23.7 billion rubles to 11,175 skilled care for people suffering from rare diseases

 

Expert Council Meeting held Igor Chernyshev - Deputy Chairman of the Federation Council Committee on Social Policy and Vitaly Omel'yanovskii - Chairman of the advisory board of the Health Committee of the Federation Council on Social Policy . 

In opening remarks, thanked the audience Igor experts noted the relevance of the theme and orphan diseases : " Today, due to lack of funds often do not realize the right to provide free medical care of patients with orphan diseases . On the other hand , today is not enough techniques that would define the order of priorities and the provision of this assistance . I would like to now in the process of the meeting we heard expert opinion on the key issues of medical care for patients with rare diseases and developed activities of advisory council for the coming months . " 

Vitaly Omel'yanovskii also noted that today there is no systematic in helping patients with orphan diseases : "Today we see that the situation with regard to drug patients with rare diseases in each subject of the Russian Federation is different. Still not developed criteria for inclusion of a disease to the list of rare diseases or exclusion from this list . We see that today the subjects of the federation can not fully provide treatment for all patients with orphan diseases , so we set ourselves the task to develop criteria for prioritizing diseases , a system that would take into account the severity of the disease , on the one hand, and the availability of more effective or less effective drugs , on the other hand . " 
At the meeting of the expert council presented research working group , whose directions are outlined at the last meeting of the Expert Council. 

Expert Council for Health of the Federation Council Committee on Social Policy, together with the Centre Technology Assessment in Health Care RANHiGS the Russian President and the Research Institute of Finance Ministry of Finance of the Russian Federation , a study was conducted to assess the financial costs necessary to provide pharmaceutical care to patients with rare diseases in RF . Results of the study at the meeting of the Expert Council introduced Maria Vladimirovna SURA - Senior Fellow at the Center for Health Technology Assessment of the Russian Academy of National Economy and Public Administration under the President of the Russian Federation. The number of registered patients with rare diseases in 83 regions of Russia in 2013 amounted to 11,175 persons. According to the analysis of calculating the necessary funds to drug therapy for the patients with rare diseases funding amounts to 23.7 billion rubles . (average of 2.12 million rubles. per patient with a rare disease in the year) . Among the most costly diseases : paroxysmal nocturnal hemoglobinuria ( Marchiafava - Micheli ) - 5.3 billion rubles . 215 registered patients ; hemolytic uremic syndrome - 3.3 billion rubles . 179 patients and idiopathic thrombocytopenic purpura ( Evans syndrome ) - 2.7 billion rubles . 2700 patients. 

"We conducted a survey of pharmaceutical companies. In the case of 50% and 100 % of patients in the Russian Federation to ensure orphan drugs maximum possible discount from the manufacturers of drugs could be 5 % and 8% respectively . In this case, the amount of required financial costs of drug therapy pathogenetic registered in Russia in 2013 patients with rare diseases could be reduced from 23.7 billion rubles . to 22.4 billion rubles and 21.7 billion rubles . respectively, " - commented M.V.Sura . 

Search approaches to solving the problem of inadequate funding of rare diseases is a worldwide problem. One way to solve it is the objective ranking of rare diseases in accordance with the level of priority for funding. 

In accordance with the decision of the expert working group of the Health Council of the Federation Council on Social Policy from 18.10.2013 was launched a study on the development of criteria for the List of rare diseases. The method of solving this problem is a comprehensive assessment of rare diseases and their rankings with the use of multi-criteria analysis. 

The report " Development of criteria for the List of rare diseases " acted Olga rib - Professor of Medical Cybernetics and Informatics Medical University "Russian National Research Medical University . NI Pirogov " 

Multi-Criteria Analysis - a comprehensive integrated accounting method formalized many aspects (criteria) , applied to the problem of rare diseases - characteristics of the disease and treatment characteristics based on indicators of quality of life for patients . 

Advantages of using a multi-criteria analysis to traditional methods of technology assessment in health care are the ability to take into account many diverse factors , as well as to ensure transparency , reproducibility and objectivity of the decision process . 

"We recommend to introduce a system of multi-criteria analysis for the formation of a ranked list of rare diseases , which allows to determine the priorities for funding at the federal and regional levels, " - says Olga rib . 

The meeting was also the first time the question was raised regulatory treatment of patients with Orff states. A report on performed Nadezhda Shostak - Head of Faculty Therapy Academician AI Nesterov Medical University "Russian National Research Medical University . NI Pirogov ". Today, treatment of patients with Orff states not regulated , which creates a problem with drug supply patients with these conditions. Lack of medical treatment of these patients , in the majority of cases , results in their early disability and death. It identifies the need to develop regulatory approaches to consolidate , except in isolated FZ ¹ 323 criteria , the concept of " progressive , chronic disabling disease course ," and the status of rare ( orphan ) drug used for these states. 

Ignoring these questions leads to different interpretations of the concept of rare ( orphan ) diseases and incorporate them in a huge number of diseases not related to this category . All this creates additional social tensions among the population. One of these conditions is chronic posttromboembolicheskaya pulmonary hypertension . So , now legally provided drug coverage only patients with idiopathic pulmonary hypertension - in accordance with Articles 16 and 83 FZN 323 "On the basis of health protection in the Russian Federation" and the RF Government Decree of April 26, 2012 ¹ 403 "On the Procedure reference the Federal Register of persons suffering from life-threatening and chronic progressive rare ( an orphan ) diseases that lead to a reduction in life expectancy of citizens or their disability and its regional segment . " However, access of patients with chronic pulmonary hypertension posttromboembolicheskoy to drug therapy is hampered by the absence of the disease in the legal documentation. 
Despite these positive changes that need to be addressed problems related to improvement of regulatory and legal regulation providing care to patients with rare diseases , financing , operation and service equipment assistance to patients with rare diseases , and many others. 

We hope that such an important problem , the solution of which depends directly on the health and lives of Russians, who in many cases are desperate for help, receive due attention in the Ministry of Health of the Russian Federation. I sincerely hope that the work of specialists on the methodology and the results of research will be needed and useful. 
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REFERENCE 

Expert advice on health at the Federation Council Committee on Social Policy 

Expert Health Council of the Federation Council Committee on Social Policy is a standing consultative body of the Committee on Health , within its jurisdiction . Expert Council was established in 2008 and has more than 60 members, including politicians , policy makers, director of federal and regional research institutes , members of the Russian Academy of Sciences and Academy of Medical Sciences , representatives of scientific and medical community and public organizations. Within the framework of the expert council working groups have in cardiology , against the spread of antibiotic resistance , according to the social and economic burden of disease , reproductive health , viral hepatitis , etc. 

Research Expert Council assessed the statistics , socio- economic burden of disease in cardiology , rheumatology , pulmonology, in some infectious diseases. The data were used in the preparation of meetings on relevant topics. 

Advisory council headed by Doctor of Medical Sciences, Professor Vitaly Omel'yanovskii.