OBJECTIVES: The health technology assessment (HTA) bar is rising across the EU5 (France, Germany, Italy, Spain, UK). As payers’ balance clinical need with budgetary constraints, proving added benefit over currently available therapies is vital for new agents entering high-cost therapeutic markets. Focusing on key oncology indications, this study examined payer and physician perspectives on the importance of clinical trial design in HTA, reimbursement and prescribing.

METHODS: Across the EU5, 250 medical oncologists were surveyed regarding their current and expected prescribing patterns, and 15 payers who influence reimbursement nationally or regionally were interviewed.

RESULTS: For interviewed payers, robust head-to-head overall survival (OS) data showcasing benefit over appropriate existing agents are key to optimal reimbursement. They stress that crossover design frequently confounds OS data, leading to suboptimal HTA, and that agents with weak or marginal OS benefits, or with surrogate end-point data only, will likely require discounts/rebates/lowering of list prices to compete favorably in the EU5 markets. These payers advise that focusing on biomarker-defined subpopulations can be an effective means of optimizing clinical benefit to meet HTA demands in competitive markets. This study also finds clinical trial design to influence the prescribing of almost all surveyed oncologists to some extent. Approximately 75% of surveyed oncologists in Italy and Spain, and at least 60% in France and the UK indicate a strong impact of clinical trial design on their prescribing decisions. Specifically, OS and progression-free survival (PFS) as co-primary end points, appropriate comparator choice, and robust safety data are the key clinical trial design-related prescribing drivers identified by physicians surveyed.

CONCLUSIONS: Meeting well-considered end-points, ideally improvement in OS versus existing standard-of-care, is crucial for favorable reimbursement and strong uptake of oncology agents in the EU5. Drugmakers must ensure their clinical trials are designed to showcase unequivocal superiority over the right comparator to secure optimal market access.