Purpose. To conduct a comparative clinical and economic analysis of the use of epoetin alpha (Eralfon) as a therapy of anemia in adult cancer patients with non-myeloid malignancies receiving chemotherapy in the conditions of real clinical practice in the Russian Federation. Method. In the study, a model of care for adult cancer patients with anemia receiving chemotherapy was built, the model takes into account various options for therapy with EPS. Results. The overall cost of treatment of cancer patients with anemia are less in the application of EPS short-acting — epoetin Alpha — 131 609 rubles, compared with the long-acting EPS — darbepoetin alpha 170 of 583.00 RUB, the difference amounted to RUB 38 of 974.00 (-23 %) in favour of epoetin alpha. Summary. Taking into account the data on the equivalent clinical efficacy of epoetin alpha and darbepoetin alpha, the use of the drug epoetin alpha (Eralphon) of domestic production as a therapy for anemia in adult cancer patients receiving chemotherapy in the conditions of real clinical practice in the territory of the Russian Federation is an economically more justified approach to the organization of drug care for cancer patients with anemia. 

Actuality. A major problem for rehabilitating patients after a stroke, other major cerebrovascular events or trauma (such as traumatic brain injury (TBI)) is increased muscle tone (spasticity) which in turn causes impairments of mobility and formation of pathological postures (which are oft en painful). In cases when it affects upper limb, the decrease in employment ability, capacity for self-service and overall quality of life of the patient becomes exceptionally pronounced. Botulinum toxin type A preparations (BTA) are first-line drug therapy for this type of disorder. Data indicating that different BTA-based drugs have differences in terms of compensation period (period during which the patient does not require re-administration of botulinum toxin) has become available. The pharmacoeconomic effect of this property within context of treating upper limb spasticity in the Russian healthcare system has not yet been investigated. Given the importance of compensation period both for patient well-being and for the burden upon the healthcare system, performing this pharmacoeconomic analysis represents significant interest. Aim. To assess the respective pharmacoeconomic performances of different botulinum toxin preparations as treatments for upper limb spasticity, specifi cally onabotulinumtoxin А (Botox®), incobotulinumtoxin А (Xeomin®), abobotulinumtoxin А (Dysport®) as well as Relatox®, a Russian botulinum toxin. Methodology. The analysis was performed from the perspective of Russian healthcare system in context of government guarantee program for provision of free medical aid. A Markov model was constructed to carry out this analysis. The following forms of analysis were performed based on modeling results: cost-effectiveness analysis, budget impact analysis, and several sensitivity analyses to assess model’s robustness to a number of assumptions as well as price fluctuations. Model accounted for differences between botulinum toxins in terms of compensation period in context of upper limb spasticity, as well as probability of treatment discontinuation for all causes which included severe adverse events and all other possible discontinuation circumstances. Analysis accounted for direct medical costs associated with botulinum toxin administration. The opportunity to optimize Botox use through utilization of diff erently sized vials was accounted for. Modelling horizon was set at one year (due to the way patient treatment discontinuation obtained from literature sources). This modeling horizon was sufficient to account for the disease’s burden upon the healthcare system. The number of patients in each simulated group for cost-effectiveness analysis was set at 1 000 patients, the number of patients for budget impact analysis was estimated based on epidemiological data for Moscow city. Result. The costs per 1 000 within the scope of one year were lowest for abobotulinumtoxin А (Dysport®) and amounted to 58 947 570 rubles. Other drugs had total costs amounting to 94 954 468 rub., 92 049 480 rub, and 97 143 620 rub for onabotulinumtoxin А (Botox®), incobotulinumtoxin А (Xeomin®) and Relatox® respectively. During cost-effectiveness analysis Dysport demonstrated lowest cost-effectiveness ratio and thus is pharmacoeconomically superior. Cost-effectiveness ratio were 316 752 rub for Dysport, 641 584 rub., 619 028 rub., 656 376 rub. for Botox, Xeomin and Relatox respectively. The Dysport superiority within cost-effectiveness amounted to 48.8 %. During budget impact analysis, Dysport as the drug with superior CEA results was compared to Xeomin. Analysis indicated that moving 25 % of patients from Xeomin to Dysport results in 11.7 % reduction of budget burden (which amounts to more than 23 million rubles when epidemiological data for Moscow city is used). Several sensitivity analysis were carried out and confirmed model robustness to price fluctuations of drugs involved up to 25 % as well as alterations of a number of underlying assumptions related to treatment discontinuation curve parameters and Botox dosing regimens. Conclusion. These results indicate that Dysport has pharmacoeconomic advantages when used for treatment of adult upper limb spasticity resulting from stroke and traumatic brain injury within the context of the Russian healthcare system.

Introduction. Regardless the pharmacoeconomic evaluation of trastuzumab emtanzine (T-DM-1) has been done before new data concerning T-DM1 effectiveness and also costs data require an update of pharmacoeconomic evaluation. Aim. To update pharmacoeconomic evaluation of T-DM1 in patients with HER2+ breast cancer (BC) and CNS metastases. Materials and methods. Cost-effectiveness analysis along with sensitivity analysis were performed. Direct medical costs and indirect costs (GDP loss) were accounted. Results. At 5 years modeling horizon total cost of trastuzumab emtanzine were higher comparing to lapatinib+capecitabine due to significantly higher overall survival (OS) observed in T-DM1 group. CER OS for T-DM1 was by 21% lower comparing to CER lapatinib+capecitabine (2 099 940 RUR/patient/year and 2 541 879 RUR/patient/year, consequently). Conclusions. The study showed T-DM1 is a cost-effective strategy in patient with HER2+ metastatic BC and CNS metastases.

Pompe disease (PD) — a rare disease with progressive muscles weakness, cardiac and pulmonary insufficiency and others vital life-threatening symptoms without enzyme replacement therapy (ERT). Treatment of PD is very expensive and data about social-economic burden of PD in the Russian Federation is absent yet. Analysis of direct costs (cost of ERT, laboratory and others tests, cost of medical devisees etc.) and non-direct costs (personal income losses due to temporary or permanent disability or necessity of care for a sick child, GDP losses etc.) have been calculated for PD with late onset. Direct medical cost in case of alglucosidase alfa use was less on 11.4 % vs standard therapy for PD onset in childhood as well as non-direct non-medical cost on above 40 % less for one year horizon of modelling. Alglucosidase alfa has high efficacy for PD treatment for patients with late onset treatment in adult age, but more costly vs standard therapy — 44.1 mln. RUR / patient / year. Expenditures for PD complications were in 2 times less in case of alglucosidase alfa usage. Cornerstone of expenditures in alglucosidase alfa group are cost of ERT, in the same time in standard treatment group — cost of PD complications, treatment of terminal phase and death. ERT could be extended to 16.4 % due to resource-saving effects of the use of alglucosidase alfa. Calculated socio-economic burden of PD has shown that, it seems appropriate to transfer funding for its treatment to the Federal level.

Introduction. Despite the high cost of life-threatening rаre diseases, not all expensive drugs for the pathogenetic treatment of orphan diseases are included in the list of vital and essential drugs. Aim. Analysis of drugs for pathogenetic treatment of diseases included in the orphan drugs lists, in order to determine the specific names not included in the list of vital and essential drugs (VET List) for medical use in 2019. Materials and methods. From the standards of medical care for rare diseases included in the Decree of the Government of the Russian Federation of April 26, 2012 № 403 and the order of the government of the Russian Federation of December 10, 2018 № 2738-p allocated 62 international non-patented  names of drugs. The data obtained from the relevant INN in the State register of drugs of the Russian Ministry of health of was analyzed. Results. 90 % of the drugs are reference, 34 % interchangeable, 81 % of the considered included in the VET List for 2019. The remaining 19 % is not necessary and therefore does not have a registered selling price. Summary. Analysis of orphan drug lists revealed the absence of some of the expensive drugs in the VET List, which makes it impossible to control the maximum selling prices of the manufacturer by the government.

The article deals with the main provisions of the basic document — «Rules of good practice of pharmacovigilance of the Eurasian economic Union», which regulates the study and prevention of adverse reactions and other problems associated with drugs in the member States of the Union, including the Russian Federation. Also, the Rules establish requirements for the organization of pharmacovigilance system in pharmaceutical companies, detail the work of manufacturers in the field of risk management, as well as determine the procedure for informing employees of the pharmaceutical industry and specialists of medical institutions of the authorized bodies about the identified safety problems in the use of medicines.