A Comparative Analysis of Similarities and Differences of RWE Standards Across Regulatory and Health Technology Assessment Bodies
Author(s)
Christianna Pepingco, MPH1, Antonio Cardoso, MSc2, Kathryn Plante, MS3.
1Market Access Consulting & HEOR, Fortrea Inc., Durham, NC, USA, 2Fortrea Inc., Lisbon, Portugal, 3Fortrea Inc., Durham, NC, USA.
1Market Access Consulting & HEOR, Fortrea Inc., Durham, NC, USA, 2Fortrea Inc., Lisbon, Portugal, 3Fortrea Inc., Durham, NC, USA.
OBJECTIVES: Real-world evidence (RWE) is becoming increasingly important in regulatory decision-making and health technology assessments (HTAs); however, standards vary across governmental bodies. This review compares RWE standards across two regulatory bodies—the FDA and EMA— and two HTA bodies—NICE and CDA-AMC.
METHODS: This systematic review evaluated RWE guidance documents from the FDA, EMA, NICE, and CDA-AMC. Key areas of interest included: data quality criteria, fit-for-purpose applications, registry requirements and external control arms acceptance. We reviewed RWE guidance documents and extracted data on similarities and differences regarding methodological standards and decision-making frameworks.
RESULTS: The FDA and EMA emphasize conduct of source data verification and regulatory compliance standards. For fit-for-purpose applications, the FDA applies more flexibility evaluating RWE as the basis for drug approvals compared to the EMA. Variations exist in RWE acceptance for external control arms; the FDA is less restrictive compared to the EMA, though both are increasingly accepting these methodologies. Compared to the FDA, the EMA demonstrates an advanced approach in registry integration maturity and governance expectations, likely due to established registry and data sharing networks. Regarding HTA, NICE focuses on cost-effectiveness and clinical benefit using RWE while CDA-AMC focuses on healthcare system impact and budget considerations. NICE emphasizes data accuracy over comprehensiveness, while CDA-AMC focuses on representativeness and provincial data consistency. CDA-AMC faces challenges in coordinating provincial systems, affecting uses of RWE.
CONCLUSIONS: While regulatory entities generally share RWE principles for data robustness and validity, harmonizing policies poses challenges, reflecting variation in regulatory mandates and decision-making frameworks. Likewise, variation is apparent in RWE uses in HTA. Better alignment across regulatory bodies may be needed to optimize global product development and approvals where RWE may be used. In contrast, HTA uses may be region-specific as seen with CDA-AMC. Future research should incorporate additional regulatory agencies and HTA bodies.
METHODS: This systematic review evaluated RWE guidance documents from the FDA, EMA, NICE, and CDA-AMC. Key areas of interest included: data quality criteria, fit-for-purpose applications, registry requirements and external control arms acceptance. We reviewed RWE guidance documents and extracted data on similarities and differences regarding methodological standards and decision-making frameworks.
RESULTS: The FDA and EMA emphasize conduct of source data verification and regulatory compliance standards. For fit-for-purpose applications, the FDA applies more flexibility evaluating RWE as the basis for drug approvals compared to the EMA. Variations exist in RWE acceptance for external control arms; the FDA is less restrictive compared to the EMA, though both are increasingly accepting these methodologies. Compared to the FDA, the EMA demonstrates an advanced approach in registry integration maturity and governance expectations, likely due to established registry and data sharing networks. Regarding HTA, NICE focuses on cost-effectiveness and clinical benefit using RWE while CDA-AMC focuses on healthcare system impact and budget considerations. NICE emphasizes data accuracy over comprehensiveness, while CDA-AMC focuses on representativeness and provincial data consistency. CDA-AMC faces challenges in coordinating provincial systems, affecting uses of RWE.
CONCLUSIONS: While regulatory entities generally share RWE principles for data robustness and validity, harmonizing policies poses challenges, reflecting variation in regulatory mandates and decision-making frameworks. Likewise, variation is apparent in RWE uses in HTA. Better alignment across regulatory bodies may be needed to optimize global product development and approvals where RWE may be used. In contrast, HTA uses may be region-specific as seen with CDA-AMC. Future research should incorporate additional regulatory agencies and HTA bodies.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR5
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways
Disease
No Additional Disease & Conditions/Specialized Treatment Areas