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Ultra-orphan drugs for rare diseases

Published on 27 September 2019

All territorial NHS boards pool funds to meet the costs for a small number of ultra-orphan drugs for patients resident in Scotland. This scheme is called the Ultra Orphan Drugs Risk Share. Our National Services Division manages it.

The majority of drugs the scheme covers are for people with inherited metabolic disorders. This long-standing funding mechanism is currently separate from the arrangements for drugs approved as part of the Scottish Medicine Consortium’s (SMC) new Ultra Orphan Drugs Pathway.

The Ultra Orphan Drugs Risk Sharing Scheme is currently under review.

List of drugs included in the scheme

We fund all of the medicines below if they're prescribed by the Scottish Inherited Metabolic Disorders Service.

Drugs for the treatment of Fabry's disease:

  • Agalsidase alpha (Replagal)
  • Agalsidase beta (Fabrase/Fabrazyme)
  • Migalastat (Galafold®)

Drugs for the treatment of Type 1 Gaucher's disease:

  • Imiglucerase (Cerezyme)
  • Miglustat (Zavesca/Vevesca)
  • Velaglucerase (Vpriv)
  • Eliglustat (Cerdelga ®)

Drugs for the treatment of other diseases:

  • Mifamurtide (Mepact) for the treatment of high-grade resectable non-metastatic osteosarcoma in children, adolescents and young adults
  • Carbaglu (N-carbamoyl-L-glutamic acid) for the treatment of hyperammonaemia due to Nacetylglutamate synthase deficiency (restricted use)
  • Alglucosidase alfa (Myozyme) for the treatment of Pompe disease (acid maltase deficiency)
  • Laronidase (Aldurazyme) for the treatment of MPS 1, Hurler-Scheie or Scheie syndrome
  • Idursulfase (Elaprase) for the treatment of MPS II (Hunter's syndrome)

NSD contacts

Programme Manager, Dr Anke Roexe – anke.roexe@nhs.net – 0131 3141053

Senior Programme Manager, Gordon Kirkpatrick – gordon.kirkpatrick@nhs.net – 0131 2756456