OBJECTIVES: The Early Access to Medicines Scheme (EAMS) launched in April 2014 with the aim of providing patients with severe, life-threatening diseases without adequate treatment options to access to medicines prior to their marketing authorization. Thus, for products with an EAMS designation, there will be real world evidence (RWE) with the product prior to health technology assessment (HTA) decision-making by the National Institute for Health and Care Excellence (NICE). This research aimed to assess if, and how, EAMS data is being utilised in the NICE appraisal process.

METHODS: All EAMS designations as of 26/06/2017 were identified from the MHRA website. For products with final NICE guidance, the full manufacturer submission, as well as all publically available NICE documentation was reviewed.

RESULTS: CONCLUSIONS: Our research shows that experience of products under EAMS has primarily been used to support patient and physician advocacy during the appraisal process. Currently, no RWE outcomes data has been collected while a product was available through the EAMS scheme has utilised to support NICE submissions.