OBJECTIVES: To analyze the agreements reached during P&R negotiation with AIFA by orphan drugs (ODs) designated by EMA, with P&R determination published in the Italian Official Journal (OJ) in the period January 2016-May 2021.

METHODS: ODs first P&R determinations published on OJ from January 2016 to May 2021 were collected and examined. The relevant data has been included in the database: ODs classification (A or H), negotiating conditions, presence of monitoring registers, financial and clinical/outcomes-based MEAs and Innovation Status recognition (IS).

RESULTS: From 2016 to May 2021, 73 P&R determinations were published, and 59 ODs were reimbursed (class A or H), 22 (37%) for oncological indications, 37 (63%) for non-oncological indications; no agreement was reached for 14 (19%) ODs, which were classified in C.

Regarding IS, 28 (48%) ODs obtained the status by CTS: 15 Full and 13 Conditional IS. Referring to therapeutic area, 13 (35%) non-oncological ODs and 14 (38%) oncological ODs obtained IS.

50 (85%) ODs obtained financial MEA, 2 (3%) ODs had a clinical MEA and 4 (7%) obtained both, financial and clinical MEAs. In addition, 40 (68%) presented a monitoring register. Concerning to therapeutic area, 32 (87%) non-oncological ODs had financial MEA, 2 (5%) clinical MEA and 1 (3%) both. On the other side, 18 (82%) oncological ODs had financial MEA and 3 (14%) had clinical and financial MEAs.

CONCLUSIONS: During the period included in the study, the majority of ODs achieved reimbursement, most of them with one or more MEAs. In particular, there was a predominance of financial MEAs compared to clinical/outcomes-based MEAs. The analysis proved that therapeutic area didn’t impact on MEAs or IS assignment, in fact, two groups show similar percentages.