Ultra-orphan medicines

This scheme pools funds from all Scotland’s health boards for a small number of approved medicines for very rare diseases.

In June 2018 the Scottish Government announced its new ultra-orphan pathway. This new process makes medicines for very rare diseases available to Scottish patients.

It’s hoped that the pathway will:

• improve early access to ultra-orphan medicines
• support the collection of more real world data to support future Scottish Medicine Consortium (SMC) reassessment and decision making about the continued availability of medicines the initiative covers

The Scottish Board Chief Executives (BCEs) agreed, in April 2020, to set up a new risk sharing arrangement to fund products approved through the new process. This scheme replaces the old Ultra Orphan Drugs Risk Sharing scheme created in 2005. Medicines for inherited metabolic diseases (IMD), which the old scheme funded, are now covered by the IMD medicines risk share. We also host this scheme.

The new (2020) Ultra-Orphan Drug Risk Share provides funding for:

• medicines that have approval via the ultra-orphan medicines pathway
• a small number of medicines for extremely rare conditions that have been accepted by SMC outside the new ultra-orphan process

List of medicines and indications included in the scheme

Find further information about the ultra-orphan medicines pathway on the gov.scot website.

Healthcare Improvement Scotland is another useful resource.