Search

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.

Background: Leber congenital amaurosis is the first form of inherited retinal dystrophy (IRD) treated with a gene therapy approach using voretigene neparvovec (VN). To date, 42 patients have been treated, 25 of whom, treated at Clinica Oculistica Vanvitelli (Naples), have shown – after 45 days of treatment – a significant increase in dim-light visual function, a widening of the visual field, and an increase in central visual acuity. Objective: A workshop has been organized to foster rapid access, investigate the current organizational scenario, and identify a value framework suitable for making gene therapy delivery efficient in terms of efficacy, safety, and sustainability. Methods: The workshop involved 14 experts in clinical genetics, ophthalmology, and vitreoretinal surgery. All experts were subjected to questions related to three topics: diagnostic-therapeutic pathway of IRD patients, essential requirements for gene therapy delivery centres, and standardized model for therapy delivery. All contributions were judged to be of equal value. Results: The panel identified: the steps of the diagnostic pathway to achieve early diagnosis; the essential criteria that delivery centres must possess in terms of experience, multidisciplinary team, and technical equipment; a standardized model for VN administration. A network of few centres was identified as the best organizational model for VN delivery since it would ensure the gaining of experience and clinical excellence. Conclusion: This paper provides a perspective that can be used as a starting point to standardize the diagnostic-therapeutic pathway of IRD patients and define the essential requirements that centres must meet for VN administration.

Spasticity is a well-known motor dysfunction occurring after a stroke. A group of Italian physicians' experts in treating post-stroke spasticity (PSS) reviewed the current scientific evidence concerning the state-of-the-art clinical management of PSS management and the appropriate use of botulinum toxin, aiming to identify issues, possible actions, and effective management of the patient affected by spasticity. The participants were clinicians specifically selected to cover the range of multidisciplinary clinical and research expertise needed to diagnose and manage PSS. When evidence was not available, the panel discussed and agreed on the best way to manage and treat PSS. To address the barriers identified, the panel provides a series of consensus recommendations. This systematic review provides a focused guide in the evaluation and management of patients with PSS and its complications. The recommendations reached by this panel of experts should be used by less-experienced doctors in real life and should be used as a guide on how to best use botulinum toxin injection in treating spasticity after a stroke.

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.

This article illustrates a consensus opinion of an expert panel on the need and usefulness of a framework for price and reimbursement (P&R) process and managed entry agreements (MEAs) for orphan medicines in Italy. This opinion was gathered in three rounds: an introductory document was sent to the panel and discussed during a recorded online meeting. A second document was sent to the panel for their review. In the third step the final document was validated. Members of the expert panel are the authors of the article. The panel agreed that Italy does not need a specific value framework for orphan medicines, driving the P&R process. Rather, a more structured value framework for all medicines tailored to the specific drugs can be useful. For orphan drugs, the panel advocated for a multidisciplinary approach and the contribution of different stakeholders to value assessment, and acknowledged the importance of addressing, more than for other drugs, unmet needs, equity issues and societal value. The panel raised the need of increasing the importance of patient-reported outcomes. Experts, acknowledging the growing criticisms in implementation of outcome-based agreements in Italy, expressed their position against their abandonment in favour of discounts only and supported orphan medicines as natural candidates for these agreements. Finally, the panel made some recommendations on the appraisal process for orphan medicines, including an early discussion on the uncertainty of the evidence generated and the adoption of a structured approach to identify the agreement, which better responds to the uncertainty.

OBJECTIVE: Compassionate Drug Use (CDU) allows patients with a specific disease and no further treatment option to access unauthorized treatments. In this study, we analyzed the requests of CDU approved by the Ethics Committee of Fondazione Policlinico Gemelli in the period January 1, 2018-June 30, 2021. We also estimated the economic impact of CUs.MATERIALS AND METHODS: CDU requests were analyzed by year, by frequency and by regulatory status of the medicines requested. If an ex-factory price was available at the cutoff date of June 30, 2021, we estimated what would have been the costs for the National Health System (NHS) if the price was already negotiated at the time of CDU request.RESULTS: In the study period, 463 CDU requests were processed by the Ethics Committee. The number of requests increase linearly from 45 in 2018 to an estimated number of 260 in 2021. The requests included 68 medicines or combinations of medicines; 16 products out of 68 accounted for 75% of all requests. For 7 of these 16 highly requested treatments, accounting for 110 requests out of 463, it was possible to estimate the costs of therapies according to their ex-factory prices. If these products were to be purchased by the NHS, the estimated cost was euro 5.472.225.CONCLUSIONS: The access to unauthorized drugs through CDUs is undergoing a huge increase in the last few years. Such increase meets the ethical need to provide patients with the most recent, often innovative, therapeutic options.

Seasonal flu vaccination is one of the most important strategies for preventing influenza. The attitude towards flu vaccination in light of the COVID-19 pandemic has so far been studied in the literature mostly with the help of surveys and questionnaires. Whether a person chooses to be vaccinated or not during the COVID-19 pandemic, however, speaks louder than any declaration of intention. In our teaching hospital, we registered a statistically significant increase in flu vaccination coverage across all professional categories between the 2019/2020 and the 2020/2021 campaign (24.19% vs. 54.56%, p < 0.0001). A linear regression model, based on data from four previous campaigns, predicted for the 2020/2021 campaign a total flu vaccination coverage of 30.35%. A coverage of 54.46% was, instead, observed, with a statistically significant difference from the predicted value (p < 0.0001). The COVID-19 pandemic can, therefore, be considered as an incentive that significantly and dramatically increased adherence to flu vaccination among our healthcare workers.

Concerns are growing about CNS drug prescription because of professional responsibility. This is due to Regulatory Authorities reimbursement policies aimed at promoting generic drugs because of their cost saving potential; this attitude, however, often does not take into account the primary goal of maintaining patient adherence to therapy. This situation is more complex in the psychiatric setting for chronic patients with cognitive or personality disorders. This article provides a review of regulatory, pharmacological and clinical issues (e.g. compliance) involved in the appropriate prescription of brand and generic drugs. It is important that medical prescribers are aware of these issues in order to comply with good clinical practice – given the specific characteristics of psychiatric patients – for a successful antipsychotic therapy in the era of generic drugs.