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Background: Compassionate drug use (CDU) provides early access to not yet authorised medicines and is funded by pharmaceutical companies. The observational retrospective study Compass-O monitored the CDU of onco-haematological drugs, managed by seven Italian units for cytotoxic drug preparations (Unità Farmaci Antiblastici [UFA]), between 1 January, 2016 and 31 December, 2021. Objective: We aimed to evaluate the CDU of onco-haematological drugs managed by seven Italian UFA, between 2016 and 2021. Methods: The seven UFA provided anonymised data concerning CDU approved in the study period. The early access and potential cost savings for the National Health System (Servizio Sanitario Nazionale [SSN]) were analysed for CDU concerning drug-therapeutic indication combinations with complete data and reimbursed by SSN up to December 2023 (date of study execution), according to the executive decision of the Italian Medicines Agency (Agenzia Italiana del Farmaco [AIFA]). Both analyses distinguished solid/liquid tumours and categorised the combinations as innovative (fully/conditionally) or non-innovative based on AIFA assessments. Results: Compass-O collected 783 CDU authorisations, with 572 (73.1%) analysable in terms of early access and cost savings. On average, early access amounted to 514 days and the total cost savings was €376,115,801. Compassionate drug use approvals involved mainly solid tumours (93.7% vs 6.3% for liquid tumours), and the combination of trastuzumab emtansine-breast cancer was the most dispensed (n = 73; early access = 426 days; potential cost savings: €610,388). Out of 572 CDU approvals, 200 (35%) were innovative drug-therapeutic indication combinations, with 598 days of early access and a total potential saving of €113,124,069. Conclusions: The study Compass-O showed a significant economic burden of CDU and a relevant need for early access, particularly for innovative drugs. However, there is currently no structured monitoring of CDU in Italy, suggesting the need for a national observatory, of which Compass-O can be the pilot phase. [ABSTRACT FROM AUTHOR]

Objective: The term Spinal Muscular Atrophy (SMA) identifies a group of genetic disorders affecting spinal motor neurons. It is caused by the loss of the SMN1 gene, resulting in degeneration of spinal alpha motor neurons and muscle atrophy. This study is focused on innovative gene therapies with onasemnogene abeparvovec approved in Italy in March 2021 with full reimbursement by the National Health Service. The objective pursued is verify, by means of the CHOP-INTEND scores obtained, whether therapy with onasemnogene abeparvovec led to an improvement in the clinical picture of the treated subjects and any adverse reactions that occurred. Methods: this study was conducted by evaluating the scores in the different re-evaluations of individual patients treated in our hospital (Fondazione Policlinico Universitario Agostino Gemelli IRCCS – Rome) and comparing them with the results of the CL-303 study described in SPC (Summary of Product Characteristics). The data were extracted from the patients' clinical records on the AIFA (Agenzia Italiana del Farmaco – Italian Medicines Agency) registries, also collecting information on any post-infusion ADRs. Everything was then represented graphically to have a clear comparison with the data from the study registered for drug approval. Results: from the data obtained, 7 out of 8 patients improved their health status post infusion with, in some cases, a significant increase in score. Conclusions: this result allows us to understand how crucial it is to start treatment as soon as possible after the diagnosis of the condition as the greatest improvements were seen in subjects who received treatment within 2 months of birth. [ABSTRACT FROM AUTHOR]

This document illustrates the results of the work of two interdisciplinary and multistakeholder panels (researchers, public institutions, and industry representatives) on drug territorialization and digitalization, organized as part of a residential seminar held on 30 September and 1st October 2021. Arising from some considerations about the demand for health and the provisions of the National Recovery and Resilience Plan (PNRR), the discussion touched various aspects of managing the transition from current to future management models. The importance of identifying criteria for prioritizing interventions in the area emerged: different methods of drug delivery, scientific information and measurement, re-evaluation of pathologies that can be managed in this area. Finally, the role of digitization within this change was explored. The opinions provided by the experts move towards making the most of the opportunities arising from PNRR, in terms of investments in healthcare and data application, with a view to improve health system efficiency, patient care and related outcomes. [ABSTRACT FROM AUTHOR]

In this article the pivotal role of hospital pharmacists in the multidisciplinary management of epilepsy is discussed. Hospital pharmacists are members of national and local ethics committees, oversee clinical trials, and ensure adherence to regulations for patient access to novel therapeutic treatments. They actively contribute to regulatory processes and the definition of prescribing centers. In the post-launch phase, hospital pharmacists are a key member in the multidisciplinary team, they are involved in decisions relating to the local introduction of drugs, in the management of the drug within the hospital structure and with the direct distribution, and to ensure proper and timely treatment. The pharmacovigilance network, including hospital and community pharmacists, monitors and prevents adverse effects related to epilepsy medications and enhances a collaborative approach with specialists to promote prescription appropriateness, targeting therapy for better patient outcomes. Finally, the potential benefits of deprescribing are briefly discussed, underscoring the importance of a multidisciplinary approach involving doctors and clinical pharmacists to gather comprehensive data and enhance patient care in epilepsy management., (© 2024 The Authors.)

Background: Clostridioides difficile infection (CDI), a leading cause of nosocomial deaths, is a microbiota-mediated disease. As such, the use of broader spectrum antibiotics, such as vancomycin and metronidazole, can prime the gastrointestinal tract to become more prone to CDI recurrences. Fidaxomicin, a narrow-spectrum antibiotic, has been demonstrated to be superior in preventing recurrence and in preserving the intestinal microbiota; however, widespread employment worldwide has been hindered due to high acquisition costs., Objectives: To integrate the currently available guidelines on the management of CDI and to shed light on the timeliest employment of fidaxomicin., Methods: An expert panel was gathered to obtain consensus using Delphi methodology on a series of statements regarding the management of CDI and on appropriate antibiotic use., Results: Consensus was reached on 21 of the 25 statements addressing the management of CDI., Conclusions: Delphi methodology was used to achieve consensus on the management of CDI, on the identification of patients at risk of recurrences or severe infection, and on the most appropriate use of fidaxomicin, with the final aim of fostering clinical practice application of treatment algorithms proposed by previous guidelines, in absolute synergy. It could be an important tool to promote more appropriate and cost-effective CDI treatments in European settings with limited resources, like Italy., (© The Author(s) 2024. Published by Oxford University Press on behalf of British Society for Antimicrobial Chemotherapy.)