OP81 Outcome-Based Managed Entry Agreements For Rare Disease Therapies: Learnings From The Belgian Experience

Sep 29, 2026

Uploaded by Oliver

Rising costs and limited evidence for rare disease therapies strain health technology assessment and payer decisions, prompting wider adoption of outcome-based managed entry agreements that tie post-launch evidence generation to payment. This study analyzes Belgium’s managed entry agreements for reimbursed rare disease therapies from January 2012 to August 2024, identifying OBMEAs, uncertainties, collected outcomes, and how clinical and real-world evidence influenced reassessment. Findings show coverage with evidence development, median 24-month duration, modest definitive listings, and frequent RWE rejection due to validity and registry gaps.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages1

Reading Time3 min

AccessFree to download

Introduction

Methods

Results

Conclusions

OP80 Cost-Utility Analysis Of Glucocorticoids Versus Standard Care For The Treatment Of Knee Osteoarthritis – Health Payer Perspective Danielle Stringer, Magdalena Moshi ([college.asernip@surgeons.org](college.asernip@surgeons.org)), Alvin Atlas, John Rathbone and Thomas Vreugdenburg Introduction: Knee osteoarthritis (OA) is a prevalent and debilitating condition with a significant impact on patient quality of life and healthcare costs. Intra-articular glucocorticoid injections (IAGI) are used in Switzerland for conservative management. A systematic literature review identified one existing economic evaluation on this topic globally. Therefore, the aim ofthis study was to evaluate the cost effectiveness ofIAGI in knee OA(relative to standard care)within the Swiss healthcare context. Methods: A cost-utility analysis was conducted using a healthcare payer perspective. Health outcomes were measured using qualityadjusted life year (QALY) estimates. Clinical outcomes were mapped into a preference-based utility measure. The clinical data showed IAGI only improved pain at one month but not beyond. Only direct costs in2024CHF were considered. A time horizon ofsix months was used to capture differences in costs and outcomes. Given the shortterm clinical improvement, only a single IAGI injection was modeled. Both costs and outcomes were discounted at three percent per annum. Both deterministic sensitivity analyses (DSA) and probabilistic sensitivity analyses (PSA) were conducted. The research project was funded by the Swiss Federal Office of Public Health. Results: The base case incremental cost-effectiveness ratio for IAGI in knee OA was CHF12,456 (USD15,735) per QALY gained. PSA showed a 71.9 percent probability of cost effectiveness at a hypothetical willingness-to-pay (WTP) threshold ofCHF50,000 (USD63,164) per QALY gained and 75.0 percent at CHF100,000 (USD126,322) . There was a 22.0 percent chance that IAGI is dominated by standard care (i.e., IAGI is more costly and less effective) . The DSA identified the key model drivers as IAGI administration costs and changes in health-related quality of life post-treatment. Conclusions: IAGI for knee OA appeared to be a cost-effective adjunct to standard care under hypothetical thresholds of CHF50,000 (USD63,164) and CHF100,000 (USD126,322) per QALY gained. Incorporating both DSA and PSA allowed for a robust exploration of uncertainties, highlighting critical drivers of cost effectiveness. Future research should refine long-term effectiveness estimates, particularly the impact of IAGI on progression to joint replacement, and evaluate real-world outcomes to ensure optimal resource allocation. OP81 Outcome-Based Managed Entry Agreements For Rare Disease Therapies: Learnings From The Belgian Experience Alessandra Blonda ([alessandra.blonda@kuleuven.be](alessandra.blonda@kuleuven.be)), Karen Facey, Inneke Van de Vijver, Yvonne Denier, Isabelle Huys and Steven Simoens Introduction: The rising costs of rare disease therapies pose significant challenges for health technology assessment (HTA) decisionmakers and payers, particularly given the limited clinical evidence available at the time of appraisal. This has created a growing need for outcome-based managed entry agreements (OBMEAs) that link post-launch evidence generation to appropriate payment models. This study examined the implementation of OBMEAs in Belgium and offers best practices to optimize their use globally. Methods: A document analysis was conducted of managed entry agreements (MEAs) for all rare disease therapies reimbursed between January 2012 and August 2024 . Through a standardized template, OBMEAs were identified and classified based on type of uncertainties included and outcomes tobe collected. HTA reports were analyzed to assess the impact of clinical and real-world evidence (RWE) on thereassessment of these therapies at the end of the OBMEA. Results: The Belgian payer implemented outcome-based managed entr

FAQs

Why are outcome-based managed entry agreements needed for rare disease therapies?

They address HTA and payer challenges arising from high costs and limited clinical evidence at the time of appraisal by linking post-launch evidence generation to appropriate payment models.

How was Belgium’s experience analyzed in this study?

A document analysis reviewed managed entry agreements for rare disease therapies reimbursed between January 2012 and August 2024, using a standardized template to identify OBMEAs and classify uncertainties and outcomes to be collected.

What were the key results regarding OBMEAs in Belgium?

Belgium implemented OBMEAs for 57 orphan drugs with a median duration of 24 months, while only 8% led to definitive listing; real-world evidence was often rejected during reassessment due to concerns about validity and incomplete registries.

What recommendations does the study provide to improve OBMEAs?

Establish clearer data collection protocols and improve transparency across multiple levels to enhance the effectiveness of these agreements, especially in addressing long-term uncertainties.

Sep 29, 2026

Uploaded by Oliver