Background and Importance Some oncological treatments are used in special conditions due to the lack of therapeutic alternatives. The use of drugs in exceptional circumstances or special situations (compassionate use (CU), off-label use (OLU), EMA approved drugs without a refund price in the country, and drugs not included in the hospital’s pharmacotherapeutic guide (non-HPG)) is frequent. These treatments must be approved by an internal committee at the hospital level according to Spanish legislation.

Aim and Objectives To analyse the requests and effectiveness of drugs in special situations in oncology patients at a third-level university hospital.

Material and Methods An observational, single-centre, retrospective study was performed to analyse the prescription of special situation drugs in oncology patients between January 2021 and December 2022. Data were collected regarding the nature of the special situation, cancer type, treatment setting (curative or palliative), ESMO clinical benefit, treatment acceptance, clinical response, discontinuation reasons, number of administered cycles, and associated cost, which was calculated based on the treatment cycles administered until the end of the study.

Results 1045 requests were submitted to the hospital committee: 204 (19.52%) belonged to the oncology field (solid tumours). Among these, the types of special drug petitions were: CU (n=46, 22.55%), OLU (n=102, 50%), without a refund price in the country (n=44, 21.57%), and non-HPG (n=12, 5.88%).

Curative setting (n=30): ESMO benefit categories A (n=25, 83.33%), B (n=0, 0%), C (n=4, 13.33%) and not applicable (n=1, 3.33%). Metastatic disease (n=174) ESMO benefit scale 1 (n=32, 18.39%), 2 (n=12, 6.90%), 3 (n=58, 33.33%), 4 (n=55, 31.61%), 5 (n=0, 0%), and not applicable (n=17, 9.77%).

Patients who did not start the treatment (8.33%) were those with no further therapeutic alternatives who progressed and did not have time to initiate this therapeutic approach. The average ± SD of administered cycles was 5.89 (±5.74), amounting to a total cost of €2.597.220.

Conclusion and Relevance There is a high percentage of medication requests in special situations in the oncology field, most of them in the palliative setting (85.29%), with significant economic impact. It is crucial to regulate special-use medications to ensure equal treatment opportunities among cancer patients of different country hospitals.

Conflict of Interest No conflict of interest.