farmaecFARMAKOEKONOMIKA. Modern Pharmacoeconomics and PharmacoepidemiologyФАРМАКОЭКОНОМИКА. Современная фармакоэкономика и фармакоэпидемиология2070-49092070-4933IRBIS LLC10.17749/2070-4909.2015.8.2.003-010farmaec-98Research ArticleOriginal ArticleОригинальные статьиREASONS FOR DIFFICULTY OF THE PHARMACOECONOMIC ANALYSIS FOR ORPHAN DRUGS. WAYS FOR SOLUTIONSПричины сложности проведения фармакоэкономического анализа для орфанных лекарств. Пути решенияПавлышАндрей ВладиславовичPavlyshAndrey Vladislavovich

к.м.н., докторант кафедры общественного здоровья и здравоохранения,

ул. Льва Толстого, д. 6-8, Санкт-Петербург, 197022;

начальник службы медицинского снабжения и обеспечения лечебного процесса – врач-клинический фармаколог,

ул. Академика Лебедева, д. 4/2, Санкт-Петербург, 194044

MD, intramural doctoral of department of public health and health care,

Leo Tolstoy, 6-8, St. Petersburg, 197022;

head of department of medical supplies and providing medical process – clinical pharmacologist,

Acad. Lebedev St., 4/2, St. Petersburg, 194044

pavlysh_63@mail.ruКолбинАлексей СергеевичKolbinAleksei Sergeevich

д.м.н., профессор, заведующий кафедрой клинической фармакологии и доказательной медицины,

ул. Льва Толстого, д. 6-8, Санкт-Петербург, 197022;

профессор кафедры фармакологии медицинского факультета,

21-я линия, д. 8, В.О., Санкт-Петербург, 199106

MD, professor, head of department of clinical pharmacology and evidence-based medicine,

Leo Tolstoy, 6-8, St. Petersburg, 197022;

Professor of the Department of Farmakology,

21-Line, 8, VO, St. Petersburg, Russia, 199106

alex.colbin@gmail.comГапешинРоман АндреевичGapeshinRoman Andreevich

выпускник Медицинского факультета,

21-я линия, д. 8, В.О., Санкт-Петербург, 199106

MD, graduate,

21-Line, 8, VO, St. Petersburg, 199106

gapeshin.ra@gmail.comМалышевСтанислав МихайловичMalyshevStanislav Mikhailovich

выпускник Медицинского факультета,

21-я линия, д. 8, В.О., Санкт-Петербург, 199106

MD, graduating student, 

21-Line, 8, VO, St. Petersburg, 199106

malyshev.stm@gmail.comПервый Санкт-Петербургский государственный медицинский университет им. академика И.П. Павлова; Федеральное государственное бюджетное учреждение «Всероссийский центр экстренной и радиационной медицины им. А.М. Никифорова» МЧС РоссииРоссияFirst St. Petersburg State Medical University; The Federal State Budgetary Institute «The Nikiforov Russian Center of Emergency and Radiation Medicine» The Ministry of Russian Federation for Civil Defense, Emergencies and Elimination of Consequences of Natural DisastersRussian FederationПервый Санкт-Петербургский государственный медицинский университет им. академика И.П. Павлова; Санкт-Петербургский государственный университетРоссияFirst St. Petersburg State Medical University; First St. Petersburg State UniversityRussian FederationСанкт-Петербургский государственный университетРоссияSt. Petersburg State UniversityRussian Federation20152409201582310Copyright © Pavlysh A.V., Kolbin A.S., Gapeshin R.A., Malyshev S.M., 20152015Павлыш А.В., Колбин А.С., Гапешин Р.А., Малышев С.М.Pavlysh A.V., Kolbin A.S., Gapeshin R.A., Malyshev S.M.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.pharmacoeconomics.ru/jour/article/view/98

Orphan drugs are used for treatment of rare life-threatening diseases. There is no universal definition of a rare disease and no universal approach to orphan drugs legislation. As a result, decision-making in this field requires results of pharmacoeconomic analysis, which structure and interpretation need a specific approach due to high costs of the drugs and limited sample size. Cost-utility analysis, risk management, and international rare diseases patient registries could be used to solve these problems.

Орфанные лекарственные средства (ЛС) применяют для лечения редких жизнеугрожающих заболеваний. Единого определения редкого заболевания не существует, как не существует и единого подхода к контролю орфанных ЛС. Механизм образования стоимости орфанных препаратов многими авторами расценивается как непрозрачный, и в целом затраты на них оказываются очень высокими. В связи с этим для принятия обоснованных решений в этой области требуются данные фармакоэкономического анализа, структура и интерпретация результатов которого требуют особого подхода с учетом высокой стоимости ЛС и ограниченности контингента пациентов. Для решения данной проблемы применяют анализ «затраты-полезность», системы управления рисками, а также формируют международные регистры пациентов с орфанными заболеваниями.

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The authors declare that there are no conflicts of interest present.