OBJECTIVES: Since 2001, 5 products have been given a CHMP opinion for compassionate use at EMA level, while in member states (MS), at any one time there are tens of products listed on compassionate use programs (CUP). Here we investigate the difference in EU to MS CUP listing processes and discuss what that means to a patient’s ability to access innovative therapies early.

METHODS: Research focused on EMA and MS CUP lists and assessment documentation (where available), as well as targeted literature sources. Countries: France, Germany, Italy, the Netherlands, Spain, Sweden and the UK.

RESULTS: Of 28 European Union (EU) MS, 20 have CUP in place, 18 of these have national-level regulations; the remaining 10 have none. In the UK, the Early Access to Medicines Scheme (EAMS) has seen 41 submissions to date, but only 14 approvals. In 2016, the temporary authorisation for use (ATU) program in France approved 201 products, from therapeutics to devices and vaccines. The most common products to gain access to CUPs are those for oncology. Alectinib, gained access to French, German and Dutch CUPs in 2016, but not EAMS, whilst Nivolumab (for lung and kidney cancers) gained access to UK and Dutch CUPs (2015) but not French or German.

CONCLUSIONS: There is considerable disparity between CUPs across MS for innovative treatments that target high unmet need conditions. Avoidance of EU-wide schemes by manufacturers is perhaps to be expected given the cost of CUP assessment processes, and supply of products for free to patients – a regulatory stipulation. However, the result is that only patients in targeted countries gain access to innovative therapies. While CUPs offer the chance to build clinical experience and real-world data, the targeting of MS for CUP launch could be creating an early access lottery for patients.