OBJECTIVES: In April 2014 the Scottish Medicines Consortium (SMC) introduced a new framework for medicines licensed to treat an eligible population of 1 per 50,000 or less. Sponsor companies could provide a health economic analysis with a broader perspective within a framework of explicit decision-making criteria. Patient and clinician experience is also captured separately. Submissions assessed using this framework were reviewed to determine the type of economic analysis used and whether the wider societal costs and benefits of the medicine were incorporated

METHODS: Submissions assessed using the ultra-orphan framework published September 2014 - April 2017 were identified. The following data were extracted: category of medicine; basis of economic analysis; inclusion of wider costs and benefits within sensitivity analyses; and SMC acceptance rates.

RESULTS: Twenty seven submissions were assessed using the ultra-orphan framework. The majority (n=20) were cancer medicines used to treat very rare tumour types, in highly targeted mutations of common tumour types, or as last line therapies for advanced, refractory disease. The remainder were mainly medicines for very rare genetically acquired conditions. In all 27 submissions companies used conventional health economic analysis and sensitivity analysis incorporating wider costs and benefits was presented in only two submissions. Most of the cancer medicines were accepted for use (19/20) whereas the majority of non-cancer medicines were not recommended (6/7).

CONCLUSIONS: Three years’ experience of the new framework suggests a greater than intended range of medicines has been classed as ultra-orphan. Companies have generally not taken the opportunity to present a broader economic analysis for these medicines, providing a conventional cost-utility analysis in most cases. Work is underway to implement a revised SMC ultra-orphan definition to focus on medicines that treat extremely rare conditions.