Presentation (CTI)

OBJECTIVES: In the context of Health Technology Assessment (HTA) frameworks, Patient-Reported Outcomes (PROs) are increasingly recognized as key evidence for assessing treatment benefits from the patient perspective. This study aims to analyze the integration of PROs in medicines reimbursed in Italy between 2020 and 2025, with a specific focus on innovativeness recognition.
METHODS: A descriptive analysis was conducted on medicinal products reimbursed in Italy between 2020 and 2025. The therapeutic area, the presence of specific regulatory designations and the role of PROs within the clinical development program were assessed. In addition, a subgroup analysis was performed on medicines that received an innovativeness designation.
RESULTS: Between 2020 and 2025, a total of 180 medicines excluding generics and biosimilars were reimbursed in Italy. Of these, 21 (11.7%) included at least one PRO measure in the clinical documentation of the Marketing Authorization Application. PROs were used as primary endpoints in 4 cases (19.0%) and as secondary endpoints in 17 cases (81.0%). Overall, 14 medicines (7.8%) were granted innovativeness status. However, none of these products included PRO-based evidence as part of the documentation supporting innovativeness recognition. PRO adoption was more frequent in dermatology, immunology, gastroenterology, and rare diseases. The most common PRO instruments included generic health-related quality-of-life measures alongside disease-specific tools.
CONCLUSIONS: Although the use of PROs in medicines reimbursed in Italy has increased over time, the absence of PRO-based evidence among medicines granted AIFA innovativeness status suggesting that PROs currently have a limited role in this assessment process. In this context, the new AIFA criteria for price and reimbursement dossiers on added therapeutic value, together with the updated criteria for innovativeness assessment, introduced in 2025, may strengthen the role of PROs in the evaluation process.