Orphan disease status of cancer cachexia in the USA and in the European Union: a systematic review

Cachexia has significant impact on the patients' quality of life and prognosis. It is frequently observed in patients with cancer, especially in advanced stages, but prevalence data for the overall population are lacking. Good quality estimates of cancer cachexia in general and for each of the major cancer types would be highly relevant for potential treatment development efforts in this field. Both the USA and European Union (EU) have implemented special clinical development rules for such rare disorders what are called ‘orphan diseases’. The cut‐off level for a disease to be considered an orphan disease in the USA is 200 000 people (0.06% of the population) and EU is 5 per 10 000 people (0.05% of the population).

• Publication year

  2019

• Venue

  Journal of Cachexia, Sarcopenia and Muscle

• Publication date

  2019-02-01

• Fields of study

  Medicine

• Identifiers
  DOI 10.1002/jcsm.12402PMID 30920776PMCID 6438416
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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