OBJECTIVES: In April 2014 the UK Medicines and Healthcare products Regulatory Agency (MHRA) launched the Early Access to Medicines Scheme (EAMS) to enable patients with severe, life-threatening diseases without adequate treatment options to access medicines prior to their marketing authorization. EAMS is a voluntary scheme whereby the medicine is provided free of charge by the manufacturer. EAMS comprises two key steps: (i) Promising Innovation Medicine (PIM) designation and (ii) EAMS scientific opinion. We aimed to systematically evaluate all EAMS appraisals to date.

METHODS: Publically-available EAMS documentation was screened from the MHRA website (to 08/06/2017).

RESULTS: Of 50 PIM designation applications, 68% were granted; 16% refused; 4% withdrawn, with 12% pending. Only 40% (20/50) of medicines granted PIM status had applied for an EAMS scientific opinion, 75% (15/20) of which were awarded. 73% (11/15) of awarded opinions were in oncology. 82% (9/11) oncology agents were for anti-PD-1/L1 therapies: nivolumab, pembrolizumab and atezolizumab. Only 1/15 was from a small-/medium-size enterprise (SME). 73% (11/15) awarded opinions had expired when EU marketing authorization was granted. Medicines were available under EAMS for an average of 93 days (range: 18–327). In the mean of 441 days (range: 128–720) since these EAMS opinions had expired, only 6/11 had been NICE-appraised (all recommended) and 8/11 SMC-appraised (75% accepted/restricted) at an average delay of 202 and 270 days post-EAMS expiry, respectively.

CONCLUSIONS: Over the past three years, EAMS has enabled medicines for nine drugs across fifteen indications (mostly within oncology) to be made available to patients with severe unmet prior to marketing authorization. However, this was for an average time period of only three months and was followed by an average delay of over six months until NICE/SMC appraisal completion. Reforming EAMS to ensure patients can continue to access therapies post-marketing authorisation until a NICE/SMC appraisal should be considered.