Rita Banzi

Mario Negri Institute for Pharmacological Research | Mario Negri · Mario Negri - Milan

Introduction Access to medicines is one of the biggest challenges to health systems, affecting society and individuals. This study aims to explore citizens' opinions, perceptions and attitudes on the model of medicines' research and development (R&D) and price setting of medicines reimbursed by the Italian National Health Service. Materials and Me...

Objectives: Guidelines and essential medicine lists (EMLs) bear similarities and differences in the process that lead to decisions. Access to essential medicines is central to achieve universal health coverage. The World Health Organization (WHO) EML has guided pri-oritization of essential medicines globally for nearly 50 years, and national EMLs (...

Essential medicine lists (EMLs) are important medicine prioritization tools used by the World Health Organization (WHO) EML and over 130 countries. The criteria used by WHO’s Expert Committee on the Selection and Use of Essential Medicines has parallels to the GRADE Evidence-to-Decision (EtD) frameworks. In this study, we explored the EtD framework...

Antipsychotic use in Alzheimer disease (AD) is associated with adverse events and mortality. Whilst postulated to cause/exacerbate orthostatic hypotension (OH), the exact relationship between antipsychotic use and OH has never been explored in AD—a group who are particularly vulnerable to neuro-cardiovascular instability and adverse effects of medi...

This project of Health technology assessment was aimed at defining the impacts of offering a cystic fibrosis (CF) carrier screening to the general population, compared to the current situation, where the test is offered to individuals at high-risk to give birth to a child with CF. Results revealed: i) a lack of robust and updated data; ii) a return...

Background Many interventions, especially those linked to open science, have been proposed to combat the reproducibility crisis. To what extent these propositions are based on scientific evidence from empirical evaluations is not clear. Aims The primary objective is to identify interventions that have been formally investigated regarding their infl...

Background Germinal matrix hemorrhage and intraventricular hemorrhage (GMH‐IVH) may contribute to neonatal morbidity and mortality and result in long‐term neurodevelopmental sequelae. Appropriate pain and sedation management in ventilated preterm infants may decrease the risk of GMH‐IVH; however, it might be associated with harms. Objectives To su...

When conducting randomised clinical trials, the choice of methodology and statistical analyses will influence the results. If the planned methodology is not of optimal quality and predefined in detail, there is a risk of biased trial results and interpretation. Even though clinical trial methodology is already at a very high standard, there are man...

[This corrects the article DOI: 10.1155/2021/9996193.].

Background Personalised medicine is a medical model that aims to provide tailor-made prevention and treatment strategies for defined groups of individuals. The concept brings new challenges to the translational step, both in clinical relevance and validity of models. We have developed a set of recommendations aimed at improving the robustness of pr...

Biological medicines have improved patients’ outcomes, but their high costs may limit access. Biosimilars, alternatives that have demonstrated high similarity in terms of quality, safety, and efficacy to an already licensed originator biological product, could increase competition and decrease prices. Given the expanding number of biosimilars, pati...

The introduction of personalized medicine, through the increasing multi-omics characterization of disease, brings new challenges to disease modeling. The scope of this review was a broad evaluation of the relevance, validity, and predictive value of the current preclinical methodologies applied in stratified medicine approaches. Two case models wer...

Background: Rising expenditure for new cancer medicines is accelerating concerns that their costs will become unsustainable for universal healthcare access. Moreover, early market access of new oncology medicines lacking appropriate clinical evaluation generates uncertainty over their cost-effectiveness and increases expenditure for unknown health...

Objective Personalised medicine (PM) allows treating patients based on their individual demographic, genomic or biological characteristics for tailoring the ‘right treatment for the right person at the right time’. Robust methodology is required for PM clinical trials, to correctly identify groups of participants and treatments. As an initial step...

Personalized medicine requires large cohorts for patient stratification and validation of patient clustering. However, standards and harmonized practices on the methods and tools to be used for the design and management of cohorts in personalized medicine remain to be defined. This study aims to describe the current state-of-the-art in this area. A...

Background We aimed to elucidate which vaccines were accepted by European countries as valid proof of vaccination against COVID-19 for international travelers. Method On 27-September-2021 a cross-sectional study was conducted on VisaGuide.World, that reports on valid vaccines for international travelers. Other databases, lay press and regulatory a...

Background Transient tachypnoea of the newborn (TTN) is characterised by tachypnoea and signs of respiratory distress. It is caused by delayed clearance of lung fluid at birth. TTN typically appears within the first two hours of life in term and late preterm newborns. Although it is usually a self‐limited condition, admission to a neonatal unit is...

Sales of medicines continue to grow world-wide driven in part by increasing expenditures on biological medicines leading to concerns with the long-term sustainability of European healthcare systems. The increasing use of biosimilars at lower costs can potentially address this. However, there can be concerns with their prescribing. There are also co...

Questo articolo riflette su alcune delle attuali sfide della medicina basata sulle prove di efficacia (evidence-based medicine - EBM) in Italia. Gli autori, che condividono un impegno ventennale nel campo della ricerca clinica, discutono quella che definiscono una fase di “stagnazione” nella pratica e nell’insegnamento dei metodi e degli strumenti...

Objective: To review biomarker discovery studies using omics data for patient stratification which led to clinically validated FDA-cleared tests or laboratory developed tests, in order to identify common characteristics and derive recommendations for future biomarker projects. Design: Scoping review. Methods: We searched PubMed, EMBASE and Web...

Background. Diabetes mellitus rates and associated costs continue to rise across Europe enhancing health authority focus on its management. The risk of complications is enhanced by poor glycaemic control, with long-acting insulin analogues developed to reduce hypoglycaemia and improve patient convenience. There are concerns though with their consid...

Background Antiplatelet drugs may prevent recurrent ischemic events after ischemic stroke but their relative effectiveness and harms still need to be clarified. Within this network meta-analysis we aimed to summarize the current evidence for using antiplatelet drugs for secondary stroke prevention. Methods We searched MEDLINE, EMBASE and CENTRAL u...

Measurements of serum concentrations of therapeutic antibodies and anti-drug antibodies (ADA) can support clinical decisions for the management of non-responders, optimizing the therapy. In the present study we compared the results obtained by classical ELISA and a recently proposed surface plasmon resonance (SPR)-based immunoassay, in 76 patients...

Background: Diabetes mellitus rates continue to rise, which coupled with increasing costs of associated complications has appreciably increased global expenditure in recent years. The risk of complications are enhanced by poor glycaemic control including hypoglycaemia. Long-acting insulin analogues were developed to reduce hypoglycaemia and improve...

Simple Summary In 2019, the Fondazione Italiana Linfomi research team proposed a new project to summarize the evidence on late sequelae after treatment of classical Hodgkin lymphoma and diffuse large B cell lymphoma in long-term survivors (>5 years free of disease). Six systematic reviews were conducted. Clinical research questions focused on the i...

Background: Diabetes mellitus rates continue to rise, which coupled with increasing costs of associated complications has appreciably increased global expenditure in recent years. The risk of complications are enhanced by poor glycaemic control including hypoglycaemia. Long-acting insulin analogues were developed to reduce hypoglycaemia and improve...

Introduction: The MonCOVID study aimed to assess the feasibility of early home management and monitoring of patients with suspected or confirmed CoViD-19, and to evaluate if this would ensure a rapid and adequate transfer to hospital care. Methods: This pilot, open-label, single-arm study included adult symptomatic patients with suspected or con...

Background: the Covid-19 pandemic has provoked a huge of clinical and epidemiological research initiatives, especially in the most involved countries. However, this very large effort was characterized by several methodological weaknesses, both in the field of discovering effective treatments (with too many small and uncontrolled trials) and in the...

Objectives Antidepressant use is often reported as a risk factor for Orthostatic Hypotension (OH), however this relationship has never been explored in those with mild/moderate Alzheimer Disease (AD), who may represent a particularly vulnerable cohort. Methods We performed a cross‐sectional analysis of baseline data from the NILVAD study. Particip...

Background: Given the increasing number and heterogeneity of data repositories, an improvement and harmonisation of practice within repositories for clinical trial data is urgently needed. The objective of the study was to develop and evaluate a demonstrator repository, using a widely used repository system (DSpace), and then explore its suitabilit...

Imatinib, the first tyrosine kinase inhibitor (TKI) for the treatment of chronic myeloid leukemia (CML), improves overall survival (OS), but the introduction of newer TKIs requires the definition of the optimal first-line TKI for newly diagnosed Philadelphia chromosome–positive (Ph+) chronic-phase (CP) CML. This systematic review of randomized cont...

Objective to assess the inter-rater reliability (IRR) and usability of the revised Cochrane risk-of-bias tool for randomised trials (RoB 2). Study design cross-sectional study. Four raters independently applied RoB 2 on the primary outcome of a random sample of individually randomized parallel-group trials (RCTs). We calculated the Fleiss’ Kappa f...

Background: Given the increasing number and heterogeneity of data repositories, an improvement and harmonisation of practice within repositories for clinical trial data is urgently needed. The objective of the study was to develop and evaluate a demonstrator repository, using a widely used repository system (DSpace), and then explore its suitabilit...

Purpose: To review the marketing authorization of biosimilars and provide a critical analysis of the pivotal trials supporting their approval by the European Medicines Agency (EMA). Methods: EMA website to identify the biosimilars approved up to July 2019 and the European Public Assessment Report for information on pivotal trial design, duration...

The protocol of a Cochrane review has no abstract. Why it is important to do this overview Although TTN usually resolves within 48 hours, the infant may benefit from neonatal care with respiratory support provided during this time. This intensive care might be traumatic for parents, can limit parent-child bonding, and might delay the first breast-...

Several outpatient models for the follow-up of cancer survivors have been developed worldwide. A multidisciplinary approach is often necessary to guarantee the best monitoring of long-term toxicities. Guidelines also indicate a close education on healthy lifestyles. In this context, we have analyzed the Italian follow-up modalities of lymphoma surv...

The Food and Drug Administration (FDA) and European Medicines Agency (EMA) now have expedited review procedures for new drugs. We compared the review times of medicines licensed by the 2 agencies and explored differences in the evidence submitted. In 2015–2017 the FDA licensed 113 drugs, 66 of which reached Europe. The median review time was longer...

Importance: Sophisticated evidence-based information resources can filter medical evidence from the literature, integrate it into electronic health records, and generate recommendations tailored to individual patients. Objective: To assess the effectiveness of a computerized clinical decision support system (CDSS) that preappraises evidence and...

BACKGROUND: Hypertension is common among patients with Alzheimer disease. Because this group has been excluded from hypertension trials, evidence regarding safety of treatment is lacking. This secondary analysis of a randomized controlled trial assessed whether antihypertensive treatment increases the prevalence of orthostatic hypotension (OH) in p...

Objective: To assess the inter-rater reliability (IRR) and usability of the risk of bias in nonrandomized studies of interventions tool (ROBINS-I). Study design and setting: We designed a cross-sectional study. Five raters independently applied ROBINS-I to the nonrandomized cohort studies in three systematic reviews on vaccines, opiate abuse, an...

Background Statins may prevent recurrent ischemic events after ischemic stroke. Determining which statin to use remains controversial. We aimed to summarize the evidence for the use of statins in secondary prevention for patients with ischemic stroke by comparing benefits and harms of various statins. Methods We searched for randomized controlled...

Background Data repositories have the potential to play an important role in the effective and safe sharing of individual-participant data (IPD) from clinical studies. We analysed the current landscape of data repositories to create a detailed description of available repositories and assess their suitability for hosting data from clinical studies,...

Objectives To gather knowledge on the current debate, opinions and attitudes of Italian patient and citizen groups on individual participant data (IPD) sharing from clinical studies. Design Cross-sectional online survey. Setting and participants A 22-item online questionnaire was sent by email to 2003 contacts of patient and citizen groups in Ita...

BACKGROUND: This study reports the findings of the first large-scale Phase III investigator-driven clinical trial to slow the rate of cognitive decline in Alzheimer disease with a dihydropyridine (DHP) calcium channel blocker, nilvadipine. Nilvadipine, licensed to treat hypertension, reduces amyloid production, increases regional cerebral blood flo...

Detailed statistical methods. (DOCX)

Study design and treatment. (DOCX)

Personal correspondence. (DOCX)

ADAS-Cog 12 sex subgroup analysis results. ADAS-Cog 12, Alzheimer's Disease Assessment Scale Cognitive-12. (DOCX)

Trial-associated boards. (DOCX)

Distribution of treatment arms recruited in each site. (DOCX)

ADAS-Cog 12 severity subgroup analysis results. ADAS-Cog 12, Alzheimer's Disease Assessment Scale Cognitive-12. (DOCX)

Clinical chemistry and haematology findings at screening visit and week 78. (DOCX)

ADAS-Cog 12 APOE ε4 subgroup analysis results. ε4, epsilon 4 allele; ADAS-Cog 12, Alzheimer's Disease Assessment Scale Cognitive-12; APOE, Apolipoprotein E gene. (DOCX)

MedDRA coded adverse events per group. MedDRA, Medical Dictionary for Regulatory Activities. (XLSX)

Background: New direct-acting antiviral agents (DAAs) approved for the treatment of patients infected by Hepatitis C virus (HCV) are well tolerated and increase sustained virological response (SVR) rate. We summarize current evidence on the efficacy and safety from comparative randomized controlled trials (RCTs) of DAAs. Methods: We systematical...

Objective: To review the evidence supporting the European Union marketing authorization of drugs for multiple sclerosis (MS) and assess how far postmarketing research addresses information gaps at the time of approval. Methods: Through its database, we identified drugs approved by the European Medicines Agency and gathered data on pivotal trials...

Background: In recent years, a cultural change in the handling of research data has resulted in the promotion of a culture of openness and an increased sharing of data. In the area of clinical trials, sharing of individual participant data involves a complex set of processes and the interaction of many actors and actions. Individual services and to...

Objectives: To assess the inter-rater reliability of AMSTAR and ROBIS in judging individual domains and overall methodological quality/risk of bias of systematic reviews, the concurrent validity of the tools and the time required to apply them. Study design and setting: Cross-sectional. Five raters independently read 31 systematic reviews and ap...

Background: In recent years, a cultural change in the handling of data from research has resulted in the strong promotion of a culture of openness and increased sharing of data. In the area of clinical trials, sharing of individual participant data involves a complex set of processes and the interaction of many actors and actions. Individual servic...

Background: The use of e-learning, defined as any educational intervention mediated electronically via the Internet, has steadily increased among health professionals worldwide. Several studies have attempted to measure the effects of e-learning in medical practice, which has often been associated with large positive effects when compared to no in...

Objectives. We examined major issues associated with sharing of individual clinical trial data and developed a consensus document on providing access to individual participant data from clinical trials, using a broad interdisciplinary approach. Design and methods. This was a consensus-building process among the members of a multistakeholder task fo...

Using the best quality of clinical research evidence is essential for choosing the right treatment for patients. How to identify the best research evidence is, however, difficult. In this narrative review we summarise these threats and describe how to minimise them. Pertinent literature was considered through literature searches combined with perso...

Objectives We examined major issues associated with sharing of individual clinical trial data and developed a consensus document on providing access to individual participant data from clinical trials, using a broad interdisciplinary approach. Design and methods This was a consensus-building process among the members of a multistakeholder task for...

Background Evidence-based clinical practice is challenging in all fields, but poses special barriers in the field of rare diseases. The present paper summarises the main barriers faced by clinical research in rare diseases, and highlights opportunities for improvement. Methods Systematic literature searches without meta-analyses and internal Europ...

This is a protocol for a Cochrane Review (Overview). The objectives are as follows: To summarize the evidence from systematic reviews regarding the effects and safety of pharmacological interventions related to pain and sedation management in order to prevent GMH-IVH in ventilated preterm infants.

The process fails to improve the evidence after early licensing Like other expedited procedures for early marketing authorisation, conditional approval12 stems from the assumption that immediate availability of new drugs offers patients a benefit that outweighs the risks of limited clinical information. To some extent, this assumption applies to a...

Purpose: Early access to new treatment options should not preclude accurate research planning, especially for rare diseases and fragile populations. Taking neuroblastoma as a model case, we analyzed the rationale supporting the search for future therapeutic strategies in the light of preclinical and clinical evidence. Methods: We reviewed ongoin...

Background Computerized decision support systems (CDSSs) are computer programs that provide doctors with person-specific, actionable recommendations, or management options that are intelligently filtered or presented at appropriate times to enhance health care. CDSSs might be integrated with patient electronic health records (EHRs) and evidence-bas...

BACKGROUND: Computerized decision support systems (CDSSs) are computer programs that provide doctors with person-specific, actionable recommendations, or management options that are intelligently filtered or presented at appropriate times to enhance health care. CDSSs might be integrated with patient electronic health records (EHRs) and evidence-ba...

Evidence-based healthcare requires the integration of the best research evidence with clinical expertise and patients' values. International publishers are developing evidence-based information services and resources designed to overcome the difficulties in retrieving, assessing and updating medical information as well as to facilitate a rapid acce...

Introduction: One of the aims of Evidence-Based Medicine is to improve quality and appropriateness of care by the expedition of the knowledge transfer process. Computerized Decision Support Systems (CDSSs) are computer programs that provide alerts to the prescribing doctor directly at the moment of medical examination. In fact, alerts are integrat...

Introduction The opening of research data is emerging thanks to the increasing possibilities of digital technology. The opening of clinical trial (CT) data is a part of this process, expected to have positive scientific, ethical, health, and economic impacts thus contributing to research integrity. The January 2016 proposal by the International Cou...

Background The age gap between participants in trials and patients who could benefit from the drugs studied has been widely documented across different clinical areas. Patients with dementia included in clinical research are systematically younger than those in the general population. We examined the age gap between participants in recent clinical...

This is the protocol for a review and there is no abstract. The objectives are as follows: We assessed the efficacy and safety profile of STN DBS surgery compared to the best available medical treatment or other deep brain surgery procedures in patients with idiopathic PD.