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Double bad luck: Should rare diseases get special treatment?
  1. Adam Hutchings
  1. Dolon Limited, London, UK
  1. Correspondence to Adam Hutchings, Dolon Limited, London, UK; adam.hutchings{at}dolon.com

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In June 2021, an 1856 British Guiana 1c magenta stamp sold for US$8.3m. It is the only known specimen of its kind in existence and on a gram-for-gram basis the most valuable item in the world. Clearly, in some spheres of human engagement, rarity carries a premium. Should this logic be applied in healthcare?

Magalhaes thinks not.1 They explore the topic of whether pricing and reimbursement (P&R) systems should give a premium to orphan drugs for rare diseases. They argue that rarity is irrelevant to pharmaceutical policy on ethical grounds, and instead drug incentives should be prioritised by disease severity. At a time when incentives for orphan medicines are being revisited globally, Magalhaes is provoking an important discussion.

Philosophical clarity, messy reality

The author seeks to disentangle the interwoven concepts of rarity, severity and unmet need (lack of effective treatments) that factor into decisions on orphan drug policy. They perhaps overstate the emphasis on rarity within existing regulatory and P&R frameworks. Yes, this is one of the criteria for orphan drug designation, but in payer systems rarity is seldom explicitly referenced. For example, of the five largest European countries, only the United Kingdom formally adjusts willingness-to-pay thresholds by prevalence. In contrast, concepts of severity and unmet need feature widely in global regulatory processes (eg, Breakthrough Therapy designation from …

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Footnotes

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests None declared.

  • Provenance and peer review Commissioned; internally peer reviewed.

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