment was number of days patient spend in remission state. Cost effectiveness analysis (CEA), cost utility analysis (CUA) and budget impact analysis (BIA) were performed based on modelling results, and sensitivity analysis (SA) was performed to ascertain model robustness. Result. Incobotulinumtoxin А in fl­exible dose regimen demonstrated pharmacoeconomic superiority during CEA for cervical dystonia and blepharospasm indications. When indirect costs are accounted for, superiority of incobotulinumtoxin А increases. Similar results were obtained during CUA. BIA indicates that use of ­flexible-dose regimen of incobotulinumtoxin А allows to achieve reduction of budgetary burden. SA has conrmed the robustness of these results. Conclusion. Use of incobotulinumtoxin А in a ­flexible-dose regimen is pharmacoeconomically justied.

State institutions determine the rules for conducting clinical trials of drugs; therefore, they must directly monitor their conduct. The main form of control over clinical trials of drugs by the state is the inspection. This is article describes the types of inspections, the main identified violations, proposed further actions and consequences.