Patient-Centered PICO Scoping in EU HTA: A Pilot Study for Structured Consensus Building Using a Delphi Panel
Author(s)
Emanuele Arca`, MSc1, Adele Barlassina, MSc1, Valentina Strammiello, MA2.
1OPEN Health, Rotterdam, Netherlands, 2European Patients Forum, Brussels, Belgium.
1OPEN Health, Rotterdam, Netherlands, 2European Patients Forum, Brussels, Belgium.
OBJECTIVES: The EU Health Technology Assessment Regulation (HTAR) mandates inclusive and harmonized Joint Clinical Assessments (JCAs) across Member States, with PICO (Population, Intervention, Comparator, Outcome) frameworks at their core. A notable innovation is the mandate to incorporate patients’ perspectives in PICO scoping. This study aimed to pilot a structured consensus-building method to integrate patient perspectives into PICO scoping using a modified Delphi panel.
METHODS: Co-developed with the European Patients’ Forum (EPF), a modified Delphi panel was conducted in 2025. Twelve EU patient representatives, completed iterative surveys to reach consensus (≥80% agreement) on PICO elements. Quantitative agreement thresholds and qualitative feedback were used to refine statements across rounds. The case study involved PICO scoping simulation for the use of CAR-T therapy in paediatric spinal muscular atrophy.
RESULTS: Consensus was reached on: (1) five Population items, including a wider population of interest and the importance of considering patient and caregiver preferences; (2) eighteen Intervention items, highlighting the need for patient-cantered care, and therapy burden management; (3) six Comparator items, supporting the use of no treatment and best supportive care as comparators, and emphasizing informed decision-making; (4) eleven Outcome items, prioritizing quality of life outcomes; and (5) seventeen Study Design items, endorsing the use of diverse evidence types, including real-world and patient-reported outcomes. Key areas of non-consensus included comparator selection, trial inclusion criteria, and patient preferences regarding treatment accessibility versus stronger evidence-based treatments.
CONCLUSIONS: Integrating patients views into PICO scoping is a key challenge of the HTAR. The Delphi approach supports the transparent, inclusive and rigorous patient engagement in the process. This pilot demonstrates the feasibility and value of using Delphi methodology to integrate patient perspectives into PICO scoping for EU-HTA. Further research is needed to formalize this process and explore its integration into regulatory frameworks and evidence packages.
METHODS: Co-developed with the European Patients’ Forum (EPF), a modified Delphi panel was conducted in 2025. Twelve EU patient representatives, completed iterative surveys to reach consensus (≥80% agreement) on PICO elements. Quantitative agreement thresholds and qualitative feedback were used to refine statements across rounds. The case study involved PICO scoping simulation for the use of CAR-T therapy in paediatric spinal muscular atrophy.
RESULTS: Consensus was reached on: (1) five Population items, including a wider population of interest and the importance of considering patient and caregiver preferences; (2) eighteen Intervention items, highlighting the need for patient-cantered care, and therapy burden management; (3) six Comparator items, supporting the use of no treatment and best supportive care as comparators, and emphasizing informed decision-making; (4) eleven Outcome items, prioritizing quality of life outcomes; and (5) seventeen Study Design items, endorsing the use of diverse evidence types, including real-world and patient-reported outcomes. Key areas of non-consensus included comparator selection, trial inclusion criteria, and patient preferences regarding treatment accessibility versus stronger evidence-based treatments.
CONCLUSIONS: Integrating patients views into PICO scoping is a key challenge of the HTAR. The Delphi approach supports the transparent, inclusive and rigorous patient engagement in the process. This pilot demonstrates the feasibility and value of using Delphi methodology to integrate patient perspectives into PICO scoping for EU-HTA. Further research is needed to formalize this process and explore its integration into regulatory frameworks and evidence packages.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA268
Topic
Health Technology Assessment, Methodological & Statistical Research, Study Approaches
Topic Subcategory
Value Frameworks & Dossier Format
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), No Additional Disease & Conditions/Specialized Treatment Areas