Ultra-orphan medicines

A new approach to the assessment of ultra-orphan medicines is being introduced, in line with the Scottish Government announcement in June 2018 on a new pathway.

Read the Scottish Government announcement

Revised definition: ultra-orphan medicine

To be considered as an ultra-orphan medicine all criteria listed should be met:

  1. The condition* has a prevalence of 1 in 50,000 or less in Scotland, 
  2. The medicine has an EMA orphan designation for the condition and this is maintained at time of marketing authorisation, 
  3. The condition is chronic and severely disabling and
  4. The condition requires highly specialised management. 

* The condition is that defined in the European Medicines Agency's (EMA) Orphan Maintenance Assessment Report (OMAR) or the condition in the original orphan designation if the OMAR is not available.

Submissions for medicines that are validated as ultra-orphan according to this definition will be assessed by SMC and will then be available to prescribers for a period of at least three years while further clinical effectiveness data are gathered. After this period SMC will re-assess the data and make a final decision on routine use of the medicine in NHSScotland.

New ultra-orphan pathway

1. Validation as ultra-orphan medicine

In order for SMC to validate a medicine as ultra-orphan, companies are encouraged to provide relevant supporting evidence  before receiving a Committee for Human Medicinal Products (CHMP) opinion. In addition, when we contact companies following a CHMP positive opinion, we will ask the company to indicate whether a medicine is expected to meet the ultra-orphan definition and complete a proforma (if not previously received).

Ultra-orphan proformas should be submitted by 12 noon on the first Monday of the month. We will then proceed with validation and confirm eligibility for the ultra-orphan pathway before the company makes a submission. The validation process is expected to take around eight weeks. This validation process is now in place (October 2018).

We will contact companies with the outcome of the validation and, in the case, of disagreement, there will be an opportunity for the company to appeal.

Note that in the event of a high number of proformas being received we may prioritise to take account of the date and time received and actual / estimated date of product availability in the UK. 

Download the ultra-orphan proforma

2. Initial SMC assessment

Following validation companies will be encouraged to submit for the initial assessment using the SMC ultra-orphan framework. Further information on the submission requirements will be provided following engagement with the pharmaceutical industry, patient groups and other key stakeholders.

View the ultra-orphan framework

Submissions will be assessed using the new ultra-orphan approach from April 2019. This will ensure ongoing submissions for medicines already confirmed as ultra-orphans according to the current definition are completed before the new approach is introduced.