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Background and Importance Since 2012, an annual registry of orphan drug (OD) use in patients with rare diseases (RD) has been maintained. ODs are essential for treating RDs, but their utilisation, and economic impact represent a challenge for healthcare systems.Aim and Objectives The objective was to assess the prevalence and evolution of OD use over a 14-year period and its economic impact on the hospital pharmacy budget (HPB).Material and Methods This was a retrospective, observational, longitudinal study from January 2011 to December 2024, of adult and paediatric patients with RD treated with ODs, both oncologic (excluding CAR-T therapies) and non-oncologic. Data were extracted from the official inpatient and outpatient pharmacy software (Farmatools), using OD classifications validated by ORPHANET (last update: April 2024). Drug prices were based on those published by the national health authorities. Variables analysed included the number and type of ODs (ATC classification), the number of treated patients, drug utilisation, and OD-related costs (percentage of HPB).Results 141 patients received treatment with 36 different ODs (97% adults, 3% paediatric). The highest number of patients was recorded in 2015 (n=41). OD use peaked in 2015 and 2020 (15 drugs), with the lowest in 2023–2024 (five drugs). Overall, 53% of ODs were oncologic and 47% non-oncologic. The most commonly used ODs were daratumumab (14.8%), nintedanib (11.3%), ibrutinib (10%), azacitidine, and bosentan (7.8%). OD distribution by ATC group was dominated by L (n=19), followed by C and A (n=4 each).The average economic impact of ODs on the hospital pharmacy budget was 7.93%, ranging from 3.42% (2011) to a peak of 12.90% (2022). Group L represented 64.7% of total OD expenditure, with daratumumab alone accounting for 21.1% of total spending. Paediatric patients represented 11.5% of OD expenditure despite being only 3% of the sample.Conclusion and Relevance Oncologic ODs represented 53% of treatments and accounted for 64.7% of OD-related spending. Daratumumab was the most used and costliest OD, while bosentan was the most frequent non-oncologic OD. The average OD budget impact over 14 years was 7.8%. This long-term project reflects a strong commitment to improving care and visibility for rare diseases, while highlighting the financial challenges of ODs in small healthcare systems.Conflict of Interest No conflict of interest