OBJECTIVES: Harmonizing PICO (Patient, Intervention, Comparator, Outcomes) requirements across the EU, as established by new EU HTA Regulation, presents challenges due to heterogenous healthcare systems and diverse stakeholder perspectives. A notable innovation is the mandate to incorporate patients’ perspectives in PICO scoping. This research aims to achieve consensus among patient experts in spinal muscular atrophy (SMA) on PICO scoping, and to evaluate the relevance, viability, and appropriateness of the modified Delphi methodology for this purpose.

METHODS: A 3-step modified Delphi technique was employed, comprising a single 1:1 remote interview round and two online survey rounds. Input from a steering committee comprising a policy, clinical and patient expert, guided the development of the consensus survey, and the selection of panellists. Quantitative data on the level of agreement of panellists were collected using Likert-type ranking scales.

RESULTS: Twelve SMA patient experts, with a foundational understanding of HTA, participated across several EU countries. The statements covered all elements of the PICOS framework relevant to patients. The survey yielded consensus on several PICO elements, including age groups, sex, and relevant comorbidities. A wide range of outcomes affecting the quality of life of the patient, side-effects and treatment outcomes were captured. Patients generated consensus on population and sub populations characteristics, outcomes, and patients’ acceptability around study designs and uncertainty.

CONCLUSIONS: Through a rigorous consensus-building process, this study provides an example of the valuable contribution of patient experts in informing PICO scoping for SMA therapies across the EU. This aligns with the EU HTA Regulation, which establishes requirements for developing PICOs that incorporate patients’ perspectives and addresses differences across EU member states. This study highlights the need for a structured approach and additional resources to facilitate early engagement with patients and patient organizations in PICO development to facilitate recruitment across EU countries, within very tight timelines.