Background and Importance Compassionate use of drugs (CU) allows patients with serious diseases and no further treatment options to access treatments not yet approved. Specialist referral centres provide a reference point for access to these medicines, with significant benefits both for patient health and for avoided costs to the healthcare system.¹

Aim and Objectives The aim of this study is to describe the impact, in terms of clinical outcomes and saved costs, of CU at a University Hospital.

Material and Methods An 18-month retrospective analysis of approved CU at the Azienda Ospedale-Università Padova (AOUP) was conducted. A monitoring activity was implemented by the AOUP’s Clinical Research Unit through creation of follow-up forms submitted to corporate Operational Units’ physicians, which made it possible to track the number of patients involved in CU programmes, their clinical outcomes and duration of therapy. The economic impact was assessed by calculating cost-therapy for each patient based on drug dosage, duration of treatment, and ex-factory price published in the Official Gazette of Italian Republic, for drugs available on the market.

Results In the analysed period, a CU regimen was approved for 84 patients mainly in the haematology (17 patients) and paediatric (24) settings. Of the total, five patients did not start therapy due to death, clinical deterioration, or personal reasons. The remaining 79 underwent treatment. In 81% of cases, this resulted in a partial or complete improvement in the clinical status or, when degenerative diseases occurred, a stabilisation of the disease. On the economic side, avoided costs amounted to €7,130,668, 62% of which resulted from CU of burosumab in patients with X-linked hypophosphatemic osteomalacia.

Conclusion and Relevance CU in a University Hospital brings both clinical benefits and potentially significant economic savings. Early access to experimental therapies both enhances patients‘ life expectancy and quality and facilitates the gathering of valuable clinical data on promising investigational drugs. Cost savings generated from this approach can be reinvested to expand, enhance, and make the national healthcare system more sustainable. Collaboration between teaching hospitals, pharmaceutical companies and regulatory authorities is essential to optimise CU programmes and ensure equitable access to potentially lifesaving treatments.

References and/or Acknowledgements 1. Pilunni D, et al. Eur Rev Med Pharmacol Sci 2021;25,20, 6365.

Conflict of Interest No conflict of interest.