Presentation (CTI)

OBJECTIVES: ATMPs represent a paradigm shift in medical treatment. Supporting the clinical assessments of these drugs with traditional evidence, such as large RCTs of appropriate duration against an active comparator, represents challenges. The ethical, statistical, and scientific limitations have resulted in submissions using evidence associated with poorer reliability. Since 2017, 25 ATMPs that launched in key European markets secured reimbursement, using less traditional evidence. Data from open-label, single-arm non-randomized studies with external/indirect comparator data have been commonly used. Our research aims to examine whether over time, HTAs have evolved, and how this has impacted P&MA outcomes such as likelihood of reimbursement, access restrictions and time to reimbursement.
METHODS: We analysed ATMPs, including CAR-T and gene therapies authorized in the EU4 and UK from 2017-2025 using clinical trials, HTA assessments and GlobalData’s HTA/pricing databases. We extracted clinical trial design methodologies, presence and type of comparator, patient numbers and study duration. For each drug we examined reimbursement decision outcome, including any access restrictions beyond label. Time-to-access was measured from launch date to positive reimbursement decision. Country-specific pricing data was collected for each ATMP. We examine what, if any, correlations exist, and most importantly whether any of the parameters change over time.
RESULTS: Our analysis identified 20 ATMPs that secured reimbursement in key European markets. Analysis of one CAR T-cell therapy showed single-arm Phase II evidence (n=111) and used historical comparators which met European HTA standards. There was variability in reimbursement including conditional reimbursement, outcomes-based rebates and phased payment. Time to reimbursement ranged from 2 month to 12 months while annual cost was €245-327k.
CONCLUSIONS: Early results suggest that for drugs approved in later years, “lower quality” evidence of effectiveness is more likely to be associated with positive decisions and/or quicker time to access for ATMPs.