OBJECTIVES: Scoping defines each HTA institute’s setting of the research question with its components Population, Intervention, Control, Outcomes (PICO). The scoping process will be key for the subsequent EU HTA. The future scoping process was simulated within our European network.

METHODS: Mock-up was done for Onasemnogene abeparvovec (Zolgensma^®), as ATMPs are within the first wave of EU HTA, ATMPs pose special methodological challenges within HTA, and this was a widely discussed product launch. Scoping consists of 2 phases, survey and consolidation. Survey was done considering actual assessment and reimbursement restrictions of the respective countries. Written feedback from Italy, Spain, France, and Germany was checked for consistency and follow-up questions asked for clarification. Consolidation was performed by TE and AB.

RESULTS: Assessment and reimbursement are not completely separate processes in all 4 countries. Germany was the only country for which the complete label was relevant for reimbursement. France excluded symptomatic patients with SMA type 3. Italy and Spain only included symptomatic patients with SMA type 1 and presymptomatic patients with up to 2 copies of the SMN2 gene. For the symptomatic patients Italy and Spain defined restrictions regarding weight and age of patients. In addition, Spain also set an age restriction for the presymptomatic population.

Nusinersen was considered a relevant comparator in all countries at some point of the evaluation, whereas risdiplam was only considered by France during reassessment which occurred later. Initially, Germany did not define a comparator.

Not all outcomes have had the same relevance in every jurisdiction, but there seemed to be reasonable consensus.

CONCLUSIONS: In summary, whereas intervention, control, and outcomes mostly converge, no two countries aligned on the reimbursed population. Due to the special situation final assessment was determined by the data and was to some extent independent of a predefined PICO scheme.