Protsenko Marina Valeryevna
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.
To assess the level of development of pharmacoeconomics in the Russian Federation as a branch of science analysis of pharmacoeconomic and clinical economic studies published in the scientific electronic library “eLIBRARY.RU” (RSCI) for the period from 2005 through 2015 was performed. As a result, it was determined that the number of such studies from year to year is steadily increasing. The leader among the countries who publish their researches on the platform of the scientific electronic library “eLIBRARY. RU” was Russia, the second and the third places were shared by Ukraine and Belarus, respectively. The research was carried out in 52 subjects of the Russian Federation, which is represented by 63 Universities. Leader out of them was the I.M. Sechenov First Moscow State Medical University. It turned out that most pharmacoeconomic and clinical-economic research during the specified time interval was devoted to cardiovascular, pulmonary, oncological and endocrinological diseases. As a result of analysis, it was found that among pharmacoeconomic research methods most commonly used “cost– effectiveness” analysis (45,1%). In addition, on the basis of the analyzed data of Russian science citation index and the total number of published works, the rating of the authors who conducted research in the field of pharmacoeconomics in the country.
First Moscow State Medical University I.M. Sechenov (Sechenov University), Moscow, Russia
The article presents updated and advanced results of a pharmacoeconomic study conducted in 2016 on the treatment of patients with pulmonary arterial hypertension II and III functional classes with ambrisentan and bosentan preparations in Russia. The evaluation was provided through cost analysis, “cost minimization” analysis, “cost-utility” analysis, “budget impact” analysis. Еhe cost of pharmacotherapy with Volibrys (ambrisentan) of arterial pulmonary hypertension, regardless of the functional class, was 1 200 000 rubles. and 1 300 000 rubles. respectively, with the need for 12 and 13 packs of the drug per year. The average annual cost of treatment with bosentan ranges from 1 368 900 rubles. up to 1 471 644 rubles. respectively, with the need for 13 and 14 packs of the drug. The results of the “cost minimization” analysis, carried out on the assumption of equal effectiveness of ambrisentan and bosentan, showed that per one patient for one year, the preparation of Volibris (ambrisentan) in comparison with the preparations of bosentan (Traklir and Bozeneks) 175 760 to 178 504 rubles. The values of the “cost-utility” coefficient for ambrisentan in the treatment of pulmonary arterial hypertension II and III of the functional class varied depending on the dosage (5 or 10 mg) from 1,831,563 rubles. up to 2 012 040 rubles for QALY and 2 667 711 rubles up to 2 787 728 rubles. for QALY. At the same time, similar averaged values for bosentan amounted to 2 480 073 rubles and 3 271 174 rubles for QALY for II and III functional class pulmonary arterial hypertension, respectively. The “budget impact” analysis based on the estimated number of patients with pulmonary arterial hypertension in the Russian Federation, which is 2936 people, showed that the transfer of this number of patients treated with the bosentan in the form of original (Tracler) and reproduced (Bozeneks) 1: 1, for treatment with the drug ambrisentan (Volibris), will allow for 2 years to save 853.2 million rubles, which is 9.7% of the budget required for the treatment of bosentan.
Possessing a high prevalence and disability, psoriatic arthritis (PsA) is a serious socio-economic burden for the patient and for society. Therefore, the choice of the PsA treatment strategy requires focusing on not only the prevention and control of the disease but also the effective allocation of the available resources of the healthcare system. Objective. In this regard, the purpose of this study was to conduct a comparative pharmacoeconomic evaluation of biologics: secukinumab (Cosentyx), golimumab (Simponi®), certolizumab pegol (Cimzia®), Ustekinumab (Stelara®), adalimumab (Humira®), etanercept (Enbrel®) and infliximab (Remicade®, Flammegis®, Infliximab produced by local BIOCAD company). Materials and methods. Based on data on the effectiveness of compared treatment regimens, data on the cost of drugs and medical services, as well as the frequency of their provision, an analysis of direct costs, cost-effectiveness analysis, as well as budget impact analysis were carried out. Results. The cost analysis educed that the amount of direct costs for treatment with secukinumab 150 mg for ‘biologic-naive’ patients with active PsA is on average 28% and 40% lower than the cost of treatment with certolizumab pegol, golimumab, ustekinumab, adalimumab, etanercept and infliximab in the first and subsequent years of therapy, in accordance. Estimation of costs and efficacy showed that treatment of PsA with secukinumab 150 mg is characterized by lower costs per unit of effectiveness (ACR 20/50/70 response), relative to the indicators of the compared drugs in the first and subsequent years of therapy. The assumed prescription of secukinumab instead of certolizumab pegol, golimumab, ustekinumab, adalimumab, etanercept, and infliximab (including biosimilar) 19% of patients with active PsA leads to budget savings of 468 million rubles and 1.5 billion rubles for the first and three years of treatment, in accordance, on the calculated patient population. Conclusion. According to the results of the study, the pharmacoeconomic feasibility of using secukinumab 150 mg as a first-line line biologic for the treatment of active PsA has been established.
Wide range of available platforms for phacoemulsification of cataracts and limited financing of the health care system make crucial of optimal decisions in choosing specific models of platforms. In this regard, for the first time in Russia and in the world, an assessment of healthcare technology (HTA) of 6 platforms for cataract phacoemulsification was carried out - Centurion, Infiniti, Stellaris, Stellaris PC, Whitestar Signature, Visalis 500. Our study revealed the limitations of the existing evidence base for comparative purposes evaluations of various phacoemulsification platforms from a health system perspective. Due to this we made an assumption of the use expert assessment of the analized platforms in terms of ultrasound time and the volume of aspirated liquid allowed us to provide HTA, the results of which on the one hand confirm the applicability of the HTA methodology itself for the analysis of the type of medical device under consideration, and with available cost data demonstrated «cost-effectiveness ”of the Centurion platform and its advantage in terms of budget impact analysis among other platforms of the latest generation. The full use of HTA for cataract phacoemulsification platforms requires the creation of an evidence base, taking into account perspective of organizational decision-making process.
Pharmacoeconomic assessment is an obligate element for including a drug in the reimbursement lists – EDL and state federal reimbursement drug list, which in turn represents an important step in ensuring the availability of drug therapy. In this regard, the registration of a new drug of the group of janus kinase inhibitors, baricitinib in Russia, used in the treatment of rheumatoid arthritis in patients with moderate and severe degree of intolerance or lack of adequate treatment for one or more basic antirheumatic drugs, actualized the task of its pharmacoeconomic assessment. The pharmacoeconomic analysis of baricitinib by the cost-effectiveness methods compared with adalimumab and the “budget impact” compared with the entire group of genetically engineered biological products showed that, from the point of view of the cost-effectiveness evaluation, baricitinib is characterized as a dominant alternative, and with the position of the “budget impact” evaluation, the use of baricitinib is accompanied by budget savings. In accordance with the current rules of pharmacoeconomic assessment, the drug baricitinib gains + 9 points and, by decision of the Commission of Ministry of Health, was included in the lists of EDL and state federal reimbursement drug list, approved by Order of the Government of the Russian Federation 2406-R of October 12, 2019.
Budget impact analysis of treatment of ankylosing spondylitis was carried out on the for cohort of 100 patients in the Russian Federation. With the current distribution of patients, adalimumab received 26%, golimumab – 14%, infliximab – 26%, etanercept 18%, certolizumab pegol 7%, secukinumab – 9%. With the simulated distribution of patients, the share of the most economical drug, secukinumab, was increased to 21.8%, while the shares of adalimumab, golimumab, infliximab, certolizumab pegol and etanercept were 21.6%, 13.8%, 13.8%, 5, 8%, 13.8%, respectively. Also, in the simulated distribution, the shares of recently registered drugs – netakimab - 6.8%, and ixekizumab – 2.6% were taken into account. With the current distribution of patients between drugs the budget is 92,868,680 rubles in the first year and 258,825,970 rubles for three years. With a simulated distribution, the costs in the first year are 86,626,248 rubles and 240,444,152 rubles for three years. Thus, when calculating per 100 patients, the simulated distribution of patients in comparison with the current one provides savings in the amount of 6,242,431 rubles in the first year and 18 381 818 rubles for three years. With the saved money, 9 patients can be additionally treated with secukinumab.
The presence in Russia of legally approved rules for the pharmacoeconomic assessment of drugs when considering their inclusion in the lists of vital and essential drugs (reimbursement), as well as in the list of medical nutrition for children with disabilities, has features of pharmacoeconomic analysis of parenteral and enteral nutrition. Since parenteral and enteral nutrition can have different legal status - a drug (MP), biologically active additive (BAA) and even medical products, the rules for pharmacoeconomic assessment at the federal level will be different for them.
The article describes the principles of the international classification of rare diseases, proposed by the organization Orphanet. The supranosological nature of rare diseases predetermined the task of developing their new classification, which is more convenient for coding in the framework of their treatment than ICD-11, but coordinated with it. For these purposes, the international organi- zation Orphanet, formed in France in 1997, together with WHO, is developing an additional classification of rare diseases with the assignment of each noso- logical unit its own unique code based on the collection of information and the formation of its own database of their nomenclature. The Orphanet nomencla- ture includes the following elements: a unique Orpha disease code, a common or most common disease name, synonymous disease names, keywords often used to search for a given disease, if any, and the disease definition itself.
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.
As with any socially significant disease, in chronic heart failure (CHF), the control of diagnostic and treatment, including monitoring the condition of patients in order to maintain optimal therapy, is of decisive importance. The specificity of CHF is the high frequency of repeated hospitalizations for acute decompensation of heart failure (ADHF). One of the key aspects of monitoring the patient’s condition is the assessment of congestion - the identification and determination of the degree of it for the timely optimization of the treatment in order to prevent re-hospitalization. In this regard, of great interest is a new technology for non-invasive measurement of fluid in the lungs - Remote Dielectric Sensing (ReDS), implemented in a medical device under the ReDS Pro brand (manufacturer - Sensible Medical Innovations, Israel). ReDS technology is a quantitative non-invasive method for measuring the total volume of fluid in the lungs in patients with signs of volume overload, including those with CHF. The technology is based on the following principle: low-power electromagnetic radiation passes through the lung tissue from the emitter to the receiver, the assessment of changes in radio wave parameters provides an accurate measurement of the total volume of fluid in the tissue. The result of the examination is a quantitative indicator that reflects the percentage of total fluid in the total volume of the lung. The emergence of a new technology makes its assessment relevant for monitoring treatment and diagnostic measures for CHF in the Russian Federation at the inpatient and outpatient stages of medical care. The described study of health technology assessment was based on the developed model, which, taking into account the clinical advantages of ReDS technology, made it possible to analyze its “budget impact” both at the federal and regional levels from the standpoint of the inpatient or outpatient stages of medical care. The assessment of the economic and clinical feasibility of introducing ReDS tech- nology was carried out separately for the inpatient and outpatient stages (for the outpatient stage, the calculation was carried out both for a model that takes into account the costs of additional visits to patients for scheduled examinations, and without them) in conditions of achieving the technology availability indicator for at least 95% of the population. The results of the assessment showed that at a given level of implementation of ReDS technology at the inpatient level, the number of re-hospitalizations of patients with CHF in the Russian Federation will decrease from 375,703 to 252,208 per year (by 32.9%). The introduction of technologies at the outpatient level will reduce the number of readmissions from 375,703 to 82,782 per year (by 78.0%). This corresponds to a decrease in the annual cost of hospitalizations for CHF (both primary and recurrent) with the introduction of ReDS in hospitals from 24.93 billion rubles to 20.96 billion rubles (decrease by 15.9%); when implementing the method at the outpatient level from 24.93 billion rubles to 15.52 billion rubles (decrease by 37.8%), and taking into account the cost of additional visits to patients at the outpatient level from 24.93 billion rubles to 19.70 billion rubles (decrease by 21.0%). At the same time, one-time costs for the purchase of a ReDS medical device in outpatient and inpatient clinics amount to 12.06 billion rubles and 11.04 billion rubles respectively. The results of the “budget impact” analysis (taking into account the discount factor of 3.5%) of the introduction of ReDS technology at the outpatient level demonstrate that over 7 years it provides savings in the amount of 20,335,833,423 rubles (including costs for additional visits) up to 45,926,532,070 rubles (excluding the cost of additional visits), and at the inpatient level - 13,613,327,627 rubles. Evaluation of the introduction of ReDS technology into the Russian healthcare system at the federal level, both at the outpatient and inpatient stages of medical care, with a total need for a ReDS medical device in the range of 1129-1234 units, providing coverage of more than 95% of the population with this technology, showed its clinical and economic benefit, which is expressed in a decrease in the number of readmissions and net savings in the health care system already during the third year of using the ReDS technology.
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.
To assess the level of development of pharmacoeconomics in the Russian Federation as a branch of science analysis of pharmacoeconomic and clinical economic studies published in the scientific electronic library “eLIBRARY.RU” (RSCI) for the period from 2005 through 2015 was performed. As a result, it was determined that the number of such studies from year to year is steadily increasing. The leader among the countries who publish their researches on the platform of the scientific electronic library “eLIBRARY. RU” was Russia, the second and the third places were shared by Ukraine and Belarus, respectively. The research was carried out in 52 subjects of the Russian Federation, which is represented by 63 Universities. Leader out of them was the I.M. Sechenov First Moscow State Medical University. It turned out that most pharmacoeconomic and clinical-economic research during the specified time interval was devoted to cardiovascular, pulmonary, oncological and endocrinological diseases. As a result of analysis, it was found that among pharmacoeconomic research methods most commonly used “cost– effectiveness” analysis (45,1%). In addition, on the basis of the analyzed data of Russian science citation index and the total number of published works, the rating of the authors who conducted research in the field of pharmacoeconomics in the country.
First Moscow State Medical University I.M. Sechenov (Sechenov University), Moscow, Russia
The article presents updated and advanced results of a pharmacoeconomic study conducted in 2016 on the treatment of patients with pulmonary arterial hypertension II and III functional classes with ambrisentan and bosentan preparations in Russia. The evaluation was provided through cost analysis, “cost minimization” analysis, “cost-utility” analysis, “budget impact” analysis. Еhe cost of pharmacotherapy with Volibrys (ambrisentan) of arterial pulmonary hypertension, regardless of the functional class, was 1 200 000 rubles. and 1 300 000 rubles. respectively, with the need for 12 and 13 packs of the drug per year. The average annual cost of treatment with bosentan ranges from 1 368 900 rubles. up to 1 471 644 rubles. respectively, with the need for 13 and 14 packs of the drug. The results of the “cost minimization” analysis, carried out on the assumption of equal effectiveness of ambrisentan and bosentan, showed that per one patient for one year, the preparation of Volibris (ambrisentan) in comparison with the preparations of bosentan (Traklir and Bozeneks) 175 760 to 178 504 rubles. The values of the “cost-utility” coefficient for ambrisentan in the treatment of pulmonary arterial hypertension II and III of the functional class varied depending on the dosage (5 or 10 mg) from 1,831,563 rubles. up to 2 012 040 rubles for QALY and 2 667 711 rubles up to 2 787 728 rubles. for QALY. At the same time, similar averaged values for bosentan amounted to 2 480 073 rubles and 3 271 174 rubles for QALY for II and III functional class pulmonary arterial hypertension, respectively. The “budget impact” analysis based on the estimated number of patients with pulmonary arterial hypertension in the Russian Federation, which is 2936 people, showed that the transfer of this number of patients treated with the bosentan in the form of original (Tracler) and reproduced (Bozeneks) 1: 1, for treatment with the drug ambrisentan (Volibris), will allow for 2 years to save 853.2 million rubles, which is 9.7% of the budget required for the treatment of bosentan.
Possessing a high prevalence and disability, psoriatic arthritis (PsA) is a serious socio-economic burden for the patient and for society. Therefore, the choice of the PsA treatment strategy requires focusing on not only the prevention and control of the disease but also the effective allocation of the available resources of the healthcare system. Objective. In this regard, the purpose of this study was to conduct a comparative pharmacoeconomic evaluation of biologics: secukinumab (Cosentyx), golimumab (Simponi®), certolizumab pegol (Cimzia®), Ustekinumab (Stelara®), adalimumab (Humira®), etanercept (Enbrel®) and infliximab (Remicade®, Flammegis®, Infliximab produced by local BIOCAD company). Materials and methods. Based on data on the effectiveness of compared treatment regimens, data on the cost of drugs and medical services, as well as the frequency of their provision, an analysis of direct costs, cost-effectiveness analysis, as well as budget impact analysis were carried out. Results. The cost analysis educed that the amount of direct costs for treatment with secukinumab 150 mg for ‘biologic-naive’ patients with active PsA is on average 28% and 40% lower than the cost of treatment with certolizumab pegol, golimumab, ustekinumab, adalimumab, etanercept and infliximab in the first and subsequent years of therapy, in accordance. Estimation of costs and efficacy showed that treatment of PsA with secukinumab 150 mg is characterized by lower costs per unit of effectiveness (ACR 20/50/70 response), relative to the indicators of the compared drugs in the first and subsequent years of therapy. The assumed prescription of secukinumab instead of certolizumab pegol, golimumab, ustekinumab, adalimumab, etanercept, and infliximab (including biosimilar) 19% of patients with active PsA leads to budget savings of 468 million rubles and 1.5 billion rubles for the first and three years of treatment, in accordance, on the calculated patient population. Conclusion. According to the results of the study, the pharmacoeconomic feasibility of using secukinumab 150 mg as a first-line line biologic for the treatment of active PsA has been established.
Wide range of available platforms for phacoemulsification of cataracts and limited financing of the health care system make crucial of optimal decisions in choosing specific models of platforms. In this regard, for the first time in Russia and in the world, an assessment of healthcare technology (HTA) of 6 platforms for cataract phacoemulsification was carried out - Centurion, Infiniti, Stellaris, Stellaris PC, Whitestar Signature, Visalis 500. Our study revealed the limitations of the existing evidence base for comparative purposes evaluations of various phacoemulsification platforms from a health system perspective. Due to this we made an assumption of the use expert assessment of the analized platforms in terms of ultrasound time and the volume of aspirated liquid allowed us to provide HTA, the results of which on the one hand confirm the applicability of the HTA methodology itself for the analysis of the type of medical device under consideration, and with available cost data demonstrated «cost-effectiveness ”of the Centurion platform and its advantage in terms of budget impact analysis among other platforms of the latest generation. The full use of HTA for cataract phacoemulsification platforms requires the creation of an evidence base, taking into account perspective of organizational decision-making process.
Pharmacoeconomic assessment is an obligate element for including a drug in the reimbursement lists – EDL and state federal reimbursement drug list, which in turn represents an important step in ensuring the availability of drug therapy. In this regard, the registration of a new drug of the group of janus kinase inhibitors, baricitinib in Russia, used in the treatment of rheumatoid arthritis in patients with moderate and severe degree of intolerance or lack of adequate treatment for one or more basic antirheumatic drugs, actualized the task of its pharmacoeconomic assessment. The pharmacoeconomic analysis of baricitinib by the cost-effectiveness methods compared with adalimumab and the “budget impact” compared with the entire group of genetically engineered biological products showed that, from the point of view of the cost-effectiveness evaluation, baricitinib is characterized as a dominant alternative, and with the position of the “budget impact” evaluation, the use of baricitinib is accompanied by budget savings. In accordance with the current rules of pharmacoeconomic assessment, the drug baricitinib gains + 9 points and, by decision of the Commission of Ministry of Health, was included in the lists of EDL and state federal reimbursement drug list, approved by Order of the Government of the Russian Federation 2406-R of October 12, 2019.
Budget impact analysis of treatment of ankylosing spondylitis was carried out on the for cohort of 100 patients in the Russian Federation. With the current distribution of patients, adalimumab received 26%, golimumab – 14%, infliximab – 26%, etanercept 18%, certolizumab pegol 7%, secukinumab – 9%. With the simulated distribution of patients, the share of the most economical drug, secukinumab, was increased to 21.8%, while the shares of adalimumab, golimumab, infliximab, certolizumab pegol and etanercept were 21.6%, 13.8%, 13.8%, 5, 8%, 13.8%, respectively. Also, in the simulated distribution, the shares of recently registered drugs – netakimab - 6.8%, and ixekizumab – 2.6% were taken into account. With the current distribution of patients between drugs the budget is 92,868,680 rubles in the first year and 258,825,970 rubles for three years. With a simulated distribution, the costs in the first year are 86,626,248 rubles and 240,444,152 rubles for three years. Thus, when calculating per 100 patients, the simulated distribution of patients in comparison with the current one provides savings in the amount of 6,242,431 rubles in the first year and 18 381 818 rubles for three years. With the saved money, 9 patients can be additionally treated with secukinumab.
The presence in Russia of legally approved rules for the pharmacoeconomic assessment of drugs when considering their inclusion in the lists of vital and essential drugs (reimbursement), as well as in the list of medical nutrition for children with disabilities, has features of pharmacoeconomic analysis of parenteral and enteral nutrition. Since parenteral and enteral nutrition can have different legal status - a drug (MP), biologically active additive (BAA) and even medical products, the rules for pharmacoeconomic assessment at the federal level will be different for them.
The article describes the principles of the international classification of rare diseases, proposed by the organization Orphanet. The supranosological nature of rare diseases predetermined the task of developing their new classification, which is more convenient for coding in the framework of their treatment than ICD-11, but coordinated with it. For these purposes, the international organi- zation Orphanet, formed in France in 1997, together with WHO, is developing an additional classification of rare diseases with the assignment of each noso- logical unit its own unique code based on the collection of information and the formation of its own database of their nomenclature. The Orphanet nomencla- ture includes the following elements: a unique Orpha disease code, a common or most common disease name, synonymous disease names, keywords often used to search for a given disease, if any, and the disease definition itself.
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.
As with any socially significant disease, in chronic heart failure (CHF), the control of diagnostic and treatment, including monitoring the condition of patients in order to maintain optimal therapy, is of decisive importance. The specificity of CHF is the high frequency of repeated hospitalizations for acute decompensation of heart failure (ADHF). One of the key aspects of monitoring the patient’s condition is the assessment of congestion - the identification and determination of the degree of it for the timely optimization of the treatment in order to prevent re-hospitalization. In this regard, of great interest is a new technology for non-invasive measurement of fluid in the lungs - Remote Dielectric Sensing (ReDS), implemented in a medical device under the ReDS Pro brand (manufacturer - Sensible Medical Innovations, Israel). ReDS technology is a quantitative non-invasive method for measuring the total volume of fluid in the lungs in patients with signs of volume overload, including those with CHF. The technology is based on the following principle: low-power electromagnetic radiation passes through the lung tissue from the emitter to the receiver, the assessment of changes in radio wave parameters provides an accurate measurement of the total volume of fluid in the tissue. The result of the examination is a quantitative indicator that reflects the percentage of total fluid in the total volume of the lung. The emergence of a new technology makes its assessment relevant for monitoring treatment and diagnostic measures for CHF in the Russian Federation at the inpatient and outpatient stages of medical care. The described study of health technology assessment was based on the developed model, which, taking into account the clinical advantages of ReDS technology, made it possible to analyze its “budget impact” both at the federal and regional levels from the standpoint of the inpatient or outpatient stages of medical care. The assessment of the economic and clinical feasibility of introducing ReDS tech- nology was carried out separately for the inpatient and outpatient stages (for the outpatient stage, the calculation was carried out both for a model that takes into account the costs of additional visits to patients for scheduled examinations, and without them) in conditions of achieving the technology availability indicator for at least 95% of the population. The results of the assessment showed that at a given level of implementation of ReDS technology at the inpatient level, the number of re-hospitalizations of patients with CHF in the Russian Federation will decrease from 375,703 to 252,208 per year (by 32.9%). The introduction of technologies at the outpatient level will reduce the number of readmissions from 375,703 to 82,782 per year (by 78.0%). This corresponds to a decrease in the annual cost of hospitalizations for CHF (both primary and recurrent) with the introduction of ReDS in hospitals from 24.93 billion rubles to 20.96 billion rubles (decrease by 15.9%); when implementing the method at the outpatient level from 24.93 billion rubles to 15.52 billion rubles (decrease by 37.8%), and taking into account the cost of additional visits to patients at the outpatient level from 24.93 billion rubles to 19.70 billion rubles (decrease by 21.0%). At the same time, one-time costs for the purchase of a ReDS medical device in outpatient and inpatient clinics amount to 12.06 billion rubles and 11.04 billion rubles respectively. The results of the “budget impact” analysis (taking into account the discount factor of 3.5%) of the introduction of ReDS technology at the outpatient level demonstrate that over 7 years it provides savings in the amount of 20,335,833,423 rubles (including costs for additional visits) up to 45,926,532,070 rubles (excluding the cost of additional visits), and at the inpatient level - 13,613,327,627 rubles. Evaluation of the introduction of ReDS technology into the Russian healthcare system at the federal level, both at the outpatient and inpatient stages of medical care, with a total need for a ReDS medical device in the range of 1129-1234 units, providing coverage of more than 95% of the population with this technology, showed its clinical and economic benefit, which is expressed in a decrease in the number of readmissions and net savings in the health care system already during the third year of using the ReDS technology.
