OBJECTIVES: As governments throughout the EU adjust their policies to address healthcare deficits, drug manufacturers are facing increasing pricing pressures that could hinder patient access. While all drug classes are at risk, cell and gene therapies (CGTs) are exceptionally vulnerable due to their high cost and price. In this poster, we identify and examine the critical success factors for pharmaceutical companies to successfully navigate Europe’s cost-saving environment during launch planning.

METHODS: An extensive literature review was conducted of regulatory decisions over the past 5 years to evaluate critical success factors, opportunities, and challenges for European reimbursement negotiations. An “Access Checklist” has been developed which serves to guide future launches, encompassing effective clinical trial design and pricing strategy.

RESULTS: Our research found three pillars as the linchpin to successfully navigate Europe’s cost-saving environment and enable patient access to therapy: 1) strategic evidence generation, 2) optimized use of OBAs and 3) leveraging RWE. Successful implementation of these pillars is challenging. In theory, there is a desire to achieve optimized use of OBAs and, despite payers in EU markets expressing a need for OBAs to cope with high costs, there remain structural barriers. For example, in Germany, where legislators introduced a cost containment bill to reduce the nation’s €17B healthcare deficit payers are calling for administrative simplicity to make OBAs cheaper and easier to implement. Manufacturers must work in partnership with payers to lobby and co-create conditions that enable the latent potential of OBAs to flourish.

CONCLUSIONS: To ensure an effective launch we have developed an “Access Checklist” to help navigate the structural barriers for OBAs as well as challenges associated with RWE evidence inclusion. These must be started at an early stage, alongside clinical trial design, to establish synergy between the launch conditions and product profile.