Extract
In the past few years, interest in rare diseases has grown, as demonstrated by the agendas of politicians and health authorities, but too little attention is still paid to ultra-rare diseases. Although no legal definition of an “ultra-rare” disease has yet been established, this subcategory was informally introduced by the National Institute for Health and Care Excellence for drugs with indications for diseases that have a prevalence <1 per 50 000 persons [1–3].
Abstract
Too little attention is still paid to ultra-rare diseases. It is important to disseminate the best information on less common diseases in order to improve the clinical managment and improve care. https://bit.ly/2NougE5
Footnotes
Provenance: Commissioned article, peer reviewed
Conflict of interest: S. Harari reports grants and personal fees from Roche, Actelion and Boehringer Ingelheim, outside the submitted work.
Conflict of interest: M. Humbert reports personal fees from Acceleron, GSK, Merck, Novartis, AstraZeneca and Sanofi, and grants and personal fees from Actelion and Bayer, outside the submitted work.
- Received June 16, 2020.
- Accepted June 18, 2020.
- Copyright ©ERS 2020.
This article is open access and distributed under the terms of the Creative Commons Attribution Non-Commercial Licence 4.0.
