BARRIERS AND POTENTIAL SOLUTIONS TO THE USE OF REAL-WORLD DATA AND EVIDENCE IN DECISION-MAKING FOR ADVANCED THERAPY MEDICINAL PRODUCTS: A MULTI-STAKEHOLDER QUALITATIVE STUDY
Author(s)
Charlotte Van Isterdael, Master1, Vicky Steyfkens, Master1, Lore Croughs, Bachelor1, Josefien Dehouck, Bachelor1, Ngoc Ho Nguyen, Bachelor1, Eline Vanhulsel, Bachelor1, Koen Debackere, PhD2, Peter Vandenberghe, PhD3, Johan Van Eldere, PhD4, Isabelle Huys, PharmD, PhD1.
1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium, 2Department of Oncology, KU Leuven, Leuven, Belgium, 3Department of Human Genetics, KU Leuven, Leuven, Belgium, 4European University Hospital Alliance (EUHA), Brussels, Belgium.
1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium, 2Department of Oncology, KU Leuven, Leuven, Belgium, 3Department of Human Genetics, KU Leuven, Leuven, Belgium, 4European University Hospital Alliance (EUHA), Brussels, Belgium.
OBJECTIVES: Advanced therapy medicinal products (ATMPs) offer promising treatment opportunities but are often associated with evidence uncertainties at the time of regulatory and reimbursement decision-making (DM), partly due to small trial populations and limited long-term follow-up data. While real-world data (RWD) and real-world evidence (RWE) may help address evidence gaps, their integration into DM remains limited and complex for involved stakeholders. This study aims to identify key barriers and potential solutions to improve RWD/RWE integration in ATMP DM.
METHODS: Semi-structured interviews were conducted across European stakeholder groups, including regulators, ATMP developers, HTA assessors/payers and patient representatives. Interviews were transcribed verbatim and analyzed thematically.
RESULTS: The interviews (N = 36) revealed that RWD/RWE were considered increasingly important throughout the ATMP lifecycle for addressing uncertainties in DM, although mainly in a supportive role. Key barriers covered four interrelated domains. First, available guidance was considered insufficiently actionable. Second, stakeholders consistently preferred disease-specific over product-specific registries, as these may support treatment comparisons, capture care pathways and enable long-term sustainability. However, their complex organization was perceived as challenging. Third, regarding data governance and access, stakeholders reported inconsistent GDPR interpretations across countries and institutions, limited early alignment on evidence expectations, restricted data access and poor interoperability across data sources. Finally, data quality challenges reported by stakeholders comprised missing data and limited representativeness. Proposed solutions centered on a coordinated data lifecycle approach, including early multi-stakeholder dialogue to define decision-relevant evidence needs upfront, harmonized regulatory and data standards, and sustainable registry infrastructures. Privacy-preserving data access and clear consent procedures were considered important to facilitate access and patient trust.
CONCLUSIONS: While RWD/RWE is widely recognized as essential for reducing uncertainty in ATMP DM, its effective use is constrained by challenges across the data lifecycle. Coordinated multi-stakeholder efforts are needed to ensure fit for purpose RWD/RWE.
METHODS: Semi-structured interviews were conducted across European stakeholder groups, including regulators, ATMP developers, HTA assessors/payers and patient representatives. Interviews were transcribed verbatim and analyzed thematically.
RESULTS: The interviews (N = 36) revealed that RWD/RWE were considered increasingly important throughout the ATMP lifecycle for addressing uncertainties in DM, although mainly in a supportive role. Key barriers covered four interrelated domains. First, available guidance was considered insufficiently actionable. Second, stakeholders consistently preferred disease-specific over product-specific registries, as these may support treatment comparisons, capture care pathways and enable long-term sustainability. However, their complex organization was perceived as challenging. Third, regarding data governance and access, stakeholders reported inconsistent GDPR interpretations across countries and institutions, limited early alignment on evidence expectations, restricted data access and poor interoperability across data sources. Finally, data quality challenges reported by stakeholders comprised missing data and limited representativeness. Proposed solutions centered on a coordinated data lifecycle approach, including early multi-stakeholder dialogue to define decision-relevant evidence needs upfront, harmonized regulatory and data standards, and sustainable registry infrastructures. Privacy-preserving data access and clear consent procedures were considered important to facilitate access and patient trust.
CONCLUSIONS: While RWD/RWE is widely recognized as essential for reducing uncertainty in ATMP DM, its effective use is constrained by challenges across the data lifecycle. Coordinated multi-stakeholder efforts are needed to ensure fit for purpose RWD/RWE.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD177
Topic
Health Policy & Regulatory, Health Technology Assessment, Real World Data & Information Systems
Topic Subcategory
Data Protection, Integrity, & Quality Assurance, Health & Insurance Records Systems
Disease
Personalized & Precision Medicine, Rare & Orphan Diseases