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Enhancing Patient-Centered Health Technology Assessment: A Modified Delphi Panel for PICOS Scoping in Spinal Muscular Atrophy
Objectives
This study explores the feasibility and value of integrating structured patient input into the PICOS (Population, Intervention, Comparator, Outcome, Study design) scoping process for Joint Clinical Assessments under the EU Health Technology Assessment Regulation.
Methods
A modified Delphi panel, led by a steering committee composed of two clinicians, one patient expert, and one policy expert, engaged 12 individuals representing patient organizations across 12 European Member States to reach consensus on PICOS elements for CAR-T therapy in pediatric spinal muscular atrophy.
Results
The Delphi process effectively facilitated PICOS consolidation and consensus among the 12 patient experts representing diverse EU contexts. Through 3 iterative rounds integrating quantitative rankings and qualitative feedback, the panel achieved strong agreement on key outcomes, intervention delivery, and study design elements, with population eligibility and comparator selection showing heterogeneity. Patient engagement was central: participants emphasized inclusive eligibility criteria, shared decision-making, and the inclusion of caregiver perspectives. The integration of qualitative insights allowed nuanced interpretation of dissent, distinguishing between genuine disagreement and framing effects, thereby enhancing transparency and scientific validity. Importantly, the process revealed patient priorities for outcomes, treatment burden, and evidence trade-offs, informing both PICOS refinement and future health technology assessment (HTA) strategies. This structured, participatory approach demonstrates the feasibility and value of incorporating patient voices systematically into early-stage EU HTA, fostering robust, credible, and context-sensitive consensus on complex rare-disease interventions.
Conclusions
The study demonstrates the potential of consensus-building methodologies to enhance transparency, reduce heterogeneity, and support patient-centered evidence generation and decision-making in HTA.
Authors
E Arcà, A Barlassina, A Eze, V Strammiello
Journal
Journal of Market Access & Health Policy
Therapeutic Area
Neurology
Center of Excellence
Patient-Centered outcomes, Market Access Strategy
Year
2026
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