European pricing & access for an orphan haematology disorder​

• A US biotech was developing a new therapy for an ultra-rare disorder with a high mortality burden​
• The client required support defining the critical success factors for meeting commercial objectives in Europe (to inform a go/no-go launch decision)​
• Key considerations were the need for a range of financial/outcomes-based managed entry agreements, and the need for alignment of the list pricing strategy between the US and EU​

‍

• Define the requirements to support positive HTA outcomes and reimbursed patient access within the target list/net price range​
• Determine pricing and affordability models to reflect the likely clinical evidence available at launch and commercial objectives for the manufacturer ​
• Development of a roadmap to support European evidence generation, reimbursement and optimal pricing for the new therapy​

• Detailed analogue analysis of previous launches in Europe (including drivers/ challenges for payer value demonstration, pricing and managed entry agreements)​
• Payer primary research to explore the minimum/optimal evidence requirements to support patient access, feasible list/net pricing ranges and feasible innovative access agreements (considering clinical outcomes measurement and payment milestones)​

‍

• The client received a strategic report including critical success factors for meeting pricing and patient access objectives for the new therapy across priority European markets ​
• The report included an assessment of strategic priorities and associated tactics, including recommendations for innovative access agreements in priority European markets (and how these could be customised to fit specific healthcare systems/market dynamics) ​