OBJECTIVES

: Early Access Programs allow cohorts of patients with severe diseases with no therapeutic alternatives, access to unlicensed therapies in clinical development. The schemes in the UK, France and Sweden are the Early Access to Medicines Schemes (EAMS), cohort Temporary Authorization for Use (cohort-ATU) and Compassionate Use Program (CUP), respectively. Their main distinction relates to reimbursement: the cohort-ATU allows manufacturers to charge whereas under EAMS and CUP the manufacturer must provide for no charge. This research compares the drugs available under EAMS, cohort-ATU, and CPU schemes.

METHODS

: Medicines accepted for use under EAMS, CPU, 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. 86% (19/22) have expired after a mean 95 days (range:19-308). 59% (13/22) were for oncology and 18% (4/22) had orphan designations. 49 drug:indication pairings attained cohort-ATUs. 65% (32/49) closed after a mean of 261 days (range:61-1001 days). 41% (20/49) were for oncology and 35% (17/49) had orphan designations. 7 drug:indication pairings have been accepted onto CUP. 56% (4/7) were for oncology and 14% (1/7) had orphan designations. Only 2 drug:indication pairings (patisiran and venetoclax) were accepted onto cohort-ATUs, CUP, and EAMS. Despite similar start dates, time on cohort-ATU scheme was numerically higher than on EAMS and CUP (mean: 114 vs. 94 vs. 75 days).

CONCLUSIONS

: The EAMS, CUP and cohort-ATU schemes appear to attract a largely distinct group of therapies and the cohort-ATU scheme has attracted over double and triple the drug:indication pairings than EAMS and CUP respectively. 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.