This study seeks to inform manufacturers of advanced therapy medicinal products (ATMPs) in the Big5EU about the key drivers for securing adoption at a commercially viable reimbursed price through identification of relevant pricing, reimbursement and market access (P&R&MA) considerations.The research strategy: 1) Using secondary research to identify the market access pathways relevant to ATMPs 2) Conducting qualitative interviews with national, regional, and local-level payers and their relevant advisors to validate the secondary research findings and address any data gaps in the primary research 3) Compare the results across countries by consolidating all the primary and secondary findings P&R&MA processes across different geographies are largely based on the incremental clinical benefit. Budget impact is crucial factor, regardless of geography. While cost-effectiveness analyses are increasingly being utilized, only the United Kingdom has a defined threshold that links the cost per quality-adjusted life year (QALY) to the reimbursed price. Funding mechanisms for new and high-cost therapies are available in all countries, albeit with varying degrees of coverage. Smaller patient populations with high disease burden tend to have higher willingness to pay. To address data gaps and uncertainties associated with trials in niche populations, outcomes modelling and risk-sharing agreements (RSAs) can be employed as strategies. The challenges associated with the clinical adoption of ATMPs at a commercially viable reimbursed price is associated with their high-development cost and the uncertainty around their long term claims. To best capitalise on patient healthcare cost offsets and maximise clinical benefit of the product, it is advantageous to target populations of high disease burden and unmet needs. Furthermore, reducing both payers’ budget impact concerns and reimbursement risk can be realised by targeting small populations. |