Interim Access in Scotland: Successful Innovation or a Cautionary Tale?

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. Since 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 systematically evaluates medicines with interim acceptance decisions and medicines under the ultra-orphan pathway.

METHODS: Interim acceptance and outcomes of ultra-orphan medicines were identified from the SMC website (30-Jan-2024).

RESULTS: A total of 10 products have received an interim acceptance decision (3 in 2020, 2 in 2021, 3 in 2022, 2 in 2023), while 12 products have been accepted via the ultra-orphan pathway (3 in 2020, 1 in 2021, 3 in 2022, 5 in 2023). 6/10 (60%) of interim acceptance decisions and 2/12 (17%) of ultra-orphan medicines were approved based on non-comparative data. None of the products with interim acceptance has received a re-assessment outcome, although the data collection deadline has passed for 2 products. For ultra-orphan medicines, only 1 has been re-evaluated with a positive recommendation, while the data collection deadline has passed for 2 other products with no re-assessment outcome available yet (1 in progress).

CONCLUSIONS: The interim acceptance decision and ultra-orphan pathway have provided access to medicines whilst evidence generation activities are ongoing, including products that launched with non-comparative data, with one example of successful re-assessment. However, the re-assessment delay for several products suggest potential challenges with the process and/or quality of the data collection. It would 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.