Harmonization or Hindrance? A Strategic Analysis of Divergent National Requirements Under the EU Joint Clinical Assessment
Author(s)
Preety Rajora, MPH1, Alosh Greeny, MPH1, Jatin Gupta, MBA, MPH2, Mohd Kashif Siddiqui, MBA, MPH, PharmD2.
1EBM Health Consultants, New Delhi, India, 2EBM Health Consultants, Cleckheaton, West Yorkshire, United Kingdom.
1EBM Health Consultants, New Delhi, India, 2EBM Health Consultants, Cleckheaton, West Yorkshire, United Kingdom.
OBJECTIVES: A fundamental and critical component of Joint Clinical Assessment (JCA) is the development of a harmonised PICO framework (Population, Intervention, Comparator, Outcome) reflecting the diverse needs of its Member States (MS). Whether harmonised PICO truly serve the diverse needs of all stakeholders remains a pressing strategic question. As JCA evaluation has just begun, understanding the real-world implications of harmonised PICO is crucial for pharmaceutical manufacturers, academic stakeholders, and consulting partners involved in evidence planning and submission strategy.
METHODS: We performed a thorough analysis of the PICO scoping guidance under the health technology assessment regulation (HTAR) enforcement of regulation (EU) 2021/2282, and the published critiques from HTA bodies and industry experts. The key factors examined include divergence across MS, evidence planning alignment, and industry's adaptive strategies.
RESULTS: Harmonised PICO reduces redundancy in HTA submissions but introduces critical strategic conflicts. Key challenge is misalignment with National HTA criteria especially of pivotal markets like France (emphasis on unmet need/treatment progress), and Germany (concentration on standard-of-care comparators). This divergence forces Health Technology Developers (HTDs) to model multi-scenario comparator strategies and justify choices across jurisdictions leading to resource inefficiencies. JCA scoping starts after the pivotal trial design is locked, which reduces the opportunity to align evidence generation with harmonised requirements. As a result, HTDs require urgent PICO strategy development such as mapping hypothetical comparators, endpoint validation, and inclusion of non-randomised/real word evidence.
CONCLUSIONS: Certain variations in the MS’s PICO looks minor but are fundamental, often irreconcilable methodological conflict. Thus, policymakers should avoid a one-size-fits-all approach that risks undermining both scientific relevance and timely market access. Whereas HTDs must adopt early adaptive evidence strategies that anticipate both convergence and divergence, and most importantly focus on market prioritisation. A market-prioritised, scenario-based, and evidence-diversified approach will be crucial to for successful EU-wide product access and reimbursement success.
METHODS: We performed a thorough analysis of the PICO scoping guidance under the health technology assessment regulation (HTAR) enforcement of regulation (EU) 2021/2282, and the published critiques from HTA bodies and industry experts. The key factors examined include divergence across MS, evidence planning alignment, and industry's adaptive strategies.
RESULTS: Harmonised PICO reduces redundancy in HTA submissions but introduces critical strategic conflicts. Key challenge is misalignment with National HTA criteria especially of pivotal markets like France (emphasis on unmet need/treatment progress), and Germany (concentration on standard-of-care comparators). This divergence forces Health Technology Developers (HTDs) to model multi-scenario comparator strategies and justify choices across jurisdictions leading to resource inefficiencies. JCA scoping starts after the pivotal trial design is locked, which reduces the opportunity to align evidence generation with harmonised requirements. As a result, HTDs require urgent PICO strategy development such as mapping hypothetical comparators, endpoint validation, and inclusion of non-randomised/real word evidence.
CONCLUSIONS: Certain variations in the MS’s PICO looks minor but are fundamental, often irreconcilable methodological conflict. Thus, policymakers should avoid a one-size-fits-all approach that risks undermining both scientific relevance and timely market access. Whereas HTDs must adopt early adaptive evidence strategies that anticipate both convergence and divergence, and most importantly focus on market prioritisation. A market-prioritised, scenario-based, and evidence-diversified approach will be crucial to for successful EU-wide product access and reimbursement success.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA168
Topic
Health Policy & Regulatory, Health Technology Assessment, Organizational Practices
Topic Subcategory
Systems & Structure
Disease
No Additional Disease & Conditions/Specialized Treatment Areas