ASSESSMENT OF NECESSARY FINANCIAL EXPENSES ON THE DRUG SUPPLY OF THE PATIENTS WITH ORPHAN DISEASES IN THE RUSSIAN FEDERATION

the article presents the results of cost calculation on the drug supply of the patients with orphan diseases, included in the list of orphan diseases (24 diseases) by the RF Government Regulation of April 26, 2012 № 403. Calculation was performed based on the data of registered patients with orphan diseases (OD) in 83 territorial entities of the Russian Federation in 2013 and based on the average-weighted costs of their drug therapy. Data on registered patients with OD was obtained because of the official request of the Expert Board on Healthcare of the Council of the Federation Committee on Social Policy that was sent to healthcare regulatory bodies of the 83 territorial entities of the Russian Federation. Average-weighted cost calculation of drug treatment (or of specialized nutrition care) of the patients with OD was carried out in accordance with medical care standards and expert assessment. As part of the analysis of the standards the search for pathogenic therapy medications was carried out, in the absence of which – there was a search for supportive care medications that critically affect survivability of the patients with OD. According to the selected list of drugs, information about duration, frequency of prescription, average daily and course dose, was analyzed. In the absence of medical care standards, and in order to verify some questions concerning drug therapy of patients with OD consultations with experts were held. Because of conducted calculation, total average-weighted costs of pharmacotherapy for 24 orphan diseases were 15.73 bln rubles per year (6.36 bln rubles for children and 9.37 bln rubles for adults) for 11 173 registered patients that by 3.3 exceeds current amount of finance. Pathogenetic pharmacotherapy of Mucopolysaccharidoses type VI and II proved to be the most expensive treatment per one patient per year: average-weighted costs per one child with Mucopolysaccharidoses type VI amounted to 40.90 mln rubles, per one adult – to 71.50 mln rubles; for Mucopolysaccharidoses type II costs were 29.60 and 51.83 mln rubles respectively. In order to increase effectiveness of the system of drug supply of the patients with OD and to optimize state expenses for this group of patients it is necessary to approve the entire range of financially balanced and science based medical care standards, to define the legal status of orphan drugs and to carry out state regulation of prices, to ensure increase of regional funding supported by involvement of federal budget funds, to define the list of orphan drugs, to implement state regulation measures to create incentives for pharmaceutical companies to reduce prices for orphan drugs.

1. Materials for the meeting of the round table on the topic «Access to health care and drug supply patients with rare diseases in the Russian Federation.» The results of the research Center for the Study and population analysis, Demographic and Health Institute EarAzES. The State Duma of the Federal Assembly of the Russian Federation [Materialy k zasedaniyu kruglogo stola na temu «Dostupnost' meditsinskoi pomoshchi i lekarstvennogo obespecheniya bol'nykh s redkimi zabolevaniyami v RF». Rezul'taty issledovaniya Tsentra izucheniya i analiza problem narodonaseleniya, demografii i zdravookhraneniya Instituta EarAzES. Gosudarstvennaya Duma Federal'nogo Sobraniya Rossiiskoi Federatsii]. Moscow. 2014.

2. All-Russian public organization «League advocates for patients» [Obshcherossiiskaya obshchestvennaya organizatsiya «Liga zashchitnikov patsientov»] http://ligap.ru/articles/zayavlenia/zayavl/

3. Government Decree of April 26, 2012 № 403 «On the order of the Federal Register of persons suffering from life-threatening and chronic progressive rare (orphan) diseases that can shorten life expectancy of citizens or their disability and its regional segment [«Postanovlenie Pravitel'stva RF ot 26 aprelya 2012 g. № 403 «O poryadke vedeniya Federal'nogo registra lits, stradayushchikh zhizneugrozhayushchimi i khronicheskimi progressiruyushchimi redkimi (orfannymi) zabolevaniyami, privodyashchimi k sokrashcheniyu prodolzhitel'nosti zhizni grazhdan ili ikh invalidnosti, i ego regional'nogo segmenta»].

4. Government Decree of 18.10.2013 N 932 «On the program of state guarantees of free provision of medical care to citizens in 2014 and the planned period 2015 and 2016» [Postanovlenie Pravitel'stva RF ot 18.10.2013 N 932 «O programme gosudarstvennykh garantii besplatnogo okazaniya grazhdanam meditsinskoi pomoshchi na 2014 god i na planovyi period 2015 i 2016 godov»].

5. Order of the Ministry of Health on February 13, 2013 № 66 «On approval of the Strategy of medicinal maintenance of the population of the Russian Federation for the period up to 2025 and its implementation plan» [Prikaz Ministerstva zdravookhraneniya RF ot 13 fevralya 2013 g. № 66 «Ob utverzhdenii Strategii lekarstvennogo obespecheniya naseleniya Rossiiskoi Federatsii na period do 2025 goda i plana ee realizatsii»].

6. Draft Law «On Amendments to the Federal Law» On Circulation of Medicines «and Article 333.32.1. Part Two of the Tax Code of the Russian Federation» [Proekt FZ «O vnesenii izmeneniya v Federal'nyi zakon «Ob obrashchenii lekarstvennykh sredstv» i v stat'yu 333.32.1. chasti vtoroi Nalogovogo kodeksa Rossiiskoi Federatsii»]. http://www.rosminzdrav.ru/open/discuss/projects/3.

7. Reference drugs Formulary Committee [Spravochnik lekarstvennykh sredstv Formulyarnogo komiteta]. moscow. 2006; 668 s.

8. Sura M. V., Omel'yanovskii V.V., Avksent'eva M.V. i dr. Meditsinskie tekhnologii. Otsenka i vybor. 2014; 3 (17): 43-50.

9. Federal Law № 323-FZ of November 21, 2011. «On the basis of health protection in the Russian Federation» [Federal'nyi zakon RF № 323-FZ ot 21 noyabrya 2011 g. «Ob osnovakh okhrany zdorov'ya grazhdan v Rossiiskoi Federatsii»].

10. http://www.pharmvestnik.ru/publs/lenta/v-rossii/elenamaksimkina-dostup-boljnyx-k-orfannym-preparatam-budetuproschen.html.

11. European Medicines Agency (EMA): Orphan drugs and rare diseases at a glance. http://www.ema.europa.eu/docs/en_GB/document_library/Other/2010/01/WC500069805.pdf ]. Doc. Ref. EMEA/290072/2007.

12. http://www.eurordis.org/.

13. KCE reports 112C. Policies for orphandiseases and orphandrugs. 2009.

14. Orofino J., Soto J., Casado M.A., Oyagüez I. Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007. Appl Health Econ Health Policy. 2010; 8 (5): 301-315.

15. Schey C., Milanova T., Hutchings A. Estimating the budget impact of orphan medicines in Europe. 2010-2020. Orphanet Journal of Rare Diseases. 2011; 6: 62.

16. Schwabe U., Paffrath D. Arzneiverordnungsreport. 2010; http://www.springer.com/pharma/book/978-3-642-13379-4