Abstract
Currently, there are in excess of 5000 rare diseases, that is, diseases that affect only a small proportion of a given population. The majority of these conditions lack appropriate treatments for a variety of reasons, including limited markets for the development of new pharmaceutical products coupled with prohibitively high costs associated with research, manufacturing and marketing. This paper discusses the issues associated with the equity of and access to orphan drugs used in rare diseases. Options for healthcare policy across Europe are presented. The interest in using robust evaluation parameters is emphasized, despite this not being in agreement with the use of artificial indicators such as quality-adjusted life years (QALYs), which appear to lead to divergent and erroneous results. New decision-making methods must govern the policy, statistics, and economics relevant to the use of orphan drugs in rare diseases.
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