PSY120 - Assessment of Commercial and Market Access Potential and Considerations for an Ultra-Orphan Drug for Renal Disease in EU5

Abstract

An orphan drug has the potential to achieve a premium price, due to small patient numbers and a high unmet need. This study looked at market access considerations and willingness to pay in the five major European markets (EU5), for a new drug in the ultra-orphan spectrum of end-stage renal disease (ESRD) with a significant mortality and cost burden. In-depth secondary research followed by interviews with KOLs and key Payer archetypes from EU5 markets were conducted to gain understanding of the clinical landscape, unmet need, value of a potential product for such a therapy area, evidence requirements for HTAs and price potential. Based on assessment of the clinical landscape, therapeutic positioning of a potential product profile was established, and target population estimated through a funneling exercise. Key value drivers (clinical and economic endpoints) were established. In general, end-points demonstrating mortality benefit over current standard-of-care and cost-savings reflect in their willingness to pay. Despite high clinical unmet need and KOLs placing a high value in a potential orphan drug, payers are generally unwilling to pay a premium price particularly for patients who already carry a significant underlying cost-burden. More so when a significant mortality benefit is not proven. Payers are not likely to be as lenient as they were towards orphan products and require strong evidence to back reimbursement decisions.