Pharmacoeconomics: theory and practice
№2, 2015, Vol.3

Serpik V.G. 4904

Orphan drugs designed for rare disease management is a special group of drugs from the perspective of the pharmacoeconomic analysis being a mandatory part of any proposal for inclusion in the Restrictive lists. Thanks to unique opportunities offered by orphan drugs for rare disease management through acting at the pathogenetic level, these drugs have high social significance. But inherently high cost of these innovative drugs associated with their narrow market limited due to small target population, precludes from using the conventional pharmacoeconomic approach which involves comparison of pharmacoeconomic indicators — results of cost-effectiveness and budget impact analyses — of the innovative drug vs. current medical treatment (or palliative treatment in case of no treatment option available). As such, the authors have investigated the special pharmacoeconomic approach — “precedential” — in case of ruxolitinib in treatment of primary myelofibrosis. This approach implies comparison of the studied drug vs. other orphan and high-cost medicinal products included in the National drug lists. As a result, it has been shown that the pharmacoeconomic indicators of ruxolitinib are not higher than the same of the drugs included in the Essential Drug List .

Serpik V.G. 5050

Budget impact analysis and cost-effectiveness analysis for the therapy of chronic myeloid leukemia (CML) by tyrosine kinase inhibitors were performed by means of a developed analytical model of decisionmaking. This analysis defined potential budget impact of nilotinib as the second-line therapy in the frame of drug reimbursement program for subjects with hemophilia, cystic fibrosis, pituitary dwarfism, Gaucher’s disease, tumors of the lymphoid, haemopoietic and related tissues, multiple sclerosis, as well as for patients subjects to organ (tissue) transplantation (hereafter reimbursement program for high-cost drugs). Budget impact analysis has shown that conversion from imatinib to nilotinib for all subjects with chronic myeloid leukemia (CML) in Russian Federation receiving second-line therapy, based on theoretic consumption, would lead to RUR 1.985 billion increase of the federal budget (compared to imatinib only). Taking into account the actual nilotinib consumption, budget of the Scenario 1 does not exceed the real cumulative amount for chronic myeloid leukemia (CML) treatment in the framework of the reimbursement program for high-cost drugs and regional subsidized drug lists. In addition, it was demonstrated that nilotinib compared to imatinib can be characterized as a strictly preferable drug from the costeffectiveness analysis point of view, as it has a lower cost - effectiveness ratio.

Kulikov A.Y., Tishchenko D.G. 4910

In this article the economic burden of multiple sclerosis in the Republic of Belarus is presented. Analysis is performed using calculation direct and indirect costs.

Kulikov A.Y., Protsenko M.V. 5046

Chronic myelogenous leukemia (CML) is among the nosologies included in the governmental program for supply of medicines to patients with hemophilia, mucoviscidosis, pituitary dwarfism, Gaucher disease, malignant neoplasms in lymphoid, hematopoietic and related tissues, multiple sclerosis, and also after organ and (or) tissue transplantation. Only one medicine, imatinib, is currently available to CML patients within this program. However, 20% to 35% of CML patients have intolerance or develop resistance to imatinib. Therefore, this patient group should receive treatment with the second-generation tyrosine-kinase inhibitors, in particular dasatinib. Pharmacoeconomic analysis is required for dasatinib to be included in the list of expensive medicinal products. This pharmacoeconomic study investigates the use of dasatinib as a second-line CML therapy, based on «cost-effectiveness» and «budget impact» analysis. The study demonstrated that in terms of cost-effectiveness analysis, dasatinib is a strictly preferred alternative as compared to the high doses of imatinib. Moreover, taking into account consumption rate of dasatinib in the real world settings, it is possible to provide treatment to 100% of patients with resistance and/or intolerance to imatinib without additional funding above the total federal budget for CML treatment in 2014.

Babiy V.V., Kulikov A.Y. 4783

The article deals with the results of validation of the pharmacoeconomic model of emtricitabin/ rilpivirine/ tenofovir (Eviplera) inclusion in highly active antiretroviral therapy of HIV/AIDS in the Russian Federation.

Babiy V.V., Kulikov A.Y. 5095

The aim of this study was to determine optimal medical technique based on assessing cost and efficacy of treatment of von Willebrand disease using blood clotting factor concentrates (blood clotting factor VIII + von Willebrand factor): Wilate, Haemate P, Immunate. It was determined that in studies on assessing the efficiency of the concentrates more than 95% of patients rated hemostasis excellent/good. However, the studies were based on different scales of efficiency assessment, different vWF:RСo doses and different data for vWD patients (severity of the disease), therefore the efficiency of blood clotting factor concentrates might be not equal. Results of present study with the assumption about equality of groups, that were analyzed in studies on assessing the efficiency of the concentrates (although, studies on Wilate included more severe patients),evidence that prescribing blood clotting factor concentrate Wilate lead to cost savings compared to the older generation products.

Babiy V.V., Kulikov A.Y. 5079

In this study, a pharmacoeconomic analysis of chronic lymphocytic leukemia therapy in previously untreated patients was conducted, using treatment regimens obinutuzumab (Gazyva) + chlorambucil and rituximab +chlorambucil. The results of the study showed that though the costs of the first treatment course with obinutuzumab + chlorambucil are significantly higher, this regimen reduces the cumulative cost of subsequent therapy lines in patients with CLL (due to a longer progression-free survival). In the end of the third year of therapy, cumulative costs become relatively similar: with the use of the obinutuzumab + chlorambucil regimen, the cumulative cost per 1 patient/year will be 38,390 rubles higher compared to the rituximab + chlorambucil regimen. At the same time, the obinutuzumab regimen showed a lower cost-effectiveness ratio, i.e. it had an advantage over the alternative technology.

Kulikov A.Y., Pochuprina A.A. 5192

In this study we conducted pharmacoeconomic assessment of the use of canakinumab in patients with cryopyrin-associated periodic syndromes (CAPS) versus the symptomatic treatment alone. Incidence of remission in the treatment group was selected as the efficacy endpoint, and superior efficacy of canakinumab was demonstrated in the treatment group as compared to the symptomatic treatment. Based on cost-effectiveness analysis, it was determined that canakinumab treatment required considerable expenses; however, due to the small number of patients the impact on overall budget will be insignificant. It should also be noted that the treatment with this medicinal product will help reduce the costs of out-patient and policlinic care, in-patient medical care, as well as administration-related and complications management costs.

Kulikov A.Y., Serpik V.G. 4851

Pharmacoeconomic study comparing luteinizing hormonereleasing hormone agonists (LHRH-A), used for prostate cancer treatment was conducted. The study included drugs: buserelin, goserelin, triptorelin and leuprorelin. Pharmacoeconomic study included the following methods:cost analysis, cost-effectiveness analysis, cost minimization analysis and budget impact analysis. The time horizon for cost analysis, cost minimization analysis and budget impact analysis amounted to 1 year, whereas for costeffectiveness analysis it was equal to 6 months. Direct costs were taken into account. As a result, it was found that annual costs for the treatment of one patient for each of the considered drugs (buserelin, goserelin, triptorelin and leuprorelin) respectively accounted for 55 169 rubles, 90 130 rubles, 90 133 rubles и 94 599 rubles. Results of the budget impact analysis showed that the annual budget per patient using drug therapy buserelin, goserelin, triptorelin and leuprorelin respectively amounted to 129 545 rubles, 164 506 rubles, 164 509 rubles and 168 974 rubles. Cost minimization analysis demonstrated that under the assumption of equal clinical effectiveness review of medications, annual treatment of one patient using buserelin is characterized with 34 961 rubles compared with goserelin and 39 430 rubles compared with leuprorelin. Cost-effectiveness analysis using the criterion of decreasing the prostatespecific antigen (PSA) level, it was found that buserelin is characterized with the greatest rate of decrease in the PSA value, so it has the smallest value of the cost-effectiveness ratio and, thus, relative to the comparison drugs is strictly preferred drug.

Komarov E.A., Kulikov A.Y. 5045

Breast cancer (BC) is the most common type of cancer in women and one of the leading causes of death among women worldwide, including our country. According to the World Health Organization, over 11,000,000 women with diagnosed BC receive care and treatment worldwide. Each year approximately 1,200,000 new cases of breast cancer are registered and more than 500,000 women die, and it is estimated that the incidents will increase up to 1,450,000. The objective of this study was to determine a treatment regimen (pertuzumab+trastuzumab+docetaxel or placebo+trastuzumab+docetaxel) more advantageous from the pharmacoeconomic point of view, used in the treatment of HER2+ metastatic breast cancer (mBC), on the basis of comparison of costeffectiveness ratio, safety and life quality. The results of the cost-effectiveness analysis showed that cost-effectiveness ratios (effectiveness criterion – Life Years Gained, LYG) were as follows (over a period of 25 years: 1,823,530 rubles in the pertuzumab+trastuzumab+docetaxel group and 587,120 rubles in the placebo+trastuzumab+docetaxel group. The incremental costeffectiveness ratio for the health technologies compared was 8,150,535 rubles/LYG. The results of the cost-utility analysis showed that the cost-utility ratios (utility criterion – Quality Adjusted Life Years, QALY) were as follows (over a period of 25 years): 2,716,738 rubles in the pertuzumab+trastuzumab+docetaxel group and 908,787 rubles in the placebo+trastuzumab+docetaxel group. The incremental cost-effectiveness ratio for the health technologies compared was 10,187,748 rubles/QALY. The results of the budget impact analysis demonstrated that for the Perjeta-trastuzumab-docetaxel treatment regimen, the difference in the required budgetary funds was 5,711,668 rubles in comparison with the placebo-trastuzumab-docetaxel treatment regimen per treatment of one patient with BC (over a period of 25 years).

Komarov E.A., Kulikov A.Y. 5151

Although there have been improvements in the detection and treatment of breast cancer (BC) it remains the most common cancer in women and oneof the leading causes of death. In Western Europe and North America, breast cancer is the leading cause of death among women aged 35 to 54 years (20%), and the second leading cause of death in women aged over 55 years exceeded only by cardiovascular diseases. Breast cancer incidence increases with age, beginning from 40 years, with a peak at 60 to 65 years. The objective of this study was to determine, from the pharmacoeconomic point of view, the preferred treatment regimen (Kadcyla, lapatinib + capecitabine, trastuzumab + capecitabine, capecitabine), used in the treatment of HER2-positive breast cancer, on the basis of comparison of cost-effectiveness ratio, safety and life quality. The use of Kadcyla in the treatment of patients with HER2-positive breast cancer resulted in the highest values of life years gained and quality adjusted life years in comparison with lapatinib + capecitabine, trastuzumab + capecitabine, or capecitabine regimen. According to the results of the costeffectiveness analysis and cost-utility analysis it was found that the therapy with Kadcyla required higher costs to achieve LYG and QALY compared to those of the treatment regimens with the use of lapatinib + capecitabine, trastuzumab + capecitabine, and capecitabine, respectively. Incremental ratios in both analyses are higher than the willingness to pay threshold values for the Russian Federation.

Kulikov A.Y., Strunina (Rybchenko) Y.V. 4999

Follicular lymphoma is a disease requiring effective therapy that helps to improve a patient’s quality of life. In this study, a comparative analysis of subcutaneous administration of MabThera versus intravenous MabThera was performed. Considering that the formulations are equally effective, a cost minimization analysis was carried out, which showed that transition from intravenous to subcutaneous MabThera resulted in savings of 35,847 rubles per one patient during the treatment course due to the decreased drug administration expenses, medical staff costs, and expenses for hospitalization or day-time staying. The use of the subcutaneous formulation can also prevent economic damage caused by loss of the drug remaining in the vial with the use of the intravenous formulation where the dose depends on the body surface area.