RWD226 MULTIDIMENSIONAL BURDEN OF NEUROFIBROMATOSIS TYPE 1 (NF1): A SYSTEMATIC LITERATURE REVIEW

Objectives: This study aims to analyze the implementation and termination characteristics of Managed Entry Agreements (MEAs) for new drugs under Taiwan's National Health Insurance (NHI) system.MEAs are contractual arrangements between pharmaceutical companies and payers to address uncertainties in financial impact and clinical efficacy, especially for high-cost drugs targeting rare diseases or advanced-stage cancers.The research focuses on trends in MEA adoption, termination scenarios, and factors influencing agreement signing.Methods: A crosssectional retrospective observational study was conducted using publicly available records from Taiwan's NHI Pharmaceutical Benefit and Reimbursement Scheme Joint Committee Meeting between 2019 and 2023.Data on drug characteristics, financial impact estimates, clinical trial phases, and agreement types were extracted and validated by independent reviewers.Statistical analyses, including descriptive statistics and chi-square tests, were performed to identify factors affecting MEA adoption.Results: Of the 120 cases analyzed, 45.8% involved signed MEAs, while 50.8% remained undisclosed due to confidentiality.MEAs based on financial arrangements dominated (49.1%), followed by efficacy-based agreements (10.9%).Agreements were predominantly signed for two-year terms, with fixeddiscount schemes being the most common financial arrangement (54.6%).Termination scenarios revealed that 23.6% of agreements were renewed at reduced prices, while 58.2% had unknown termination statuses.Factors significantly influencing MEA adoption included drug classification (p = 0.0014), clinical trial phase (p , 0.0001), therapeutic category (p , 0.0001), and estimated financial impact over three and five years (p , 0.0001).Conclusions: MEAs have become a critical policy tool for balancing the risks of new drug reimbursement with NHI resource optimization in Taiwan.While financial-based agreements remain prevalent, efficacybased agreements are gradually increasing due to advancements in real-world evidence collection and electronic medical records.