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.
Revised definition: ultra-orphan medicine
To be considered as an ultra-orphan medicine all criteria listed should be met:
- The condition has a prevalence of 1 in 50,000 or less in Scotland,
- The medicine has an EMA orphan designation for the condition and this is maintained at time of marketing authorisation,
- The condition is chronic and severely disabling and
- The condition requires highly specialised management.
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
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 six 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.
2. Initial SMC assessment
Companies are 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.
Note: submissions for medicines identified as ultra-orphan according to the current definition will continue to be assessed using the existing ultra-orphan process. To ensure that these ongoing submissions are completed before the new approach is introduced, future submissions will be assessed using the new ultra-orphan pathway from April 2019.