Kulikov Andrey Yurievich
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.
The global pharmacoeconomic model of cost-effectiveness analysis of insulin degludec (Tresiba®) use in comparison with insulin glargine U300 in the treatment of type 2 diabetes mellitus, was validated. As the result, it was found that the model is relevant to local clinical practice, fully complies with the pharmacoeconomics methodology, takes into account the entire spectrum of costs relevant for the pharmacoeconomic evaluation of insulin in Russia, and the computations built in it is correct. Based on the actual cost data entered, the model calculated the ICER of degludec insulin, which was 214,714 rubles, which does not exceed the sum of three GDP per capita – 2,184,562 rubles and corresponds to the willingness to pay threshold in the Russian Federation. Thus, the insulin degludec (Tresiba®) can be considered as cost-effective from the standpoint of the conducted pharmacoeconomic analysis.
We conducted an budget impact analysis was made of the treatment of spinal muscular atrophy (SMA) with drugs Nusinersen and Risdiplam. Its results showed that from the point of view of the organization of drug provision for patients with SMA at current prices, treatment of patients under 18 years of age within the framework of the responsibility of the Circle of Kindness Foundation, given the current distribution of patients, in which 57.6% receive Nusinersen, and 42.4% Risdiplam, is characterized by a smaller budget already in the first year – 18.82 billion rubles, in comparison with the budget of the simulated distribution, in which 42.4% of patients receive Nusinersen, and 57.6% of Risdiplam – 19.27 billion rubles. Thus, with the current distribution over 5 years, budget savings in comparison with the simulated distribution reaches 3.24 billion rubles, excluding the discount factor, and 2.86 billion rubles, when discounting. Analyzing the long-term effect of the budget impact of the use of Nusinersen drugs per patient at the beginning of therapy at the age of two months, it was found that the total savings before the patient switches to provision through regional funding (reaching 18 years of age) can reach 59.72 million. rub. From the point of view of drug provision for patients with SMA at the regional level, that is, patients over 18 years old, the main pool of patients will be formed due to the successive transfer of patients from the Circle of Kindness Fund, since most of the patients (1039 people) are now provided precisely for the account of the Circle of Kindness Fund, and upon reaching the age of 18, this cohort of patients will be transferred to provision at the expense of the budgets of the regions of the Russian Federation. At the same time, it is noted that the costs of initiation of these patients with Nusinersen were covered by the Circle of Kindness Foundation, and therefore, from the point of view of the regions of the Russian Federation, savings on Nusinersen in comparison with Risdiplam will be noted already in the first year of therapy, based on the fact that the annual costs for Nusinersen maintenance mode are 16,957,680 rubles. per patient, and the annual cost of treatment with Risdiplam drug for one adult patient (weighing more than 20 kg) exceeds 20 million rubles. Thus, the stated results of the budget impact analysis revealed the economic feasibility of Nusinersen in the treatment of SMA in comparison with the Risdiplam.
In order to preserve the health of elderly patients, methodological approach- es to the pharmacoeconomic substantiation of the inclusion of drugs for the treatment of dyslipidemia in the list of vital and essential drugs (EDL) were adapted and an appropriate assessment of the drug of cholesterol absorption inhibition was carried out, which showed that from the point of view of the healthcare system, the presence of this drug in the list will have a positive effect: Impact on the budget by increasing the availability of this drug for patients and delaying the prescription of expensive drugs of the PCSK9 inhibitor class. From a clinical point of view, increasing the availability of cholesterol absorption inhibition drugs for patients when it is included in the list of essential drugs will ensure that the correct sequence of the therapeutic line is followed in the treatment of dyslipidemia.
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.
The global pharmacoeconomic model of cost-effectiveness analysis of insulin degludec (Tresiba®) use in comparison with insulin glargine U300 in the treatment of type 2 diabetes mellitus, was validated. As the result, it was found that the model is relevant to local clinical practice, fully complies with the pharmacoeconomics methodology, takes into account the entire spectrum of costs relevant for the pharmacoeconomic evaluation of insulin in Russia, and the computations built in it is correct. Based on the actual cost data entered, the model calculated the ICER of degludec insulin, which was 214,714 rubles, which does not exceed the sum of three GDP per capita – 2,184,562 rubles and corresponds to the willingness to pay threshold in the Russian Federation. Thus, the insulin degludec (Tresiba®) can be considered as cost-effective from the standpoint of the conducted pharmacoeconomic analysis.
We conducted an budget impact analysis was made of the treatment of spinal muscular atrophy (SMA) with drugs Nusinersen and Risdiplam. Its results showed that from the point of view of the organization of drug provision for patients with SMA at current prices, treatment of patients under 18 years of age within the framework of the responsibility of the Circle of Kindness Foundation, given the current distribution of patients, in which 57.6% receive Nusinersen, and 42.4% Risdiplam, is characterized by a smaller budget already in the first year – 18.82 billion rubles, in comparison with the budget of the simulated distribution, in which 42.4% of patients receive Nusinersen, and 57.6% of Risdiplam – 19.27 billion rubles. Thus, with the current distribution over 5 years, budget savings in comparison with the simulated distribution reaches 3.24 billion rubles, excluding the discount factor, and 2.86 billion rubles, when discounting. Analyzing the long-term effect of the budget impact of the use of Nusinersen drugs per patient at the beginning of therapy at the age of two months, it was found that the total savings before the patient switches to provision through regional funding (reaching 18 years of age) can reach 59.72 million. rub. From the point of view of drug provision for patients with SMA at the regional level, that is, patients over 18 years old, the main pool of patients will be formed due to the successive transfer of patients from the Circle of Kindness Fund, since most of the patients (1039 people) are now provided precisely for the account of the Circle of Kindness Fund, and upon reaching the age of 18, this cohort of patients will be transferred to provision at the expense of the budgets of the regions of the Russian Federation. At the same time, it is noted that the costs of initiation of these patients with Nusinersen were covered by the Circle of Kindness Foundation, and therefore, from the point of view of the regions of the Russian Federation, savings on Nusinersen in comparison with Risdiplam will be noted already in the first year of therapy, based on the fact that the annual costs for Nusinersen maintenance mode are 16,957,680 rubles. per patient, and the annual cost of treatment with Risdiplam drug for one adult patient (weighing more than 20 kg) exceeds 20 million rubles. Thus, the stated results of the budget impact analysis revealed the economic feasibility of Nusinersen in the treatment of SMA in comparison with the Risdiplam.
In order to preserve the health of elderly patients, methodological approach- es to the pharmacoeconomic substantiation of the inclusion of drugs for the treatment of dyslipidemia in the list of vital and essential drugs (EDL) were adapted and an appropriate assessment of the drug of cholesterol absorption inhibition was carried out, which showed that from the point of view of the healthcare system, the presence of this drug in the list will have a positive effect: Impact on the budget by increasing the availability of this drug for patients and delaying the prescription of expensive drugs of the PCSK9 inhibitor class. From a clinical point of view, increasing the availability of cholesterol absorption inhibition drugs for patients when it is included in the list of essential drugs will ensure that the correct sequence of the therapeutic line is followed in the treatment of dyslipidemia.
