REAL-WORLD EVIDENCE TO SUPPORT HEALTH TECHNOLOGY ASSESSMENT AND PAYER DECISION MAKING: A REVIEW OF POLICY AND GUIDANCE ACROSS EUROPE, CANADA, AND THE UNITED STATES
Author(s)
Ines Abdelghani, PharmD1, Roua ABBES, Engineer1, Imen Soussi, Engineer1, Aleksandra Caban, MSc, PhD, MD2, Mondher Toumi, MSc, PhD, MD3.
1Clever-Access, Tunis, Tunisia, 2Clever-Access, Cracow, Poland, 3Aix-Marseille University, Marseille, France.
1Clever-Access, Tunis, Tunisia, 2Clever-Access, Cracow, Poland, 3Aix-Marseille University, Marseille, France.
OBJECTIVES: Real-world evidence (RWE) is increasingly used in submissions to regulators and health technology assessment (HTA) bodies. However, policy and guidance on RWE vary globally in scope, methodological approaches, and validity requirements, creating uncertainty for evidence-generation planning. This study aims to analyse the current policy and available guidance documents on the use of RWE in HTA and regulatory decision-making across Europe, Canada, and the United States.
METHODS: A targeted review was conducted on 05/01/2026 to identify publicly available RWE policy and guidance documents from Europe, Canada, the United States, and international organisations. Documents were categorized by context, issuing body and product scope and key content was extracted for comparative analysis.
RESULTS: Thirty documents were included (11 from HTA agencies, 13 from regulatory bodies, and 6 from other organizations) and categorised into HTA guidance (8), regulatory guidance (12), joint regulatory-HTA guidance (6), research document (1), and strategic documents (3). Seventeen documents addressed medicines, 12 addressed medical devices, and 13 addressed both. Core definitions of RWE were largely consistent, though definitions of RWE sources and types varied. Across jurisdictions, guidance uniformly positions RWE as complementary to randomized trials and emphasizes fit-for-purpose data, prespecified protocols, rigorous bias/confounding control, and sensitivity analyses. Regulatory and HTA policies converge on the importance of robust study design, with specific considerations for medical devices. Notable differences were observed in implementation and reporting standards, including data transparency, reproducibility, and disclosure of confounding adjustments. Divergences also arise between HTA and regulatory expectations: HTA guidance often stresses contextual relevance and cost-effectiveness, while regulatory guidance prioritizes binding safety and efficacy evidence.
CONCLUSIONS: While methodological principles for RWE are broadly aligned, operational criteria remain heterogeneous, and guidance on interpreting residual uncertainty is limited. Establishing standardized, decision-focused acceptance criteria could enhance predictability and support appropriate RWE integration in regulatory and HTA processes.
METHODS: A targeted review was conducted on 05/01/2026 to identify publicly available RWE policy and guidance documents from Europe, Canada, the United States, and international organisations. Documents were categorized by context, issuing body and product scope and key content was extracted for comparative analysis.
RESULTS: Thirty documents were included (11 from HTA agencies, 13 from regulatory bodies, and 6 from other organizations) and categorised into HTA guidance (8), regulatory guidance (12), joint regulatory-HTA guidance (6), research document (1), and strategic documents (3). Seventeen documents addressed medicines, 12 addressed medical devices, and 13 addressed both. Core definitions of RWE were largely consistent, though definitions of RWE sources and types varied. Across jurisdictions, guidance uniformly positions RWE as complementary to randomized trials and emphasizes fit-for-purpose data, prespecified protocols, rigorous bias/confounding control, and sensitivity analyses. Regulatory and HTA policies converge on the importance of robust study design, with specific considerations for medical devices. Notable differences were observed in implementation and reporting standards, including data transparency, reproducibility, and disclosure of confounding adjustments. Divergences also arise between HTA and regulatory expectations: HTA guidance often stresses contextual relevance and cost-effectiveness, while regulatory guidance prioritizes binding safety and efficacy evidence.
CONCLUSIONS: While methodological principles for RWE are broadly aligned, operational criteria remain heterogeneous, and guidance on interpreting residual uncertainty is limited. Establishing standardized, decision-focused acceptance criteria could enhance predictability and support appropriate RWE integration in regulatory and HTA processes.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PT41
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure
Disease
No Additional Disease & Conditions/Specialized Treatment Areas