Fertile Grounds for Outcome-based Managed Entry Agreements?
Authors:   , Callenbach Marcelien Heidrun Elisabeth 
Published: February 2025

The rise of high-cost novel therapies, such as orphan medicinal products (OMPs) and advanced therapy medicinal products (ATMPs), presents both opportunities and challenges for healthcare systems. While these therapies may offer significant patient benefits, their high costs and uncertainties regarding long-term effectiveness complicate pricing and reimbursement decisions. Outcome-based managed entry agreements (OB-MEAs) have emerged as a potential solution by linking reimbursement to actual treatment performance, helping mitigate financial risks and cost-effectiveness uncertainties.
This thesis explores the potential of OB-MEAs through structured frameworks and stakeholder insights. A key finding is that while simpler financial-based MEAs are currently the most commonly used and may often be sufficient, OB-MEAs can provide added value for specific high-cost therapies with significant clinical and/or cost-effectiveness uncertainties. Prioritising therapies suitable for OB-MEAs early in the process increases the likelihood of successful implementation and ensures timely patient access under appropriate conditions.
Reimbursement challenges can arise from varying HTA evaluations and interpretations of clinical evidence. Harmonisation efforts, such as the upcoming EU HTA Regulation, highlight the need for greater transparency and alignment in assessment processes. Early collaboration between HTA bodies and health technology developers is essential to reducing uncertainties and improving patient access.
A structured approach was developed to identify therapies best suited for OB-MEAs, evaluating their potential benefit and clinical uncertainty. The findings show that a universal approach does not work—different therapies require tailored reimbursement strategies. While financial-based MEAs address many challenges, innovative OB-MEA combinations can offer additional advantages in managing uncertainties and ensuring sustainable patient access.
Stakeholder engagement is critical in determining the feasibility and desirability of OB-MEAs. Surveys and interviews with healthcare payers and industry experts indicate a preference for outcome-based reimbursement and delayed payment models. However, financial-based reimbursement and upfront payment models remain the most commonly used due to their simplicity. Barriers to OB-MEA adoption include administrative complexities, data infrastructure limitations, high transaction costs, and evidence generation and measurement challenges. Expanding OB-MEAs beyond high-income countries requires policy adaptations and improved data collection systems to overcome these obstacles.
To support structured decision-making, a calculation tool was developed to assess the financial and practical implications of different reimbursement models under various clinical and financial conditions. A real-life case study demonstrated that while OB-MEAs are complex, they can offer a pragmatic approach to managing treatment effectiveness uncertainties with the additional benefit of collecting real-world data on treatment response, leading to better resource allocation and improved patient access. A lifecycle approach to reimbursement ensures pricing strategies evolve alongside therapy development, further optimising access and cost management.
Conclusion
OB-MEAs can offer a promising way to balance healthcare sustainability with patient access to high-cost therapies. Their suitability depends on therapy-specific reimbursement challenges, with financial-based MEAs often being sufficient. However, when clinical uncertainties and financial risks are high, OB-MEAs can add value. By fostering stakeholder collaboration, implementing policy adaptations, and using structured decision-making tools, a more effective OB-MEA ecosystem can be created, aligning the interests of healthcare payers, HTDs, and patients, leading to sustainable access to novel therapies.