The Ministry of health of the Russian Federation accepted the decision about inclusion of a drug to treat spinal muscular atrophy in the list of vital and essential drugs (EDL). This means fixing the maximum ex-works prices for this extremely expensive drug, making the treatment of patients with this disease more affordable. Yet, according to the experts, they provided only 129 of 989 people in need.The decision to make the drug in the list of vital was approved by the relevant Commission of the Ministry of health 11 votes for and nine against. Spinal muscular atrophy (SMA) — orphan (rare) genetic disease that affects motor neurons and leads to atrophy of the muscles. The state register of patients with a diagnosis of SMA no, but, according to the charity Fund “Family AGR” today in Russia the number of such patients is 989 people, of which 210 were adults, 779 children.For the treatment of patients in Russia shows extremely expensive and only registered in a country drug with the generic name nusinesses. Therapy consists in the introduction of six injections in the first year and three for each subsequent throughout the life of the patient. The cost of one injection in Russia reached 7.8 mln. According to the estimates of the Director of the “Families of SMA” Olga Getmanenko, today treatment in Russia is provided only 129 patients. Low availability of treatment is not only the high cost of the drug, but also the fact that not identified sources of funding. As noted at the July meeting of the expert Council of state Duma Committee on health protection, the Commissioner for children’s rights in the Russian Federation Anna Kuznetsova, only for the treatment of children and adults with SMA per year requires about 40 billion rubles.”unfortunately, the majority of patients receiving treatment today, I had to pursue this through the courts— says Olga Getmanenko.— In the regions, the patients often get denial treatment. Officially, the regional authorities explain this by the fact that the disease is not present in the WNV program (high-cost nosologies), the list of life-threatening orphan diseases or other targeted programmes, and the drug is not in the list of essential drugs”.This is necessary to ensure that public procurement is not dependent on changes in the price of drugs due to exchange rate fluctuations in the global market. The price for a drug included in the EDL may be indexed only in line with inflation. According to Anna Kuznetsova, has an agreement with the manufacturer on fixing the estimated exchange rate at the secondary level for 2019. “As a result, the proposed manufacturer price of the drug by nearly 25% below current average prices,” she said.”A year ago, when the drug was registered in Russia, it became obvious�� that the regions do not have sufficient funds to provide this expensive drug to patients with SMA,— the Chairman of the Duma Committee on health Dmitry Morozov.— The economic feasibility of including a drug in the EDL is that the manufacturer is ready to significantly reduce the price of the medicine, realizing that inclusion will lead to higher procurement and increased use of the drug in the country.” Dmitry Morozov considers that in the future for patients with SMA will need to organize a centralized funding from the Federal budget, which will simplify the provision of medicines for patients, to establish routing and create the accounting system.”Today, the average price per vial for regional auctions — 7.8 million rubles., the price proposed by the manufacturer for inclusion in the EDL— 5.1 mln At check prices the cost of a patient with MCA in the first year of treatment will be reduced by 13 million rubles., and in the second and subsequent years — 6.7 mln. in fact, every fourth patient with SMA will receive treatment due to the price difference from the inclusion of a drug in the list”,— says the head of the project office “Rare (orphan) diseases” FEDERAL state budgetary institution “national research Institute of public health. N. A. Semashko” Elena Krasilnikova.”However, the inclusion of a drug in the list — only the first step towards real availability of therapy,” she says. Sustainable Federal financing of preferential drug supply patients with rare diseases (including SMA) today only possible at the expense of the Federal budget in the framework of the high-cost nosologies, at the expense of the CMI program of high-tech care.Konstantin Anokhin
