OBJECTIVES: With the rapid development of Advanced Therapy Medicinal Products (ATMPs), managing the budget impact of introducing these high-cost therapies poses a key challenge for healthcare systems. Payers often restrict access to high-cost therapies due to uncertainties in clinical evidence, and to balance budgets and maintain healthcare sustainability. This analysis aims to understand the relationship between clinical evidence requirements and HTA reimbursement/pricing outcomes for ATMPs launched in Germany, France, and the UK over the past five years.

METHODS: ATMPs with marketing authorisation from 2017 onwards were identified from EMA and MHRA databases. HTA evaluations, including trial evidence, drug pricing, and reimbursement outcomes in the scope markets were collected from relevant HTA-body websites.

RESULTS: Of the 17 ATMPs with regulatory approval across selected markets, 10 had Orphan Drug Designation. Using Upstaza as an example, it received a considerable added benefit in Germany and ASMR III/SMR Important in France, with an annual list price of ~€3.5M in both markets. In the UK, it was also recommended but priced at €2.6M annually. This pricing discrepancy highlights the UK's emphasis on cost-effectiveness and variability in acceptance for single-arm, on-going trial studies.

CONCLUSIONS: The analysis highlights the significant variability in HTA outcomes and pricing for ATMPs across Germany, France, and the UK. These findings emphasize the importance of trial design, including factors like open-label status or the use of a natural history comparator arm, in influencing payers’ willingness to pay. Understanding these key drivers is crucial for assessing the robustness of trial evidence required to achieve a certain level of added clinical benefit. This knowledge informs pricing strategies and highlights the additional evidence needed to secure optimal HTA reimbursement and expedite patient access.