An ultra-rare disease is a disease that affects an extremely small percentage of the population. In some parts of the world, an ultra-orphan disease is a rare disease whose rarity means there is a lack of a market large enough to have support and resources for discovering treatments for it.[1][2][3]

Distinct countries define and provide special economic incentives for companies developing drugs that treat ultra-rare diseases. In 2018, the Scottish Government introduced a new definition of 'ultra-orphan medicines' that can treat very rare conditions affecting fewer than 1 in 50,000 people – around 100 people or less in Scotland.[4] The new definition also allows the Scottish Medicines Consortium (SMC) the ability to treat some medicines for rare orphan diseases as ultra-orphan medicines. The changes mean if the medicine meets the new definition of an ultra-orphan medicine and the SMC consider it clinically effective, then it will be made available on the NHS for at least three years while information on its effectiveness is gathered. The SMC will then review the evidence and may make a final decision on its routine use in NHS Scotland.

Scholars have recently (2022–2023) proposed adoption of a similar ultra-orphan designation in the US with a prevalence threshold of 1 in 50,000 patients or based on an arbitrary threshold of 10,000 affected US citizens.[5][6]

See also

References

  1. ↑ Crooke, Stanley T. (June 2021). "A call to arms against ultra-rare diseases". Nature Biotechnology. 39 (6): 671–677. doi:10.1038/s41587-021-00945-0. ISSN 1546-1696. Retrieved 22 September 2023.
  2. ↑ Harari, Sergio; Humbert, Marc (30 June 2020). "Ultra-rare disease: an European perspective". European Respiratory Review. 29 (156). doi:10.1183/16000617.0195-2020. ISSN 0905-9180. PMC 9488651. Retrieved 22 September 2023.
  3. ↑ Dear, JW; Lilitkarntakul, P; Webb, DJ (September 2006). "Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products". British Journal of Clinical Pharmacology. 62 (3): 264–71. doi:10.1111/j.1365-2125.2006.02654.x. PMC 1885144. PMID 16934041.
  4. ↑ "Treatments for rare conditions". www.gov.scot. 2018-06-18. Retrieved 2023-05-17.
  5. ↑ Michaeli, Thomas; Jürges, Hendrik; Michaeli, Daniel Tobias (2023-05-09). "FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis". BMJ. 381: e073242. doi:10.1136/bmj-2022-073242. ISSN 1756-1833. PMC 10167557. PMID 37160306.
  6. ↑ "ICER Publishes White Paper Evaluating Reforms to Orphan Drug Development, Pricing, and Coverage". Institute for Clinical and Economic Review. 2022-04-07. Retrieved 2023-05-17.
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