The Interim Acceptance and Ultraorphan Pathways in Scotland: An Update
Author(s)
Ethan J. Prince, MBiochem, Richard Macaulay, PhD.
Precision AQ, London, United Kingdom.
Precision AQ, London, United Kingdom.
OBJECTIVES: The Scottish Government’s Review of access to new medicines (2016) recommended that the Scottish Medicines Consortium (SMC) should have the option to accept a medicine for use subject to future reassessment. This process, known as interim acceptance, was introduced in 2018 and extended in 2021. In Oct 2018, the SMC also introduced the ultra-orphan pathway, allowing medicines targeting ultra-rare diseases to be reimbursed for up to three years while further evidence is generated for re-assessment. This research evaluates medicines with interim acceptance decisions and those in the ultra-orphan pathway.
METHODS: Interim acceptance decisions and medicines approved via the ultra-orphan pathway were identified from the SMC website (26-Jun-2025), including the decisions on reassessment (where available) and non-submissions (where applicable).
RESULTS: Since its inception, 12 products have received an interim acceptance decision. Two products have received a re-assessment outcome, with no others receiving this despite the data collection deadline having passed for 5 of 12 products; 1 product was globally discontinued. For the ultra-orphan pathway, 17 products have been accepted. Only 2/17 (12%) have been re-evaluated (both with a positive recommendations) and 1 (6%) was not recommended for use due to non-submission. However, the three-year evidence generation deadline has passed for 4 other products (24%), with no re-assessment outcome available yet.
CONCLUSIONS: The interim acceptance decision and ultra-orphan pathway have provided access to medicines whilst evidence generation activities are ongoing, with four examples of successful re-assessment across both schemes. However, there is a trend of delayed re-assessment for several products, suggesting potential challenges with the process and/or quality of data collection. It will be important to track future outcomes to examine whether these routes offer access to effective products, or risk exposing patients to costly therapies with no proven clinical benefits.
METHODS: Interim acceptance decisions and medicines approved via the ultra-orphan pathway were identified from the SMC website (26-Jun-2025), including the decisions on reassessment (where available) and non-submissions (where applicable).
RESULTS: Since its inception, 12 products have received an interim acceptance decision. Two products have received a re-assessment outcome, with no others receiving this despite the data collection deadline having passed for 5 of 12 products; 1 product was globally discontinued. For the ultra-orphan pathway, 17 products have been accepted. Only 2/17 (12%) have been re-evaluated (both with a positive recommendations) and 1 (6%) was not recommended for use due to non-submission. However, the three-year evidence generation deadline has passed for 4 other products (24%), with no re-assessment outcome available yet.
CONCLUSIONS: The interim acceptance decision and ultra-orphan pathway have provided access to medicines whilst evidence generation activities are ongoing, with four examples of successful re-assessment across both schemes. However, there is a trend of delayed re-assessment for several products, suggesting potential challenges with the process and/or quality of data collection. It will be important to track future outcomes to examine whether these routes offer access to effective products, or risk exposing patients to costly therapies with no proven clinical benefits.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA321
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure
Disease
Rare & Orphan Diseases