Outcomes-Based Agreements and Real-World Data for Health Technology Reassessment of Orphan Drugs: Learnings from Belgium

OBJECTIVES: This study aimed to characterize the use of coverage with evidence development (CED) and pay-for-performance (P4P) schemes in outcomes-based agreements for orphan drugs in Belgium. In addition, we analyzed whether real-world data (RWD) was collected and assessed by the Belgian healthcare payer (the National Institute for Health and Disability Insurance, NIHDI) and how it influenced health technology reassessment.

METHODS: We collected and analyzed the managed entry agreements (MEAs) for all rare disease (gene) therapies that were concluded up to May 2024 by the NIHDI. A standardized template was used to extract information on agreement duration, data requests, agreement type, P4P endpoints, and RWD provisions. HTA reports for all reassessments were requested to extract information on clinical and real-world data. Data were analyzed descriptively using Microsoft Excel.

RESULTS: We collected MEAs for 57 orphan drugs, including five advanced therapy medicinal products (ATMPs). All Belgian MEAs consisted of a CED scheme of which most data requests focused on budget impact (32%) and efficacy (28%). P4P schemes were included for the majority of cell and gene therapies. The median duration of initial agreements was 24 months, with extensions typically lasting 18 months. Despite considering RWD during reassessment, it was often rejected by NIHDI due to selection bias and incomplete data entry. Additionally, none of the agreements specified data sources or types, indicating a need for clearer guidelines on RWD requirements and improved data governance.

CONCLUSIONS: Our study underscores the potential of CED and P4P schemes in managing financial and clinical uncertainties for orphan drugs in Belgium. The collection and use of RWD are crucial, yet challenges in data validity and completeness persist. Enhancing data governance and providing clearer guidance on RWD collection are essential to improve these schemes' effectiveness. Addressing these aspects will facilitate better health technology reassessment and market access for innovative orphan drugs.