Ultra-orphan medicines
Following a Scottish Government announcement in 2018, SMC has developed a framework for assessing ultra-orphan medicines for very rare conditions.
Ultra-orphan medicine definition
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 a Great Britain (GB) orphan marketing authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA)
- the condition is chronic and severely disabling, and
- the condition requires highly specialised management.
* typically a recognised distinct disease or syndrome.
SMC uses the description of the orphan condition within the MHRA Orphan Register. 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 up to three years while further clinical effectiveness data are gathered. After this period the company will be asked to provide an updated submission for reassessment and SMC will make a decision on routine use of the medicine in NHSScotland.
Ultra-orphan pathway
1. Validation as ultra-orphan medicine
Companies are encouraged to seek confirmation that a medicine meets the ultra-orphan definition at an early stage, by completing an ultra-orphan proforma.
(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).
SMC will review the proforma and will confirm whether or not the medicine has been validated as ultra-orphan within around eight weeks (deadline for receipt of proformas is the first Monday of the month). Confirmation that the medicine is eligible for the ultra-orphan pathway is required before the company makes a submission for SMC initial assessment.
Ultra-orphan validation decisions will expire after 2 years. Thereafter if a product has GB marketing authorisation from the MHRA; if there are eligible patients in NHSScotland; and if no submission is forthcoming, SMC will move to issue Not Recommended advice.
2. Initial SMC assessment
An initial assessment of the clinical and cost-effectiveness of the medicine will be conducted. This will highlight uncertainties within the available evidence-base and will help to inform the data collection stage of the ultra-orphan pathway.
Companies should complete the New Product Assessment Form (NPAF) for Ultra-Orphan Medicines.
SMC will use a broad framework to appraise ultra-orphan medicines which takes account of the nature of the condition, impact of the medicine, value for money, impact beyond direct benefits and on specialist services, and costs to the NHS. Further information is available in SMC guidance to submitting companies – supplement for medicines for extremely rare conditions (ultra-orphan medicines).
3. Evidence generation
Data collection can begin at an early stage in parallel with SMC initial assessment. Guidance to support this phase of the ultra-orphan pathway is available on the Scottish Government website.
4. SMC reassessment
The company will be asked to provide a full updated submission following the period of data collection. This should include relevant new evidence for example: results of further controlled studies, observational, registry or other real world data gathered during initial availability of the medicine within the ultra-orphan pathway. SMC will then review information provided by the company as well as other sources of evidence e.g. from clinical experts, Patient Groups and, where relevant, the output from a Patient and Clinician Engagement (PACE) meeting, and will make a decision on routine use of the medicine in NHSScotland.
Refer to the ultra-orphan supplement (link above) for further details.