“the Main result, including the forum, constructively and openly discuss the most pressing issues with the participation of all parties – legislators, Ministry of health and Ministry of industry, patient organizations, medical and scientific community. This is a great achievement that in recent years managed to change the attitude of people suffering from orphan diseases, and we began to systematically engage the key areas that affect their treatment and ultimately, quality of life,” – said “RG” the co-chair of the all-Russian Union of patient associations Yury Zhulev.
One of the most acute problems – the provision of medicines. In most cases, even if the drugs registered in Russia, they are so expensive that their purchase to provide modern therapy, even a small number of patients (and for orphan diseases treatment, usually lifelong) result in enormous costs. The cost of annual treatment per patient for some diseases may reach 100 million rubles.
Orphan patients require systemic solutions
For orphan patients, there are two programs: the Federal “7 high-cost nosologies”, which last year included five diseases (“12 nosologies”), and this year added two more, and now the Federal procurement of drugs provided for patients at the 14 orphan diseases. The second program is “life-threatening and chronic progressive rare disease” includes more than 20 diseases, and these patients should be provided medicines at the regional level.
So, on the purchase of medicines for the 12 most high-cost nosologies (VZN) in 2019 has been allocated from the Federal budget 55.7 billion rubles. In 2020, with the expansion of the program, the amount increased to 61.8 billion rubles, said at the forum the head of the relevant Department of the Ministry of health Elena Maksimkina. Increased the number of purchased drugs five years ago there were 18, now 39.
“the System of authority for the provision of medicines is divided between the Federal and regional level, and if Federal purchases are made in full, and we have no lawsuits from patients in the regions of the obligation on provision of medicines is performed depending on the financial situation of the specific subject of the Federation”, – said Elena Maksimkina.
In many regions, attempts of patients to obtain drugs by the decision of the court became a frequent story.
According to the representative of the Ministry of health, federalization of purchases is the right way, the list of high-cost nosologies will continue to expand. Tendering centrally helps to optimize the purchase price. In addition, when such organization has the opportunity to reallocate purchased medicines between patients living in different regions, and in the case of release or unclaimed drugs.
Isaev: it is Necessary to include MCA in the list of orphan diseases
this forum has discussed on the improvement of procurement at the regional level – we are preparing amendments to the legislation on public procurement to allow the carry trades together in multiple regions.
the Second direction – work with the pharmaceutical companies producing orphan drugs. According to the head of the Committee on health Dmitry Morozov, it is possible to negotiate and lower prices in procurement, and about carrying out of early access (for which the derivation of a new drug on the market, the manufacturer provides the treatment of a specified number of patients on a Pro Bono basis), and registration of innovative drugs in Russia. “We are also ready to discuss at the expert group the possibility of using programs risk-sharing (risk sharing), when productiondrivers drugs receive payment for them only in the case of the impact of treatment. It could also help to reduce state expenses on the purchase of expensive drugs”, – told “RG” Dmitry Morozov.
the most Important area, which can also help to optimize costs – development and production of our own medicines for the treatment of orphan diseases. “This is a very sensitive area and the Ministry on this special attention – said at the forum the first Deputy Minister of industry and trade Sergey Tsyb. – Now developing more than 20 projects, for which the budget allocated more than 800 million rubles.”
Expanded the list of orphan diseases
Sergey Tsyb noted that in recent years have created some excellent working orphan drugs – in particular, for the treatment of hemophilia. The market of domestic developments to reduce prices of medicines and to ensure they have a greater number of patients. For example, the emergence of Russian preparations for the treatment of multiple sclerosis and juvenile arthritis allowed to purchase 25-30 percent cheaper.
Also, the first Deputy Minister noted, will be soon brought to market the drug for the treatment of Gaucher disease, at the stage of registration of drugs from aplastic anemia and juvenile arthritis, clinical trials of drugs for the treatment of hereditary angioedema and mucopolysaccharidosis type II (hunter syndrome). In 2020 is also planned preclinical studies of the Russian gene therapy drug for the treatment of spinal muscular atrophy (SMA).
great attention is paid to compliance with international standards of manufacturing practices, compliance with internal and external controlI’m in the production. Because the only way to guarantee their safety and efficiency, the official said. According to Tsyba, all pharmaceutical companies involved in the development and production of orphan drugs are new production capacity, equipped with level and even better than many of the European manufactures.