Publication - Advice and guidance

Ultra-orphan medicines pathway: guidance

Published: 1 May 2019
Directorate: Chief Medical Officer Directorate
Topic: Health and social care

Guidance for health boards, clinicians, pharmacists, patient groups and pharmaceutical companies on the new approach to the assessment of ultra-orphan medicines.

This is a new system for the assessment of medicines for very rare disease (ultra-orphan). Under this pathway medicines can be made available through the NHS in Scotland for a period of three years prior to a decision on routine use in NHS Scotland. There are a number of criteria that a pharmaceutical company must fulfil if they wish their medicine to be assessed via the new pathway.

This guidance is intended to provide information to Health Boards, clinicians, pharmacists, patient groups and pharmaceutical companies on the new approach to the assessment of ultra-orphan medicines which was introduced in Scotland in October 2018, with applications for new medicines available from April 2019.

The changes mean if the medicine meets the new definition of an ultra-orphan medicine and undergoes a full assessment of its clinical and cost-effectiveness by the Scottish Medicine Consortium (SMC), then it will be available on the NHS for up to three years while further evidence on its effectiveness is generated. The SMC will then review the evidence after three years and make a final decision on its routine use in NHS Scotland.

To enable an ultra-orphan medicine to be available through the pathway, there are four conditions that must be met. They are that a pharmaceutical company:

has the medicine validated as an ultra-orphan according to the SMC definition
makes a full submission to the SMC for the initial assessment stage that meets SMC requirements for assessment under the ultra-orphan process
offers a Patient Access Scheme (PAS) that complies with the standard terms and conditions considered acceptable by the Patient Access Scheme Assessment Group (PASAG)
supports the data collection arrangements that meets the evidence generation requirements for assessment under the ultra-orphan pathway

More information is available in the guidance attached to this page, together with guidance on the evidence generation phase of the pathway for ultra-orphan medicines.