OBJECTIVES: In the United Kingdom, the health technology assessment (HTA) bodies, namely the National Institute for Health and Care Excellence (NICE) and Scotland's Scottish Medicines Consortium (SMC), both evaluate clinical and cost-effectiveness in their decisions. However, each organization utilizes distinct criteria, methodologies, and decision-making processes for assessing orphan medicines. These differences may lead to disparate decisions, causing varying acceptance rate for orphan drugs in these countries.To evaluate the variations in reimbursement status of nononcology orphan drugs assessed by NICE and SMC

METHODS: All HTA assessments conducted by NICE and SMC from January 2021 to December 2023 were extracted from the NAVLIN HTA Database. To identify relevant cases for analysis, products categorized as orphan drugs with therapeutic areas outside of oncology were isolated. Detailed information,including the drug name, indication, and decision, was extracted and organized to highlight key differences and similarities between the NICE and SMC appraisals.

RESULTS: Between January 1, 2021, and December 31, 2023, NICE and SMC conducted 62 assessments, with each organization evaluating 31 assessments. Out of these, SMC recommended 10 drugs for reimbursement, while NICE recommended 14. SMC did not recommend eight drugs, whereas NICE did not recommend two. Additionally, SMC provided optimized recommendations for six drugs, compared to 11 optimized recommendations by NICE. There were seven assessments with no submissions in SMC and four assessments with no submissions in NICE. Overall, there were 11 NICE Health Technology Assessment (HST) appraisals and five SMC Ultra-Orphan (UO) appraisals.

CONCLUSIONS: Overall, NICE issued more positive recommendations than SMC, and SMC encountered more instances of no submission from the Marketing Authorization Holder (MAH) compared to NICE. Approximately half of the fully recommended or optimized recommended decisions were made after considering input from a Patient and Clinician Engagement (PACE) meeting for SMC.