OBJECTIVES: The 2016 Montgomery report raised concerns about the low acceptance rate for ‘true’ ultra-orphan (TUO) medicines compared to all UO medicines when appraised by the Scottish Medicines Consortium (SMC). We examined possible reasons for ‘not recommended’ SMC advice for TUO medicines and compared appraisals to those conducted by the National Institute for Health and Care Excellence (NICE).

METHODS: The SMC website was searched in April 2017 for detailed advice on all TUO medicines using the search term “ultra orphan”. TUO medicines were defined as per the definition used in the 2016 Montgomery report. The NICE website was also searched to identify equivalent appraisals for each intervention/indication pair.

RESULTS: Nine TUO medicines were identified, of which only two were accepted and one was restricted by the SMC (33% acceptance rate), despite the use of Patient and Clinician Engagement meetings and SMC decision modifiers in all appraisals. Of the six ‘not recommended’ medicines, incremental costs in pharmacoeconomic evaluations were typically in excess of £1 million, resulting in incremental cost-effectiveness ratios well above SMC thresholds, and clinical evidence from multi-arm randomised controlled trials was used in only two appraisals. Of the nine TUO medicines assessed by the SMC, four were appraised by NICE for the same indication. Three of these medicines received positive NICE guidance but were not recommended by the SMC; each of these three ‘NICE-recommended’ medicines were assessed via the Highly Specialised Technology process and included restrictions of use/a formal managed access agreement.

CONCLUSIONS: Compared to NICE, the existing appraisal process used in Scotland for UO medicines does not appear to substantially improve access to TUO medicines; as such, changes to the SMC UO appraisal process may be warranted. ‘Not recommended’ SMC advice is likely related to the high cost and limited evidence base for these medicines, as noted in the 2016 Montgomery report.