Economic assessment of drugs for rare diseases: Must it be done, if yes, which are its features?


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Abstract

The orphan drug laws enacted firstly in the USA and then in some other countries have given rise to a specific market of drugs for rare diseases and to a drastic increase in the number of proposed drugs. At the same time, the prices for innovative drugs are frequently high, have resulted in debates about their cost effectiveness and the drugs for rare diseases being frequently difficult to be tested for their efficacy due to disease rarity. The article discusses the designs of clinical trials that could enhance their effectiveness, gives examples of standard-design trials, and notes that effectiveness evaluations can be obtained for most diseases, but with a small sample, they will not be very precise, which makes cost-effectiveness analysis difficult. In this connection, a number of mechanisms proposed worldwide to solve the existing situation are discussed.

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Экономическая оценка лекарств для редких заболеваний: должна ли она выполняться, и если да, то каковы ее особенности?. - Аннотация. Принятие законов о лекарствах для редких (орфанных, "сиротских") заболеваний, первоначально в США, а затем и в ряде других стран мира привело к формированию специфического рынка препаратов для редких лекарств и резкому увеличению числа предлагаемых препаратов. Вместе с тем цены на инновационные препараты зачастую достаточно высоки, что порождает дебаты об их стоимостной эффективности и о том, что действенность лекарств для орфанных заболеваний в принципе сложно проверить ввиду их редкости. В статье обсуждаются типы дизайнов, которые могли бы способствовать повышению результативности исследований, даны примеры исследований, проводившихся в стандартном дизайне, отмечается, что для большинства заболеваний можно получить оценки эффективности, но при малом размере выборки они будут не очень точными, что затрудняет анализ стоимостной эффективности. В этой связи обсуждается ряд механизмов, которые предлагались в мире для выхода из сложившейся ситуации.
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About the authors

S L Plavinskiĭ

Email: splavinskij@mail.ru

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