1,721,143 research outputs found
A New Era of Pharmacovigilance: Future Challenges and Opportunities
No full textMedicines safety monitoring is a continuous and dynamic process throughout all the phases of the life cycle of a drug. During the drug development, safety is investigated in different phases. In preclinical
studies, the primary goal of safety evaluation is the identification of a safe dose in humans and of safety parameters for clinical monitoring. In clinical phase, phase I studies are designed to estimate the tolerability of the dose range expected to be needed for later clinical studies in healthy volunteers;
phase II studies are focused on determining appropriate range of drug doses in patients with a disease or condition of interest, while phase III clinical trials are the most important studies to refine understanding of benefit-risk profile of the drug and to identify less common adverse drug reactions. Although drug safety evaluation is very rigorous and thorough, pre-marketing clinical trials have however intrinsic limitations that do not allow to exhaustively evaluate drug safety profile. These studies are conducted on limited numbers of patients that are selected based on strict eligibility criteria and not fully representing real-world populations and have limited duration, thus preventing detection of rare and long-term adverse reactions. Therefore, the post-marketing assessment of medicines plays a key role for better defining drugs’safety profile in real-world setting and filling the evidence gap of pre-marketing studies. In the field of drug safety and regulation, a number of challenges have to be faced in the near future. First of all, COVID-19 pandemic highlighted how relevant pharmacovigilance and proper risk communication during public health emergency are. Second, the development of advanced methodologies including machine learning techniques and the availability of large amount of electronic healthcare data offer opportunity for optimizing drug benefit-risk profile evaluation in real
world setting. Finally, innovative therapeutics, such as advanced therapy medicinal products, digital therapeutics, vaccines developed based on advanced technologies, requiring special pharmacovigilance monitoring have been increasingly marketed in recent years, often upon accelerated pathway approval
The role of real-world evidence for the study of rare diseases epidemiology and the post-marketing evaluation of orphan drugs
No full textIn Europe, diseases are defined as "rare" when they have a prevalence of less than 0.05% in the general population. When available, drugs used to treat such diseases often fall into the category of orphan drugs, i.e. drugs used for the diagnosis, prevention and treatment of rare diseases. Generating evidence about the efficacy and safety of orphan drugs in the pre-marketing phase is challenging due to critical issues related to the rarity of these diseases. This highlights the need to generate robust post-marketing evidence to fill the evidence gap in the pre-marketing phase, with the aim to more accurately define the benefit-risk profile and the cost-effectiveness of orphan drugs in the real-world setting. Real-world data (i.e., the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources) has significantly increased over the last years and their use is increasingly proposed, including by regulatory agencies, to generate evidence to support the regulatory authorization processes of orphan drugs and to study rare diseases. In this article, the authors describe the main sources of real-world data and their role to support the regulatory authorization processes of orphan drugs
Farmaci antiaritmici
No full textUn libro di Farmacologia originale e tutto italiano: i capitoli rispecchiano le linee guida internazionali rispetto ai farmaci e alla terapia farmacologica così come sono interpretate e applicate dalle società scientifiche italiane.
I vari capitoli sono stati assegnati a esperti degli argomenti trattati con formazione di un team autoriale qualificato e variegato. Molta cura si è posta poi nell'opera di revisione affinché il testo risultasse omogeneo.
Il libro è suddiviso in più sezioni. In quella di carattere generale, la prima, che tratta dell'appropriatezza terapeutica, dei farmaci equivalenti, dei biosimilari, della farmacosorveglianza ecc., si offrono al lettore gli strumenti culturali per affrontare temi che sono attuali e dibattuti, tuttavia risulta evidente che gli autori hanno espresso una posizione e non si sono limitati a scrivere un distaccato documento accademico.
Un'attenzione quasi maniacale è stata posta alla lunghezza dei singoli capitoli per fare del volume un'agile opera di riferimento per la farmacologia e non un'enciclopedia. Importanti parti di testo, più adatte all'approfondimento che non allo studio, sono state pubblicate sul sito web che accompagna il volume cartaceo, sul quale si ritrovano anche la bibliografia e i test di autovalutazione.
I test di autovalutazione, pensati per ogni capitolo e disponibili online su my.zanichelli.it, serviranno allo studente per valutare la propria preparazione in modo interattivo
Dati regionali e loro ruolo nella pianificazione, nella programmazione e nel controllo a livello regionale
No full textRegional administrative data may help public administrations in organizing their information, identifying problems, defining solutions and verifying results of the actions taken. The objective of this work is to present the discussion output of the working group (GDL) “The use of regional pharmaceutical data to facilitate health planning and outcome monitoring” which was held during the Regional Pharmaceutical Policy Forum of 2023. The GDL focused on identifying a model for planning, programming and monitoring health resources based on the data-driven approach and on the use of already existing regional administrative data, defining some gaps and developing proposals to implement this strategy. In particular, were highlighted the needs to adopt a broader perspective that considers healthcare costs as a whole (and not, specifically, oriented towards the governance of pharmaceutical assistance), to create ad-hoc training courses focused on the use of real-world data, and, similarly to some European Countries, to move towards an open access policy (accessible data) that can be useful to improve citizens’ health protection services, in compliance with the GDPR
Antipsychotic drug exposure and risk of pneumonia: a systematic review and meta-analysis of observational studies
No full textPneumonia is one of the major leading causes of morbidity and mortality among persons aged 65 years or older. Recently, several studies suggested an association between antipsychotic (AP) use and risk of pneumonia in elderly patients. The aim of the present systematic review and meta-analysis of observational studies was to investigate if first-generation and second-generation AP drugs increase the risk of pneumonia in the elderly and also in the younger population, and to ascertain the risk associated with exposure to individual drugs
Reply to: Deprescribing is associated with reduced readmission to hospital: An updated meta-analysis of randomized controlled trials
No full textNo abstract availabl
Digital Therapeutics in Perspective: From Regulatory Challenges to Post-Marketing Surveillance
No full textDigital therapeutics (DTx) are innovative therapeutic interventions, in which the therapeutic activity is carried out by algorithms and software. They represent a new opportunity especially for the treatment of chronic pathologies associated with dysfunctional lifestyles and behaviors, where conventional drug therapy is not fully effective. DTx are highly customizable therapeutic tools, allowing a better involvement of the patient in the management of the disease. To date, the clinical use of DTx in Europe is still generally limited. One of the main issues related to DTx is the general lack of education of healthcare professionals in this sector that leads to a knowledge gap between data scientists, and physicians, who should identify all those clinical needs that could be better addressed through the use of DTx. From a regulatory perspective, DTx are classified as Medical Devices. However, their research and development process is similar to that of conventional drugs, since they are tested in clinical trials and their approval refers to specific therapeutic indications. For this reason, precise criteria for marketing approval, for the health technology assessment and for the reimbursement of these therapies need to be defined. Moreover, as for conventional drugs, it is also fundamental to conduct postmarketing studies on DTx, aiming at re-evaluating the benefit-risk profile and collecting information related to large-scale use in real world setting. The aim of this review is to describe the main challenges for DTx development, regulation and widespread clinical use
Real-World Evidence e Managed Entry Agreements
No full textIntroduction:: Real-World Data (RWD) are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources such as Electronic Health Records (EHRs), data from product and disease Registries, patient generated data, etc.
Real-World Evidence (RWE) is the clinical evidence about the usage and potential benefits and/or risks of a medical product derived from the analysis of Real-World Data.
This document illustrates the results of a discussion of a multi-disciplinary expert Panel on the role of RWE with specific regard to both the post-marketing (re)evaluation of the benefit-risk profile of drugs in the real-world setting and the management of uncertainty about efficacy and safety at the time of their marketing, through Managed Entry Agreements (MEAs).
Method:: The debate took place during the 7th edition of the Seminari di Mogliano Veneto, attended by experts working in different organizations.
Results:: The experts have emphasized the need (i) to introduce outcome-based MEA for drugs with high uncertainty in outcomes and a high impact on spending, (ii) to evaluate the opportunity to define any secondary objectives in order to be able to carry out additional evaluations when establishing a new Registry, and (iii) to use other data sources through linkage and interoperability of the information systems involved (e.g., administrative databases, disease Registries).
The Panel also agreed on a general need to define guidelines at national level on how to generate RWE to support the various regulatory processes including evaluation for the purposes of applying MEA and price renegotiation
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