How Does The Scottish Medicines Consortium Assess The Value Of Orphan And Ultra-Orphan Drugs?

Abstract

Orphan drugs rarely achieve standard cost-effectiveness thresholds due to high acquisition costs and a paucity of clinical trial data. The Scottish Medicines Consortium (SMC) has no stated threshold for such treatments. The National Institute for Health and Care Excellence (NICE) recently proposed guidelines for highly specialised technology (HST) appraisal for very rare conditions in which a threshold of £300,000 per quality-adjusted life-year (QALY) would be employed. This study investigates whether the SMC use an implicit cost-effectiveness threshold and any alternative aspects of value considered when assessing orphan drugs. The SMC database was searched from January 2015 to May 2017 for submissions made under the Orphan or Ultra-Orphan submission processes. Data were extracted regarding the submission process, SMC recommendations, use of patient access scheme (PAS), and incremental cost-effectiveness ratio (ICER). If a with-PAS ICER was unavailable, the without-PAS ICER was extracted. Patient and Clinician Engagement (PACE) criteria were reviewed to assess additional aspects of value taken into account by the SMC. A total of 49 submissions were identified. The SMC accept technologies for orphan drugs with ICER values around £50,000 per QALY gained. There were 15 submissions accepted with ICERs above £30,000 per QALY, the highest acceptance being £56,000 per QALY gained. The SMC reject submissions lacking satisfactory rigour even when the ICER is substantially below £50,000 per QALY. In cases with high ICER values, other frequently cited aspects of value include age of the population, whether treatment may facilitate return to work, carer burden, delay in disease progression, and unmet need. NICE’s proposed threshold for orphan diseases may lead to disparities in access to treatments across the United Kingdom. Though the SMC does incorporate PACE criteria considering additional aspects of value, transparency of SMC requirements for reimbursement of orphan drugs would be welcomed by Scottish patient groups and submitting companies.