HTA BODIES REQUEST FEWER THAN 50% OF GUIDELINE-DEFINED PICOS: EVIDENCE FROM EIGHT EU MARKETS AND LESSONS FOR EU JCA
Author(s)
Paula Skowron, MPharm1, Izabela Moszkowicz, MPharm1, Ani Stefanova, MPharm2, Peter Wagner, BMS3, Anke van Engen, MSc4, Sian Tanner, BA, PhD4, Edel Falla, MSc5.
1IQVIA, Warsaw, Poland, 2IQVIA, Sofia, Bulgaria, 3IQVIA, Frankfurt, Germany, 4IQVIA, Amsterdam, Netherlands, 5IQVIA, London, United Kingdom.
1IQVIA, Warsaw, Poland, 2IQVIA, Sofia, Bulgaria, 3IQVIA, Frankfurt, Germany, 4IQVIA, Amsterdam, Netherlands, 5IQVIA, London, United Kingdom.
OBJECTIVES: The scope of Joint Clinical Assessments (JCA) is defined by the population, intervention, comparator, and outcomes (PICO) framework. This analysis quantifies divergence between guideline-defined PICOs and PICOs requested in health technology assessments (HTA) across eight EU markets, and identifies factors driving PICO inclusion, modification, and exclusion.
METHODS: The analysis covered countries with explicit definition of the PICO scope in national HTAs - Germany, France, the Netherlands, Sweden, Poland, the Czech Republic, Denmark, and Finland. Five recently approved oncology therapies with HTAs available in ≥5/8 countries were purposively selected to cover diverse tumour types, treatment lines, and orphan designation: zolbetuximab, amivantamab, zanubrutinib, glofitamab, and trastuzumab deruxtecan. We extracted all potential PICOs from national and ESMO guidelines falling under HTA-reviewed indications, and HTA bodies’ rationales for PICOs inclusion or exclusion.
RESULTS: On average, HTA bodies requested 2.7 PICOs per assessment (range: 1.0 for the Netherlands to 6.2 for Poland), versus 19.4 identified in guidelines. Overall, 57% of guideline-defined PICOs across all products and countries were not requested in HTA, 40% were modified, and 3% were included unchanged. Exclusion rates varied by country (22.8% for France to 73.1% for the Netherlands) and product (42.2% for zolbetuximab to 79.8% for amivantamab). The most common exclusion reasons were prioritisation of more relevant comparators, different mechanism of action (for zanubrutinib), and lack of reimbursement. Modifications involved broader populations than in guidelines and grouping comparators with ‘OR’ approach or under individualised treatment. Inclusion was mainly driven by alignment with clinical guidelines, clinical expert opinion, and reimbursement status.
CONCLUSIONS: HTA bodies systematically narrow the PICO scope relative to clinical guidelines across countries and products, suggesting that guideline alignment is necessary but insufficient to predict JCA PICOs. Health technology developers should verify the guidelines recommendations against reimbursement status, published HTA reports, real-world use and clinical experts opinions to prioritize relevant PICOs.
METHODS: The analysis covered countries with explicit definition of the PICO scope in national HTAs - Germany, France, the Netherlands, Sweden, Poland, the Czech Republic, Denmark, and Finland. Five recently approved oncology therapies with HTAs available in ≥5/8 countries were purposively selected to cover diverse tumour types, treatment lines, and orphan designation: zolbetuximab, amivantamab, zanubrutinib, glofitamab, and trastuzumab deruxtecan. We extracted all potential PICOs from national and ESMO guidelines falling under HTA-reviewed indications, and HTA bodies’ rationales for PICOs inclusion or exclusion.
RESULTS: On average, HTA bodies requested 2.7 PICOs per assessment (range: 1.0 for the Netherlands to 6.2 for Poland), versus 19.4 identified in guidelines. Overall, 57% of guideline-defined PICOs across all products and countries were not requested in HTA, 40% were modified, and 3% were included unchanged. Exclusion rates varied by country (22.8% for France to 73.1% for the Netherlands) and product (42.2% for zolbetuximab to 79.8% for amivantamab). The most common exclusion reasons were prioritisation of more relevant comparators, different mechanism of action (for zanubrutinib), and lack of reimbursement. Modifications involved broader populations than in guidelines and grouping comparators with ‘OR’ approach or under individualised treatment. Inclusion was mainly driven by alignment with clinical guidelines, clinical expert opinion, and reimbursement status.
CONCLUSIONS: HTA bodies systematically narrow the PICO scope relative to clinical guidelines across countries and products, suggesting that guideline alignment is necessary but insufficient to predict JCA PICOs. Health technology developers should verify the guidelines recommendations against reimbursement status, published HTA reports, real-world use and clinical experts opinions to prioritize relevant PICOs.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA350
Topic
Health Policy & Regulatory, Health Service Delivery & Process of Care, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure
Disease
Oncology