OBJECTIVES: Patient access to potentially life-saving treatments through clinical trials (CTs) is at risk due to Brexit (March 2019). Currently, all CTs conducted in the UK require approval by the MHRA in line with EU Directive 2001/20/EC, which will be replaced by EU Regulation 536/2014 in 2019. This research aims to examine the impact on patient access to pioneering and innovative therapies through CTs in the UK.

METHODS: Publically available ABPI, clinicaltrials.gov, EFPIA, EMA, EudraCT, MHRA and NIHR data were screened up to 16/06/2017 for key information including the number of CTs involving the UK and EU Member States (MSs).

RESULTS: Approximately 4,000 CTs are conducted annually in the EEA, each involving on average 2 MSs. The UK is the second-ranked MS in terms of number of commercially sponsored CTs (85/year, 2010-2015) behind only Germany (90/year). Application of the new regulation will impact the number of the pan-European CTs with the UK as a MS (currently 6,585 ongoing CTs out of 30,506 in the EU). Approximately 605,000 individuals were involved in CTs in the UK in 2015/6, including 35,000 in commercially-sponsored CTs. A substantial number of those will have been enrolled in phase II/III trials - in 2015, 622/842 UK commercial CTs applications were for phase II/III trials, with only Germany having more phase II/III trials.

CONCLUSIONS: Brexit could potentially deny thousands of UK patients the access to pioneering and innovative candidate treatments as UK trial sites will no longer need to align to EU CT regulations. Consequentially, results of CTs conducted outside the UK will be less directly applicable to patient populations for NICE/SMC/AWMSG evaluations. The UK will either need to try and maintain harmonisation with the EU or create a more efficient and simpler regulatory process to ensure the UK remains a primary site to host CTs.