OBJECTIVES

Since October 2018, ultra-orphan therapies are subject to a new decision-making pathway in Scotland. Under this pathway, ultra-orphan medicines (definition: prevalence <1:50,000) would be made available for ≥3-years whilst additional evidence is collected to inform a final Scottish Medicines Consortium (SMC) appraisal. Prior to this, the SMC had an ultra-orphan medicine evaluation process, with a broader decision-making framework versus the standard appraisal process. This research aims to evaluate the pre-2018 SMC ultra-orphan medicine evaluation process by identifying all drugs assessed under this process and comparing their outcomes with the National Institute for Health and Care Excellence (NICE) (which has the Highly Specialized Technology [HST] program for evaluation of ultra-rare therapies).

METHODS

Publicly-available SMC guidance was screened (13/10/2014-08/04/2019) and any appraisals under the ultra-orphan process identified (alongside any corresponding NICE guidance) and key information extracted.

RESULTS

51 SMC appraisals under the pre-2018 ultra-orphan pathway were identified (average of 10 per year). 59% were accepted, 22% were optimized, and 20% were not recommended. 65% (33/51) had been assessed by NICE (although only four under the HST), with another five appraisals ongoing. 97% (32/33) NICE final appraisals were either recommended, optimized, or recommended for the Cancer Drugs Fund.

CONCLUSIONS

Although recommendation rates under the pre-2018 SMC ultra-orphan process were 80%, most of these had also been assessed by NICE, almost all of which received a positive recommendation. Further, very few of these NICE appraisals were under the HST, i.e. these high NICE recommendation rates were achieved via assessment under the standard appraisal pathway. Further research can compare how SMC recommendations of ultra-orphan medicines will evolve according to other countries following the new decision-making pathway.