Ultra-orphan medicines

A new approach to the assessment of ultra-orphan medicines is being introduced in Scotland following the Scottish Government announcement in June 2018.


Read the Scottish Government announcement

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. 

* SMC uses the description of the condition within the European Medicines Agency’s (EMA) Orphan Maintenance Assessment Report (OMAR) as a reference (or the description within 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 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.

New 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, and ideally prior to receiving an EMA Committee for Human Medicinal Products (CHMP) opinion, by completing an ultra-orphan proforma.

Download the 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. 

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.

Download the ultra-orphan NPAF form


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).

Submissions received from April 2019 onwards will be assessed using the new approach. The previous ultra-orphan process (pre April 2019) is now closed for submissions and resubmissions. 

Download the ultra-orphan supplement 


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.

 

View guidance


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.