OBJECTIVES: Early Access to Medicines Schemes (EAMS) was established in the UK in 2014 to allow cohorts of patients with severe diseases with no alternatives, access to unlicensed therapies in clinical development. The manufacturer must provide the medicine free of charge and must pay a total application fees of £29,267 (including the preliminary PIM). In France, the cohort-ATU is a similar scheme established since 1994 but where manufacturers can charge. This research compares the drugs available under the EAMS vs. cohort-ATU schemes.

METHODS: Medicines accepted for use under EAMS and cohort-ATUs were identified from the relevant websites and key information was extracted (01/01/2015-27/12/2018).

RESULTS: 22 drug:indication pairings have been accepted onto EAMS (average 5.5/year, range: 4[2018]-7[2017]). 86% (19/22) have expired after a mean 95 days (range:19-308). The majority, 9% (13/22) were for oncology and 18% (4/22) were orphan indications. In the corresponding time frame, 49 drug:indication pairings attained cohort-ATUs (average:9.8/year 8[2016]-17[2018]). 65% (32/49) have closed after a mean of 261 days (range:61-1001 days). 41% (20/49) were for oncology and 35% (17/49) were orphan indications. 5 drug:indication pairings (dupilumab, emicizumab, patisaran, sacubitril/valsartan, and venetoclax) were both accepted onto cohort-ATUs and EAMS. Overall, these drugs came onto the cohort-ATU an average of 6.2 days earlier than EAMS (range:-153 to +113). However, the time on cohort-ATU scheme was numerically higher than on EAMS (mean: 114 vs. 94 days). Whereas, time of EAMS closure is tightly linked to EC-approval date (mean difference: +1.2 days), cohort-ATUs extend beyond EC-approval date (mean difference: +88 days).

CONCLUSIONS: The EAMS and cohort-ATU schemes appear to attract a largely distinct group of therapies and the cohort-ATU scheme has attracted over double the drug:indication pairings than EAMS. The ability to charge may be driving this. Further cohort-ATUs formally extend beyond EC-approvals, preventing any gap in patient access between EC-approval and reimbursement decisions.