The Role of Real-World Evidence in Health Technology Assessment Decision Making
Author(s)
Tizian J. Mueller, MSc1, Sophie Spilles, Msc2, Nina Stroehmer, MSc2.
1IQVIA, Porto Salvo, Portugal, 2NOVA SBE, Lisbon, Portugal.
1IQVIA, Porto Salvo, Portugal, 2NOVA SBE, Lisbon, Portugal.
OBJECTIVES: This study evaluates how Real-World Evidence (RWE) is incorporated into Health Technology Assessment (HTA) processes and its influence on reimbursement decisions. It examines factors driving or hindering RWE adoption, such as regulatory frameworks, data quality, and stakeholder trust and compares RWE with Randomized Controlled Trials (RCTs) to assess their complementary value in healthcare decision-making. The study also explores global initiatives aimed at harmonizing RWE methodologies for wider applicability across health systems.
METHODS: A systematic literature review was conducted following PRISMA and Cochrane guidelines. A PICOS-based search strategy was used to retrieve peer-reviewed articles, HTA agency reports, and case studies published within the past five years from nine high-income countries. After duplicate removal and multi-stage screening, 40 sources were selected from a database of 4,109 records. Data were extracted using a standardized template and synthesized thematically. A dual-review approach was applied to ensure methodological rigor and minimize bias.
RESULTS: RWE adoption varies across HTA bodies due to differences in regulatory frameworks, stakeholder trust, and data availability. Agencies such as NICE (UK) and HAS (France) have implemented structured approaches to incorporate RWE in economic models and post-launch evaluations. In contrast, institutions like Germany’s IQWiG maintain a predominantly RCT-centric stance. Common barriers include data heterogeneity, inconsistent methodologies, and skepticism about RWE validity. Nonetheless, advances in centralized data infrastructures and analytic methods have enhanced RWE credibility. Initiatives by EMA and EUnetHTA aim to standardize practices but face challenges due to national regulatory differences.
CONCLUSIONS: RWE strengthens decision-making by complementing RCTs, providing insights into real-world effectiveness, safety, and cost-efficiency. Broader RWE integration into HTA requires improved data governance, harmonized methodologies, and greater collaboration among HTA bodies, payers, and industry stakeholders. Alignment on international standards is key to maximizing RWE’s impact on evidence-based reimbursement and healthcare equity.
METHODS: A systematic literature review was conducted following PRISMA and Cochrane guidelines. A PICOS-based search strategy was used to retrieve peer-reviewed articles, HTA agency reports, and case studies published within the past five years from nine high-income countries. After duplicate removal and multi-stage screening, 40 sources were selected from a database of 4,109 records. Data were extracted using a standardized template and synthesized thematically. A dual-review approach was applied to ensure methodological rigor and minimize bias.
RESULTS: RWE adoption varies across HTA bodies due to differences in regulatory frameworks, stakeholder trust, and data availability. Agencies such as NICE (UK) and HAS (France) have implemented structured approaches to incorporate RWE in economic models and post-launch evaluations. In contrast, institutions like Germany’s IQWiG maintain a predominantly RCT-centric stance. Common barriers include data heterogeneity, inconsistent methodologies, and skepticism about RWE validity. Nonetheless, advances in centralized data infrastructures and analytic methods have enhanced RWE credibility. Initiatives by EMA and EUnetHTA aim to standardize practices but face challenges due to national regulatory differences.
CONCLUSIONS: RWE strengthens decision-making by complementing RCTs, providing insights into real-world effectiveness, safety, and cost-efficiency. Broader RWE integration into HTA requires improved data governance, harmonized methodologies, and greater collaboration among HTA bodies, payers, and industry stakeholders. Alignment on international standards is key to maximizing RWE’s impact on evidence-based reimbursement and healthcare equity.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PT20
Topic
Health Policy & Regulatory, Health Technology Assessment, Organizational Practices
Topic Subcategory
Systems & Structure
Disease
No Additional Disease & Conditions/Specialized Treatment Areas