OBJECTIVES: In 2014, the UK Medicines and Healthcare products Regulatory Agency (MHRA) launched schemes to identify and enable patient access to promising medicines prior to marketing authorisation. Promising innovative medicine (PIM) designation and the early access to medicines scheme (EAMS) provide patients with seriously debilitating or life-threatening conditions, access to medicines where there is a clear unmet medical need. This research aimed to establish whether having this designation may accelerate access to innovative medicines and whether it increases the likelihood of gaining a positive recommendation during the subsequent NICE appraisal process.

METHODS: NICE appraisals and PIM and EAMS statistics were analysed through desk research. All assessments up to May 2018 were reviewed to ascertain the designation of PIM and EAMS before the appraisal, the assessment outcome and timelines from PIM and EAMS to review.

RESULTS: PIM designation will be issued after an MHRA scientific designation meeting on the basis of non-clinical and clinical data available on the product, in a defined disease area. There have been 51 PIM designations granted, 12 refused, 2 withdrawn and 7 still pending. The scientific opinion supports both the patient and the prescriber in order to make a decision regarding the use of the medicine prior to any licensing approval. Regarding EAMS there have been 19 awarded, 2 refused and 3 opinions still pending. Of all the appraisals identified with a PIM designation, all have received a recommendation, some with restrictions or through the Cancer Drugs Fund. Of these appraisals, 7 had not gone through EAMS.

CONCLUSIONS: Being awarded a PIM designation and going through EAMS is a positive initial step in gaining market access. This process highlights the severity and unmet need of the disease, looking into the therapeutic benefit provided to patients which is a strong basis for recommendation through the NICE appraisal.