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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.

About the Authors

Andrey Vladislavovich Pavlysh
First 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 Disasters
Russian Federation

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

Aleksei Sergeevich Kolbin
First St. Petersburg State Medical University; First St. Petersburg State University
Russian Federation

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

Roman Andreevich Gapeshin
St. Petersburg State University
Russian Federation

MD, graduate,

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

Stanislav Mikhailovich Malyshev
St. Petersburg State University
Russian Federation

MD, graduating student, 

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


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For citation:

Pavlysh A.V., Kolbin A.S., Gapeshin R.A., Malyshev S.M. REASONS FOR DIFFICULTY OF THE PHARMACOECONOMIC ANALYSIS FOR ORPHAN DRUGS. WAYS FOR SOLUTIONS. PHARMACOECONOMICS. Modern pharmacoeconomics and pharmacoepidemiology. 2015;8(2):3-10. (In Russ.)

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