Severe asthma in adults: an orphan disease?

Clin Chest Med. 2012 Sep;33(3):571-83. doi: 10.1016/j.ccm.2012.06.008. Epub 2012 Aug 2.


Severe asthma affects fewer than 10% of patients with asthma, is associated with a severe risk of death and disability, has a great impact on health and quality of life, and represents a huge cost to patients and society. Given the poor response to treatment and the side effects associated with medications for severe asthma, more efficient, cost-effective, and phenotype-specific medications are needed. Considering severe asthma as an orphan disease could encourage the pharmaceutical industry to stratify studies based on a more detailed characterization of study subjects at baseline, resulting in the development of novel therapeutic approaches.

Publication types

  • Review

MeSH terms

  • Adult
  • Anti-Asthmatic Agents / adverse effects
  • Anti-Asthmatic Agents / therapeutic use
  • Asthma* / diagnosis
  • Asthma* / drug therapy
  • Asthma* / economics
  • Cohort Studies
  • Humans
  • Phenotype
  • Rare Diseases*
  • Risk Factors
  • Severity of Illness Index*
  • Terminology as Topic
  • Treatment Outcome


  • Anti-Asthmatic Agents