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- S L Plavinskiĭ.
- Terapevt Arkh. 2014 Dec 20; 86 (12-2): 131813-18.
AbstractThe 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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