Comparing Ultra-Rare Disease Pathways Within the United Kingdom: Can Different Processes Lead to Market Access Variations?

OBJECTIVES: Ultra-orphan medicines deemed clinically effective by the Scottish Medicines Consortium (SMC) are made available to patients subject to additional evidence generation through a revised pathway as of April 2019. We examined how the SMC pathway compares to the National Institute for Clinical Excellence (NICE) Highly Specialized Technology (HST) process in terms of timely access and Health Technology Assessment (HTA) recommendations.

METHODS: Medicines assessed through the revised SMC ultra-orphan pathway between October 2019 and May 2023 were identified and cross-referenced to NICE HST appraisals. Marketing authorization (MA) dates were extracted from the European Medicines Agency and the Medicines and Healthcare Products Regulatory Agency. HTA and prescribing dates, recommendations, decision rationale and restrictions, were extracted from NICE and SMC.

RESULTS: Seven ultra-orphan medicines were assessed and conditionally recommended by both HTA bodies. The average time from MA to HTA guidance by NICE and SMC was 821.57 and 891.43 days, respectively. Following SMC guidance, it took on average 107.33 days until the medicine was prescribed, with only 42% (3/7) of treatments being available quicker than the NICE-mandated 84 days. Whilst NICE and SMC raised the same concerns around the uncertainty of the medicines’ long-term clinical effectiveness (6/7; 86%), they disagreed as to whether the medicines were considered cost-effective (agreement: 1/7; 14%). Despite agreement on the clinical uncertainty, NICE required only 29% (2/7) of manufacturers to provide additional evidence, while simple price discounts were required in 86% (6/7) of cases. SMC conditionally recommended all seven medicines subject to additional evidence generation and a simple price discount.

CONCLUSIONS: NICE offers timelier access to ultra-orphan medicines than SMC. Despite both raising identical clinical uncertainties, NICE mostly requires simple price discounts and does not restrict access to these medicines subject to evidence generation. Such differences could potentially lead to access variations within the United Kingdom.