Features of the organization of the provision of medicines for the treatment of orphan diseases in the Russian Federation

2020 ◽  
Vol 23 (1) ◽  
pp. 22
Author(s):  
R.U. Khabriev ◽  
A.V. Grechko ◽  
V.S. Malichenko ◽  
Yu.N. Kashevarova ◽  
N.S. Volkova
Keyword(s):  
Russian Federation ◽  
Orphan Diseases ◽  
The Russian Federation

Comparison and analysis of the fda approved orphane drugs registerand the vital and essential drugs list of the Russian Federation

2020 ◽  
pp. 4-16
Author(s):  
D.S. Yurochkin ◽  
◽  
A.A. Leshkevich ◽  
Z.M. Golant ◽  
I.A. NarkevichSaint ◽  
...  
Keyword(s):  
United States ◽  
Russian Federation ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
The United States ◽  
Orphan Diseases ◽  
Essential Drugs ◽  
The Russian Federation ◽  
Comparison And Analysis ◽  
The Government

The article presents the results of a comparison of the Orphan Drugs Register approved for use in the United States and the 2020 Vital and Essential Drugs List approved on October 12, 2019 by Order of the Government of the Russian Federation No. 2406-r. The comparison identified 305 international non-proprietary names relating to the main and/or auxiliary therapy for rare diseases. The analysis of the market of drugs included in the Vital and Essential Drugs List, which can be used to treat rare (orphan) diseases in Russia was conducted.

Study of the availability of gene therapeutic drugs in the Russian Federation

2021 ◽  
pp. 69-75
Author(s):  
Konstantin A. Koshechkin ◽  
Philip A. Romanov ◽  
Alexander A. Mokhov ◽  
Alexander L. Khokhlov
Keyword(s):  
Gene Therapy ◽  
Russian Federation ◽  
World Literature ◽  
World Market ◽  
European Market ◽  
Orphan Diseases ◽  
Therapeutic Drugs ◽  
The World ◽  
The Usa ◽  
The Russian Federation

The authors studied the availability of gene therapy drugs in the Russian Federation on the basis of information on the permission of the medical use of drugs of this group in the world. Literature data and information about medicines approved by the FDA, EMA and the Ministry of Health of Russia were used. In general, the FDA registered only 13 drugs (46%) of the total approved for medical use in the world, 2 of them have already been withdrawn from the market, and 2 additional clinical trials are underway. In Europe, the EMA has approved 16 drugs for medical use (57%), with 4 of them already withdrawn. Most of the drugs were first approved by the FDA, and then, on average, a year later, were approved in the European market. A total of 4 drugs were approved in the European market and were not approved by the FDA at the time the data was requested. And only 1 drug, approved in the USA, is not registered in Europe. In the Russian Federation, two medicines are allowed, Neovasculgen (2011) and Spinraza (2019). This is only 7% of the total number of gene therapy drugs on the world market. Most of the drugs are intended for the treatment of orphan diseases and are cost expensive. This can explain the unevenness of their distribution across regions.

PREVALENCE AND DIAGNOSIS OF RARE (ORPHAN) DISEASES IN PEDIATRIC POPULATION OF THE RUSSIAN FEDERATION

2020 ◽  
Vol 99 (4) ◽  
pp. 229-237
Author(s):  
T.P. Vasilieva ◽  
◽  
R.A. Zinchenko ◽  
I.A. Komarov ◽  
E.Yu. Krasilnikova ◽  
...  
Keyword(s):  
Russian Federation ◽  
Pediatric Population ◽  
Orphan Diseases ◽  
The Russian Federation

scholarly journals Analysis of the prevalence of rare (orphan) diseases («List 24») and the dynamics of detection of children's patients in the subjects of the Russian Federation

2020 ◽  
pp. 25-26
Author(s):  
И.А. Комаров ◽  
Е.Ю. Красильникова ◽  
О.Ю. Александрова ◽  
Р.А. Зинченко ◽  
С.И. Куцев
Keyword(s):  
Russian Federation ◽  
Orphan Diseases ◽  
Annual Increase ◽  
Federal Register ◽  
The Russian Federation ◽  
Low Prevalence ◽  
Timely Treatment

Проведен анализ распространенности редких (орфанных) заболеваний («Перечень 24») и динамики выявляемости пациентов детского возраста в 85 регионах РФ в период 2013-2019 гг. Показан ежегодный прирост пациентов в Федеральном регистре (2013 г. - 4962 больных, 2014 г. - 6761, 2015 г. - 7038, 2016 г. - 7625, 2017 г. - 8245, 2018 г. - 8639, 2019 г. - 9088). Динамика доли детей с заболеваниями из «Перечня 24» среди всех больных колебалась в небольших пределах (в среднем 50%). Большинством заболеваний (16 из 24) болеют в основном дети, но они встречаются также у взрослых. Распространенность заболеваний значительно варьировала между субъектами РФ. Улучшение информированности, а также ведение Федерального регистра способствуют эффективной выявляемости и своевременному назначению терапии в детском возрасте. Однако низкие значения распространенности заболеваний, проявляющихся до 1-го года жизни, говорят о сложностях их диагностики и недостаточной выявляемости. The analysis of the prevalence of rare (orphan) diseases («List of 24») and the dynamics of detection of children’s patients in 85 subjects of the Russian Federation in the period 2013-2019 showed an annual increase in patients in the Federal register (2013 - 4962 patients, 2014 - 6761, 2015 - 7038, 2016 - 7625, 2017 - 8245, 2018 - 8639, 2019 - 9088). The dynamics of the share of children with diseases from the “List of 24” among all patients fluctuated within small limits (on average 50%). Most of the diseases (16 out of 24) are mostly between children, but they also occur in adults. The prevalence of diseases varied significantly between the subjects of the Russian Federation. Improving awareness, as well as maintaining a Federal register promote effective detection and timely treatment in children. However, the low prevalence of diseases that occur before the 1st year of life indicates the complexity of their diagnosis and lack of detection.

Current problems of providing medical care for patients with orphan diseases in the Russian Federation

2020 ◽  
pp. 13-20
Author(s):  
А.Я. Гайдук ◽  
Я.В. Власов ◽  
Е.Ю. Захарова
Keyword(s):  
Medical Care ◽  
Russian Federation ◽  
Models Of Care ◽  
Diagnostic Methods ◽  
Orphan Diseases ◽  
Screening Programs ◽  
Medical Environment ◽  
Lack Of Information ◽  
Life Threatening ◽  
The Russian Federation

В статье обобщен опыт помощи пациентам с редкими заболеваниями, история развития «орфанной» медицины, проводится обзор зарубежных стратегий и основополагающих документов, формирующих современные модели оказания помощи пациентам с орфанными заболеваниями. Освещаются проблемы оказания медицинской помощи данной группе пациентов в Российской Федерации, в том числе в рамках программ «14 высокозатратных нозологий» и «17 нозологий» (17 жизнеугрожающих хронических прогрессирующих редких (орфанных) заболеваний), анализируются механизмы финансирования лекарственного обеспечения, их правовые основы, а также роль медико-генетической службы, как ключевого элемента в системе оказания медицинской помощи пациентам с редкими болезнями. В работе также приведен анализ региональных российских и зарубежных скрининг-программ, обзор актуальных диагностических методов и опыта их внедрения и использования. Авторы также затрагивают вопросы, касающиеся биотехнологических инноваций в российской и зарубежной медицине, подчёркивая актуальность, эффективность и, в некоторых случаях, незаменимость генной терапии. В заключение авторы резюмируют наиболее значимые задачи, стоящие перед орфанной медициной в Российской Федерации, а также вносят предложения по их решению. The article summarizes the world experience in helping patients with rare diseases, the history of the development of “orphan” medicine, reviews foreign strategies and the fundamental documents that form the modern foreign models of care for patients with orphan diseases. The problems of providing medical care to this group of patients in the Russian Federation are highlighted, including in the framework of the programs “14 high-cost nosologies” and “17 nosologies” (17 life-threatening chronic progressive rare (orphan) diseases). The difficulties associated with the operation of the existing system of medical care for orphan patients are highlighted, including data on assessing the lack of information on management and on the procedure for providing information about patients with rare nosologies to the appropriate structures in the medical environment. The work also analyzes regional Russian and foreign screening programs, a review of relevant diagnostic methods and the experience of their implementation and use. The authors also address issues related to biotechnological innovations in Russian and foreign medicine, emphasizing the relevance, effectiveness and, in some cases, the indispensability of gene therapy. In conclusion, the authors summarize the most significant issues and challenges facing orphan medicine in the Russian Federation, as well as make suggestions for their solution.

scholarly journals Drug demand satisfaction of patients with idiopathic pulmonary arterial hypertension

2019 ◽  
pp. 18-22
Author(s):  
E. Yu. Krasilnikova ◽  
I. A. Komarov
Keyword(s):  
Pulmonary Arterial Hypertension ◽  
Arterial Hypertension ◽  
Russian Federation ◽  
Idiopathic Pulmonary Arterial Hypertension ◽  
Orphan Diseases ◽  
Study Objective ◽  
Level Of Satisfaction ◽  
Life Threatening ◽  
Pulmonary Arterial ◽  
The Russian Federation

Objective. The study objective is to analyze data of territorial subjects of the Russian Federation on idiopathic pulmonary arterial hypertension (IPAH) – nosology included in the list of life threatening and chronic advanced rare (orphan) diseases leading to the reduction in life expectancy of citizens or to their disability.Methods: Based on regulatory legal acts and federal patients registry we have analyzed morbidity and mortality, level of satisfaction with medicines supply and medicines supply expenses for patients with IPAH.Results: Criteria of level of satisfaction with drugs availability in patients with rare (orphan) diseases are suggested. Satisfaction with medicines supply is measured up against indicators and objectives of “Strategies for drug supply for the population of the Russian Federation for the period up to 2025 and a plan for its implementation”.Conclusions: The need for further improvement of regulations of orphan diseases sphere, i.e. revision of their list and regional programs creation, is justified.

Features of the organization of the provision of medicines for the treatment of orphan diseases in the Russian Federation

2020 ◽  
Vol 23 (1) ◽  
pp. 15
Author(s):  
R.U. Khabriev ◽  
A.V. Grechko ◽  
V.S. Malichenko ◽  
Yu.N. Kashevarova ◽  
N.S. Volkova
Keyword(s):  
Russian Federation ◽  
Orphan Diseases ◽  
The Russian Federation

scholarly journals Orphan diseases and associated problems

2021 ◽  
Vol 12 (2) ◽  
pp. 28-35
Author(s):  
V. A. Shashel ◽  
V. N. Firsova ◽  
M. M. Trubilina ◽  
L. A. Podporina ◽  
N. A. Firsov
Keyword(s):  
Medical Care ◽  
Russian Federation ◽  
Rare Diseases ◽  
Western Europe ◽  
Relevant Literature ◽  
The United States ◽  
Orphan Diseases ◽  
The Russian Federation ◽  
Low Prevalence ◽  
Drug Provision

Objective: to study the main aspects of assistance to children with orphan diseases in the Russian Federation. Materials and methods: the most relevant literature sources were studied that covered a concept of orphan diseases in various countries of the world and in the Russian Federation as well as the tactics and regulation of mechanisms for helping patients with rare diseases. Results: the study showed that not all countries have legislative regulations of assistance to orphan patients. The United States and Western Europe are the most advanced in this regard. They have clear criteria for determining orphan pathology and a number of measures are taken to improve the quality of medical care for patients with rare diseases. These activities are not only aimed at improving the health care system but also encourage pharmaceutical companies to develop and produce medicines as well as contribute to the research in this area. The clinical cases covered in the article provide an idea of rare diseases, the complexity of their diagnosis, the severity of the course, and the drugs that are necessary to help patients. Conclusion: orphan diseases have been the focus of attention by the health system and national legislation in the past decades. Their extremely low prevalence in the human population creates difficulties with the timely diagnosis, provision of qualified medical care, and drug provision.

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