OBJECTIVES: NICE Highly Specialised Technology guidance only applies to England, which represents 80% of the UK population. We describe regional variations in gaining reimbursement for the estimated 70 nmDMD UK patients eligible for ataluren.

METHODS: We review the appraisal processes used to evaluate the clinical and cost-effectiveness of ataluren and the associated timelines to reimbursement and patient access.

RESULTS: Applications for reimbursement were made to England, Scotland, Wales, Northern Ireland (NI) and the Isle of Man. Detailed dossiers were submitted to NICE and SMC following different templates and requirements. EMA conditional marketing authorisation was granted in July 2014. Over a year later, the first patient received NHS-funded ataluren in Scotland via an Individual Patient Treatment Request. Individual funding requests elsewhere in the UK were unsuccessful. Following patient and political lobbying over a 13-month review, final positive NICE guidance for England was published under the conditions of a multi-stakeholder 5-year Managed Access Agreement between PTC, NICE, NHS England, the NorthStar clinician network, and the patient associations Muscular Dystrophy UK and Action Duchenne. NI and Wales endorsed NICE guidance under a patient access scheme (PAS). Despite negative SMC guidance following a shorter 5-month appraisal process, patient and political lobbying contributed to ataluren being reimbursed in Scotland with a PAS.

CONCLUSIONS: Market access in the UK does not start and stop at NICE or SMC. Through the different formal submission processes, patient and political lobbying were instrumental in achieving reimbursement outcomes across the UK. Many regions do not have fit-for-purpose processes for assessing products for ultra-rare conditions, as recognised by the recent Montgomery review in Scotland. The decision-making power of NICE and SMC in reviewing high-cost first-in-class ultra-orphan treatments is no longer clear in a regulatory and reimbursement environment where EMA assesses safety and efficacy whilst the NHS assesses willingness to pay.