The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review

The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review. Y. Schuller, C. E. M. Hollak and M. Biegstraaten; Orphanet Journal of Rare Diseases 2015, 10:92 doi:10.1186/s13023-015-0305-y

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In the European Union (EU), a disease is considered ‘orphan’ if it is a life-threatening or seriously debilitating disorder that affects fewer than 1 per 2 000. An orphan disease is defined in the EU as a disorder affecting less than 1 in 2 000 individuals. The concept of ultra-orphan has been proposed for diseases with a prevalence of less than 1:50 000. According to this classification Friedreich's Ataxia is within the group of the "orphan", although is close to the upper border of the "ultra-orphan", so share with the "ultra-orphan" many of the problems for the development of drugs and therapies.

Source: The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review