How Can We Enhance the Use of RWE in Reimbursement Decisions? Best Practices and Recommendations
Author(s)
Sian Besley, BSc, MSc1, Amanda Cole, BSc, PhD1, Raoh-Fang Jasmine Pwu, MS, PhD2, Jason Chia-Hsun Hsieh, PhD3, Tan Chuen Seng, BSc, MSc, PhD4.
1Office of Health Economics, London, United Kingdom, 2Fu Jen Catholic University, New Taipei City, Taiwan, 3Chang Gung Memorial Hospital, Taipei, Taiwan, 4Saw Swee Hock School of Public Health, National University of Singapore, City of Singapore, Singapore.
1Office of Health Economics, London, United Kingdom, 2Fu Jen Catholic University, New Taipei City, Taiwan, 3Chang Gung Memorial Hospital, Taipei, Taiwan, 4Saw Swee Hock School of Public Health, National University of Singapore, City of Singapore, Singapore.
OBJECTIVES: Innovative medicines are increasingly challenging our standard paradigms of evidence generation. Real-world evidence (RWE) can better inform reimbursement decisions in the presence of uncertainty, but is currently underutilised. This research explores best practices for the use of RWE by health technology assessment (HTA) bodies. We examine the challenges that hinder the routine use of RWE, and make recommendations for increasing its credibility, transparency, and ultimately its use in decision-making.
METHODS: We conducted a targeted literature review, focusing on guidance from established HTA bodies and highly cited academic publications. We identified common themes and actionable recommendations for improving the generation and use of RWE in HTA. We used Taiwan as a case study to explore how these insights apply in a real-world context.
RESULTS: We identified persistent barriers to the use of RWE, including concerns over internal validity, risk of bias, and lack of methodological transparency. Several guidelines and frameworks have been produced with recommendations coalescing around the need for data of sufficient quality to answer the research question; transparent evidence generation from planning through to study conduct and reporting; and the use of analytical methods to minimise bias and appropriately characterise uncertainty. The Taiwan case study demonstrated that - even with good access to de-identified data and established registries - opportunities remain to improve data quality and scientific rigor. It underscored the need for better infrastructure, stronger governance, and more systematic approaches to enable robust RWE generation that can inform local reimbursement decisions.
CONCLUSIONS: Significant work remains to standardize and strengthen the role of RWE, ultimately supporting better informed and more responsive access decisions for medicines. To fully realize the potential of RWE in reimbursement decision-making, we urge stakeholders internationally to work together to advance the use of high-quality real-world data, thereby promoting trust in its use for reimbursement decision-making.
METHODS: We conducted a targeted literature review, focusing on guidance from established HTA bodies and highly cited academic publications. We identified common themes and actionable recommendations for improving the generation and use of RWE in HTA. We used Taiwan as a case study to explore how these insights apply in a real-world context.
RESULTS: We identified persistent barriers to the use of RWE, including concerns over internal validity, risk of bias, and lack of methodological transparency. Several guidelines and frameworks have been produced with recommendations coalescing around the need for data of sufficient quality to answer the research question; transparent evidence generation from planning through to study conduct and reporting; and the use of analytical methods to minimise bias and appropriately characterise uncertainty. The Taiwan case study demonstrated that - even with good access to de-identified data and established registries - opportunities remain to improve data quality and scientific rigor. It underscored the need for better infrastructure, stronger governance, and more systematic approaches to enable robust RWE generation that can inform local reimbursement decisions.
CONCLUSIONS: Significant work remains to standardize and strengthen the role of RWE, ultimately supporting better informed and more responsive access decisions for medicines. To fully realize the potential of RWE in reimbursement decision-making, we urge stakeholders internationally to work together to advance the use of high-quality real-world data, thereby promoting trust in its use for reimbursement decision-making.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD20
Topic Subcategory
Data Protection, Integrity, & Quality Assurance
Disease
No Additional Disease & Conditions/Specialized Treatment Areas