OBJECTIVES

The National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) assess the clinical and cost-effectiveness of health technologies in the UK. The objective of this study is to compare the outcomes of NICE and SMC appraisals for ultra-orphan medicines to understand whether the two bodies are aligned in their decision making, how this has evolved over time and the reasons behind any divergences.

METHODS

SMC decisions published between October 2014 and May 2019 utilising the ultra-orphan decision making framework were cross-referenced to equivalent NICE technology appraisal or highly specialised technology (HST) guidance. Withdrawn or terminated submissions were not included. A total of 29 equivalent assessments were identified and key data related to the submission process and outcome extracted. Prices in the UK were acquired for each medicine from the IHS pricing database.

RESULTS

NICE and SMC decision-making for ultra-orphan medicines was aligned in the majority (23/29; 79%) of cases and concordance between the two agencies remained stable over time. Of the six ultra-orphan medicines with opposing decisions, five were accepted by NICE but not recommended by the SMC. All five were approved under either the NICE HST programme (3 in total) or the cancer drugs fund (CDF; 2 in total). Price level developments were analysed on a case by case basis due to variability in mechanism of action and effect size.

CONCLUSIONS

NICE and SMC decision-making for ultra-orphan medicines is aligned in the majority of cases. Our research highlights the role of the NICE HST pathway and CDF in ensuring access to ultra-orphan medicines in England and Wales. The April 2019 reforms to the SMC ultra-orphan pathway may improve access in Scotland in future and impact concordance between NICE and SMC appraisals.