# Enhancing Patient-Centered Health Technology Assessment: A Modified Delphi Panel for PICOS Scoping in Spinal Muscular Atrophy

## TL;DR

This study shows how involving patients in health assessments can improve decision-making for rare diseases like spinal muscular atrophy.

## Contribution

A modified Delphi method is introduced to harmonize patient and national perspectives in health technology assessments.

## Key findings

- A modified Delphi process achieved consensus on PICOS elements for CAR-T therapy in spinal muscular atrophy.
- Patient input emphasized inclusive eligibility and shared decision-making in health assessments.
- The method revealed patient priorities on outcomes and treatment trade-offs, enhancing HTA strategies.

## Abstract

What is the main finding?
This paper illustrates how systematic PICOS scoping can be operationalized within JCAs through Delphi consensus methods for mapping and reconciling multiple national and patient perspectives into a common framework.It highlights concrete examples of harmonization and clarifies the methodological tensions that arise when balancing rigor with flexibility.

This paper illustrates how systematic PICOS scoping can be operationalized within JCAs through Delphi consensus methods for mapping and reconciling multiple national and patient perspectives into a common framework.

It highlights concrete examples of harmonization and clarifies the methodological tensions that arise when balancing rigor with flexibility.

What are the implications of the main findings?
The findings underscore that early, structured stakeholder engagement—particularly involving patients—is essential for JCAs to be inclusive, scientifically robust, and relevant for national reimbursement decisions.Clear PICOS scoping could improve transparency while also enhancing the credibility and uptake of JCA outcomes in healthcare policy.

The findings underscore that early, structured stakeholder engagement—particularly involving patients—is essential for JCAs to be inclusive, scientifically robust, and relevant for national reimbursement decisions.

Clear PICOS scoping could improve transparency while also enhancing the credibility and uptake of JCA outcomes in healthcare policy.

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.