PHARMACOECONOMICS. Modern pharmacoeconomics and pharmacoepidemiology

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Vol 11, No 3 (2018)
View or download the full issue PDF (Russian)

Original Article

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In the article, we analyze the current version of Government Regulation No. 871 where the principles of health technologies assessment (HTA) and the reimbursement strategies in Russia have been put forward. We conclude that the HTA methodology in Russia is consistent with the multi-criteria decision analysis. Recommendations on the improvement of the assessment methodology in Regulation No. 871 are provided.
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The aim is to characterize the use of medications in pediatric inpatients with community-acquired pneumonia.

Materials and methods. Medical records of 547 children treated for community-acquired pneumonia in 4 medical organizations in St. Petersburg over 2015-2017 were analyzed.

The results of this retrospective study (N = 547) showed that children aged 1 to 3 years were most vulnerable (31.44%). Along with that, 45.70% of  patients had concomitant diseases; among those, acute respiratory infections of the upper respiratory tract prevailed (32.20%). As for the medications, the category J drugs “Antimicrobials of systemic action” were most commonly used (30.30%) and contributed the largest part in the total drug costs (80.22%). Specifically, the greatest contribution was made by the J01D group «Beta-lactam antibacterial drugs, other» (64.54%); their cost took 77.22% of the expenses and covered 48.97% of the drug list. 

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The aim was to develop a methodology for determining the willingness to pay threshold (WTPT) and its upper limit value within the Russian health care system.

Materials and methods. WTPT was calculated based on the shadow budget price (i. e. determining the WTPT by the suppling party). This method is an empirical assessment of the cost-effectiveness threshold that reflects the utmost productivity of the health care system, as determined from the relationship between changes in healthcare expenditure and health outcomes achieved. The state’s willingness to pay for improving their citizens’ healthcare was evaluated considering the population of the Russian Federation, mortality and life expectancy in different age and gender groups, as well as the volume of government spending. The cost of disability-adjusted life-year prevented (DALY) and the cost of quality-adjusted life-year saved (QALY) were determined by the suppling party, that is, they reflect the cost the state is willing to pay for improving the health of their population under conditions of limited budget. The described approach considers the performance of the country’s healthcare system over a certain period and the costs incurred in functioning of the system.

Results. As part of this study, it was found that the cost of one additionally prevented DALY would be 313,878.21 rubles, and the cost of one additionally saved QALY – 365,060.31 rubles.

Conclusion. The WTPT for medical technologies in the Russian Federation, determined by estimating the shadow budget price will amount to 313,878.21 rubles for one prevented DALY and 365 060,31 rubles for one saved QALY. With regard to clinical and economic analysis, medical technologies with the incremental cost-effectiveness indicator not exceeding the one calculated in this study can be seen as cost-effective. The obtained threshold value is a recommendation. A medical technology can be approved even with a WTPT higher than the recommended level, because this specific technology may have additional  advantages other than WTPT when compared with the reference technologies. 

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The aim is to study the clinical and economic aspects of using belimumab for the treatment of systemic lupus erythematosus (SLe) in patients with high SLe activity, the presence of anti-DNA, a low level of complement in the blood plasma and minimal lupus organ damage at the early stage.

Materials and methods. The Markov model of SLe is used. The initial probabilities of the Markov transitions are obtained from analysis of published clinical and observational studies. The prices of the relevant drugs were obtained from the VeD price registry. Medical and nonmedical expenses are calculated on the basis of the current standards for financial costs and coefficients, as well as payments for temporary and permanent disability. Indirect costs are calculated using the method of human capital.

Results. It has been found that using belimumab leads to a decrease in costs (both direct and indirect), as well as an increase in patient survival. The total costs for belimumab vs standard therapy approximated 8.84 million rubles vs 17.72 million rubles (of which 4.05 million rubles vs 5.58 million rubles were direct costs, and 2.57 million rubles vs 2.88 million rubles were indirect medical costs) on average per patient in the belimumab group vs standard therapy group, respectively.

Conclusion. The use of belimumab in the target population of patients is pharmacoeconomically justified and expedient because it leads to increased treatment efficiency and reduction of direct and indirect costs. 

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Rationale. From 2014 to 2017, the portion of antineoplastic drugs dossiers submitted to reimbursement in Russia has grown from 15% to 28%. This group of drugs is characterized by severe adverse events (Aes). The question whether drug safety is taken into consideration by decision makers is still open.

Aim: To evaluate the role of drug safety in making the decision on reimbursement.

Materials and methods. The data were taken from the reports of expert committees concerning the dossiers submitted between 2014 and 2016. The year of submission, the international drug name, the total safety score and the final decision of the committee were entered into our database. Parametric and non-parametric statistics were used to calculate the difference between the mean safety scores plotted by years and by inclusion/noninclusion into VeD lists.

Results. The mean safety score for all drugs of this group was -4,67 (95% CI from -5,04 to -4,29); for the drugs included into VeD lists it was -4,05 (95% CI from -4,68 to -3,42), that was significantly higher (p = 0,01) compared to the non-included medications (-5,03; 95% CI from -5,49 to -4,58). The mean safety scores year-by-year for the non-included drugs were lower than those for the VeD-included drugs, but the difference reached significance only in 2015 (p = 0,01). The safety scores were mainly based on Aes of type A.

Conclusion. our analysis shows that the drug safety data play a role in the decision making on reimbursement and inclusion/noninclusion into VeD lists. Yet the total safety score contributes much less than other criteria in the decision making process. The safety analysis is usually based on Aes of type A, whereas the data on Aes of types C and D are insufficient. 

Review articles

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Aim: To analyze the efficacy, safety and pharmacoeconomic aspects of using ocrelizumab in adult patients with relapsing/remitting multiple sclerosis (R/R MS).

Materials and Methods. We used the commonly accepted PICo(S) questionnaire with the following specifics: the population – patients with R/R MS; the intervention – ocrelizumab; the comparators – all disease-modifying treatments for MS; the outcomes – the annualized relapse rate, confirmed disability progression, MRI results, quality-adjusted years of survival (QALYs), adverse events, and other clinical outcomes. The search for the relevant information was conducted in 2018 by using the embase, PubMed, Cochrane and databases and the «ocrelizumab» AND «multiple sclerosis» keywords. The levels of evidence and conclusiveness of the cited studies were also assessed.

Results. Treatments with ocrelizumab resulted in a lower rate of disease progression as compared with interferon β -1a. As evidenced by a randomized clinical trial, the annualized relapse rate estimated after 96 weeks was lower with ocrelizumab than that with interferon β-1a (0.16 vs. 0.29, 47% decrease, p<0.001). For most secondary end points, patients on ocrelizumab showed better outcomes than those on interferon β-1a. In the ocrelizumab group, the most common adverse events were caused by reactions to the drug infusion, nasopharyngitis, upper respiratory and urinary tract infections, and headaches. No cases of progressive multifocal leukoencephalopathy have been reported so far. ocrelizumab is more clinically effective than the first-line disease-modifying therapies; this conclusion also refers to patients with the aggressive (highly active) form of MS. ocrelizumab showed the efficacy similar to the second-line disease-modifying therapies, but it had a more favorable safety profile. The pharmacoeconomic indices showed that using ocrelizumab had a positive impact on the budget in the long-term perspective.

Conclusions. ocrelizumab can be considered as the main treatment alternative for patients with highly active MS and patients with a high risk of progressive multifocal leukoencephalopathy. However, an additional assessment of the risk caused by rare adverse events is needed. 

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The aim of this review is to analyze reasons for the high cost of recombinant human trypsin, technological and economic obstacles limiting trypsin production and implementation, as well as practical means to solve these problems.

Materials and methods. The properties of human trypsin, the recombinant technology for its production, the marketing aspects and the prospective of enzyme therapy are addressed in this review that contains 44 references in Russian and english. Particular attention is paid to the methods that can boost the production of recombinant trypsin.

Results. Trypsin purified from the mammalian pancreas has been used for over 80 years in the enzyme therapy in various diseases. Genetically engineered human trypsin is proposed to be an innovative, safe and effective therapeutic protease. However the medical use of recombinant trypsin is slowed by its very high price and insufficient production. There is a need for novel biopharmaceutical technologies, as well optimized up-stream and down-stream processes to increase the yield of active recombinant trypsin and significantly reduce the production costs. Recombinant human trypsin that is priced similar to its animal analogues is preferable in all types of enzyme therapy.

Conclusion. Innovative biopharmaceutical technologies are expected to significantly reduce the production costs of recombinant human trypsin and stimulate its wider use in enzyme therapy and also in production of other therapeutic proteins.

ISSN 2070-4909 (Print)
ISSN 2070-4933 (Online)