OP81 Outcome-Based Managed Entry Agreements For Rare Disease Therapies: Learnings From The Belgian Experience
Alessandra Blonda, Karen Facey, Inneke Van de Vijver, Yvonne Denier, Isabelle Huys, Steven Simoens · 2025
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Description
Alessandra Blonda and five co-authors review Belgium's experience with pay-for-results deals for rare disease drugs. Such therapies cost a great deal and arrive with thin evidence, so payers increasingly tie payment to later proof. The team read every managed entry agreement for rare disease therapies reimbursed in Belgium from 2012 to mid-2024. They found 57 outcome-based deals, including five advanced therapy products, all built as coverage while evidence develops.
Budget impact and true effectiveness were the main uncertainties the deals targeted. The typical deal ran two years, though some were renewed up to five times, and only eight percent ended in a firm listing. Real-world evidence was often thrown out at review for weak methods or patchy registries. The authors recommend clearer data rules and more open reporting from the start.
Catalog record
| CREATOR | Alessandra Blonda, Karen Facey, Inneke Van de Vijver, Yvonne Denier, Isabelle Huys, Steven Simoens |
|---|---|
| YEAR | 2025 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 2 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |