A sustainable resource management model: Establishment of an orphan drug-day for the administration of patisiran
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Hygiene Unit, Department of Biomedical and Neuromotor Sciences, University of Bologna Via San Giacomo 12, 40126, Bologna, Italy
Pharmaceutical Department IRCCS Istituto delle Scienze Neurologiche di Bologna - AUSL Bologna Via Altura 3, 40139, Bologna, Italy
UOC Health Management IRCCS Istituto delle Scienze Neurologiche di Bologna - AUSL Bologna Via Altura 3, 40139, Bologna, Italy
Publication date: 2023-04-26
Popul. Med. 2023;5(Supplement):A1794
Background and Objective:
Patisiran is the first therapy based on short interfering RNA technology indicated for a lifelong treatment of hereditary transthyretin-mediated amyloidosis. It is administered through intravenous infusion at a dosage of 0.3 mg/kg of body weight (KBW) once every three weeks. Patisiran is supplied in single-use 10 mg/5mL vials, and any drug residues must be disposed of immediately; it is sold at an ex-factory price of €8,529.41/vial. This work aims to demonstrate how unifying patisiran administration sessions on a single day (‘Drug-Day’) for patients followed by different clinicians is advantageous for containing pharmaceutical expenditure.

We conducted an observational study in an Italian Research Institute for Neurological Sciences (ISNB) in the period between February 2021 and November 2022, divided into two phases: Phase 1 (February 2021-March 2022) and Phase 2 (April-November 2022), corresponding to before and after the establishment of Drug-Days, respectively. Applying this model (Drug-Day), at the beginning of Phase 2 at the ISNB the nine patients undergoing treatment with patisiran were divided into two groups for two distinct drug administration sessions according to their body weight: the sum of the total body weight of each group was a value very close to a multiple of 33 (each vial covers 33.3 KBW).

During Phase 1 patisiran monthly wastage was on average 9.2% of the vials used; during Phase 2, we recorded a drug wastage of 1.8%. The data collected show that this strategy resulted in a saving of €27.29/KBW/administration and €32,480/model patient (70 KBW)/year (corresponding to 3.8 vials).

This study showed that careful management of drug administration sessions, by reducing the leftover drug, allows creation of a more economically sustainable model and redirection of resources. Therefore, a multidisciplinary collaboration between different professionals is necessary for implementation of strategies to make pharmaceutical spending more sustainable.