Authors
Plavinski S. L.
North-West State Medical University named after I.I. Mechnikov, Saint-Petersburg, Russia
Abstract
Orphan drug laws that were enacted firstly in the USA and then in some other countries led to the creation of functioning market of drugs for rare diseases and increased number of such drugs. In the same time price tag for innovative drugs is frequently high which lead to the debates about their cost effectiveness and that effectiveness of rare diseases drug is frequently difficult to test because of disease rarity. The article discussed some types of the designs of clinical trials that could increase effectiveness and examples are given with standard design. It is mentioned that for majority of the diseases there is a possibility to get point estimates for the effectiveness, but with small sample size they will not be precise which will contribute to the difficulties with cost-effectiveness analysis. Some mechanisms are discussed that were proposed to solve the situation.
Key words
rare diseases, clinical trial design, cost-effectiveness analysis, quality-adjusted life years, reimbursement schemes
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