Uncertainty About How to Translate JCA Into National HTA Submissions (EU-4 and Poland): A Systematic Comparison From an Industry Perspective
Author(s)
Ulrike Kuchenbecker, PhD1, Malek Dimassi, MSc2, Anna Danuta Kuciara, MPH3, Clement Francois, PhD4, Simon Pannett, PhD5, Mateusz Nikodem, PhD3.
1Principal HTA&Market Access, Putnam, Mannheim, Germany, 2Putnam, Soukra, Tunisia, 3Putnam, Krakow, Poland, 4Putnam, Paris, France, 5Putnam, London, United Kingdom.
1Principal HTA&Market Access, Putnam, Mannheim, Germany, 2Putnam, Soukra, Tunisia, 3Putnam, Krakow, Poland, 4Putnam, Paris, France, 5Putnam, London, United Kingdom.
OBJECTIVES: Uncertainty exists about translating Joint Clinical Assessment (JCA) reports into national submissions. This analysis examines core JCA evidence requirements and how they differ from national health technology assessment (HTA).
METHODS: A targeted review of EU and national HTA guidance, supported by searches of HTA websites, stakeholder webinars, press releases, PubMed, and grey literature, was conducted to compare JCA and national methodological requirements across key domains. Where formal guidance was limited, expert judgement was employed.
RESULTS: The JCA assesses clinical evidence for the broader EU population, whereas national HTAs highly prioritise local relevance. In the EU dossier, manufacturers must justify the representativeness of populations, comparators, and outcomes, but country-specific data are not required. For the systematic information retrieval, the JCA aligns with international standards, incorporates the Clinical Trials Information System, and does not require Embase searches. While overall survival is an established HTA outcome, the JCA focuses on patient-centred outcomes, whereas Germany emphasises patient-relevant benefits. For the JCA, validated surrogates are accepted to replace a patient-centred outcome only if necessary. The JCA applies the concept of Minimal Important Difference for the interpretation of patient-reported outcomes, whereas Germany uses a 15% threshold to indicate a noticeable and clinically relevant difference. JCA supports fixed and random effects models for the meta-analysis, and network meta-analysis can be applied even when head-to-head studies for the comparison of interest are available. Application of population-adjusted methods is seen with caution due to concerns about multiplicity.
CONCLUSIONS: Assuming alignment on the HTA research question, substantial divergences between JCA and national submissions in evidence requirements are unlikely; With effort spent on the JCA, this should meet 95% of the clinical evidence requirements of all countries. However, country-specific considerations will remain. HTA bodies will continue to expect context-sensitive submissions. Collaborative and early engagement between agencies and manufacturers is critical.
METHODS: A targeted review of EU and national HTA guidance, supported by searches of HTA websites, stakeholder webinars, press releases, PubMed, and grey literature, was conducted to compare JCA and national methodological requirements across key domains. Where formal guidance was limited, expert judgement was employed.
RESULTS: The JCA assesses clinical evidence for the broader EU population, whereas national HTAs highly prioritise local relevance. In the EU dossier, manufacturers must justify the representativeness of populations, comparators, and outcomes, but country-specific data are not required. For the systematic information retrieval, the JCA aligns with international standards, incorporates the Clinical Trials Information System, and does not require Embase searches. While overall survival is an established HTA outcome, the JCA focuses on patient-centred outcomes, whereas Germany emphasises patient-relevant benefits. For the JCA, validated surrogates are accepted to replace a patient-centred outcome only if necessary. The JCA applies the concept of Minimal Important Difference for the interpretation of patient-reported outcomes, whereas Germany uses a 15% threshold to indicate a noticeable and clinically relevant difference. JCA supports fixed and random effects models for the meta-analysis, and network meta-analysis can be applied even when head-to-head studies for the comparison of interest are available. Application of population-adjusted methods is seen with caution due to concerns about multiplicity.
CONCLUSIONS: Assuming alignment on the HTA research question, substantial divergences between JCA and national submissions in evidence requirements are unlikely; With effort spent on the JCA, this should meet 95% of the clinical evidence requirements of all countries. However, country-specific considerations will remain. HTA bodies will continue to expect context-sensitive submissions. Collaborative and early engagement between agencies and manufacturers is critical.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA345
Topic
Health Policy & Regulatory, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas