WHAT DOES GOOD LOOK LIKE? POLICY RECOMMENDATIONS FOR ADVANCING THE ACCEPTANCE OF RWE IN REGULATORY AND HTA DECISION-MAKING
Author(s)
Omnia Bilal1, Shane Collins, MSc2, Bethany Shinkins, PhD2, Rita Maria Peeters, MBA, PhD3, Heather Colvin, MPP4.
1NICE, United Kingdom, 2NICE, Manchester, United Kingdom, 3Johnson & Johnson Medical, Machelen, Belgium, 4Johnson and Johnson, Washington, DC, USA.
1NICE, United Kingdom, 2NICE, Manchester, United Kingdom, 3Johnson & Johnson Medical, Machelen, Belgium, 4Johnson and Johnson, Washington, DC, USA.
OBJECTIVES: Real-world evidence (RWE) derived from heterogeneous health data sources is increasingly used in regulatory and health technology assessment (HTA) decision-making; however, policy frameworks guiding its use remain inconsistent. The IHI-IDERHA consortium aimed to systematically review existing policies across international jurisdictions, capture multi-stakeholder perspectives through expert workshops and public consultation, and develop actionable recommendations to strengthen the trust in and acceptance of RWE in regulatory and HTA contexts. This work provides a structured, multi-stakeholder approach to addressing key barriers to RWE acceptance.
METHODS: A mixed-method approach was employed, including (1) a systematic review of regulatory and HTA policies on RWE acceptability across international (primarily European) jurisdictions, (2) expert workshops with regulators, HTA representatives, and industry stakeholders, (3) targeted stakeholder engagement and (4) public consultation. Findings were synthesised to identify key areas of alignment and key gaps, which informed the development of policy recommendations.
RESULTS: Substantial variation was identified in how regulatory and HTA bodies define and assess RWE, with limited alignment on data quality, study design, and transparency requirements. Stakeholders highlighted uncertainty regarding what constitutes “fit-for-purpose” evidence and persistent concerns about reproducibility, both of which constrain broader use of RWE. To address these concerns, a generalised RWE study process map was developed to support the design, conduct, and evaluation of RWE studies across decision contexts. Policy recommendations were developed to address governance, methodological standards, and alignment between regulators and HTA bodies to improve consistency, transparency, and confidence in RWE.
CONCLUSIONS: Limited alignment in policy frameworks across regulatory and HTA bodies remain a key barrier to the wider use of RWE. The IHI-IDERHA recommendations provide practical, stakeholder-informed guidance to strengthen trust and acceptance of RWE from real-world health data, supporting its more consistent use in regulatory and HTA decision-making.
METHODS: A mixed-method approach was employed, including (1) a systematic review of regulatory and HTA policies on RWE acceptability across international (primarily European) jurisdictions, (2) expert workshops with regulators, HTA representatives, and industry stakeholders, (3) targeted stakeholder engagement and (4) public consultation. Findings were synthesised to identify key areas of alignment and key gaps, which informed the development of policy recommendations.
RESULTS: Substantial variation was identified in how regulatory and HTA bodies define and assess RWE, with limited alignment on data quality, study design, and transparency requirements. Stakeholders highlighted uncertainty regarding what constitutes “fit-for-purpose” evidence and persistent concerns about reproducibility, both of which constrain broader use of RWE. To address these concerns, a generalised RWE study process map was developed to support the design, conduct, and evaluation of RWE studies across decision contexts. Policy recommendations were developed to address governance, methodological standards, and alignment between regulators and HTA bodies to improve consistency, transparency, and confidence in RWE.
CONCLUSIONS: Limited alignment in policy frameworks across regulatory and HTA bodies remain a key barrier to the wider use of RWE. The IHI-IDERHA recommendations provide practical, stakeholder-informed guidance to strengthen trust and acceptance of RWE from real-world health data, supporting its more consistent use in regulatory and HTA decision-making.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD98
Topic
Health Policy & Regulatory, Health Technology Assessment, Real World Data & Information Systems
Topic Subcategory
Data Protection, Integrity, & Quality Assurance
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology