Despite the challenges posed by the current pandemic, this year we reached several achievements. We did not give up and we are carrying out all our activities. You can find some insights in this newsletter and you can visit our websitefor further updates.
If you wish to support our initiatives, including those involving patients,you can help us with afree contribution.
Thanks in advance!
ACROSS THE WORLD Insights and updates on COVID-19 emergency
FIRST VACCINE FOR COVID-19 APPROVED IN EUROPE
European Medicines Agency(EMA) has recommended granting aconditional marketing authorisationfor the vaccine Comirnaty, developed by BioNTech and Pfizer, to prevent COVID-19 in people from 16 years of age.TheEuropean Commissionauthorised Comirnaty against COVID-19 and the next step will be to ensure that the first doses are delivered, so that the vaccination programmes can start from 27 December across Europe. Discover more on the other candidate vaccines for COVID-19 under evaluation by EMA here. To learn more about the current scenario in terms of authorised treatments for COVID-19 and clinical studies performed worldwide, do not hesitate to visit our COVID-19 page!
AVAILABLE AUTHORISED MEDICINE FOR COVID-19: DEXAMETHASONE
Among the candidate treatments, EMA endorses the use of Dexamethasone in COVID-19 patients on oxygen or mechanical ventilation. EMA’s Human Medicines Committee has completed itsreviewof the results from theRECOVERY study, a phase II-III trial including an arm that involved the use of the Dexamethasone in the treatment of patients with COVID-19 admitted to hospital. Dexamethasone was first considered a potential treatment for COVID-19 because of its ability to reduce inflammation, which plays a crucial role in the disease process.
Aliyu Mande, health information officer at Ahmadu Bello Teaching Hospital, Zaria (Nigeria), replied to some questions about the COVID-19management in his country with a focus on Sickle Cell Disease (SCD) patients. The interview is in the context of the FGB activitieswithin the ARISE project. Aliyu, involved in the staff exchange programme foreseen within the project, spent some time at FGB in Bari and is committed to implement in Nigeria communication and dissemination activities on SCD.
This year the XIII FTC “Challenges for researchers and regulators facing the pandemic crisis"was held virtually and in a shorter version on 23 October 2020. It was focused on theEuropean new pharmaceutical strategythat aims to improve and accelerate patients’ access to high-quality, safe and affordable medicines and to support further innovation in the European pharmaceutical sector.
To learn more in detail about the topics discussed during the course visit our website here, where you can freely consult some of the teaching material shared during the webinar and watch the recordings of some speeches.
COVID-19 AND HAEMOGLOBINOPATHIES: THE PATIENTS’ VOICE
COVID-19, in addition to being a threat to all patients especially those suffering from chronic diseases, including hemoglobinopathies, has negatively impacted on the access to treatments. Instead, it is crucial to ensure that patients have access to their care in a safe and continuous way! For this reason, we launched a new initiative on blood transfusions and on the value of blood donations. A representative of Associazione Italiana Thalassemici – Sez. Prov. Bariagreed to share with us the patients’ point of view.
"For each patient blood represents the essence of life, being able to achieve your life projects and having an active role in the society. That is why blood donors are life donors!"
Within the ARISE project, the webinar “Newborn Sickle Cell Disease Screening“ was held on22 July 2020.
Relevant experts in the field, gave an interesting insight on SCD newborn screening, touching on all aspects, from a blueprint for developing a service to the comprehensive care of affected babies. It was especially informative for developing a new service in Low and Middle Income Countries. Discover more about the ARISE webinar series here!
FONDAZIONE BENZI CONTRIBUTES TO THE EMA DISCUSSION PAPER ON SECONDARY USE OF DATA
Fondazione Benzi provided with its contribution to the EMA Discussion Paper“The General Data Protection Regulation: Secondary Use of Data for Medicines and Public Health Purposes”. Despite the huge spread of the FAIR (Findable, Accessible, Interoperable and Reusable) data principles concept and application and the rapid technological development that could lead to fast scientific advances, challenges for the protection of personal data exist and need to be faced. Fondazione Benzi’s team went through the EMA discussion paper and provided with considerations and suggestions on relevant issues that EMA should better address.
THE ENCEPP GUIDE ON METHODOLOGICAL STANDARDS IN PHARMACOEPIDEMIOLOGY
The eighth revision of the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) Guide on Methodological Standards in Pharmacoepidemiology was published on the ENCePPwebsite.
FGB researchers, are actively committed in ENCEPP activities with special reference to the Inventory of EU data sources and methodological approaches for multi-source studies. The latest revision of the Guide highlights also the importance of the Guide for pharmacoepidemiologic studies to be conducted in the context of the COVID-19 pandemic.
PUBLICATIONS Latest publications by our researchers
MACHINE LEARNING SYSTEMS APPLIED TO HEALTH DATA AND SYSTEM
Fondazione Gianni Benzi published a new article on the special issue “Innovation and Healthcare”of the European Journal of Health Law.
The article “Machine Learning Systems Applied to Health Data and System” is focused on Artificial Intelligence, Machine Learning and Data Science, key areas of interest at Fondazione Benzi, that are rapidly developing in healthcare, as well as on their translation into laboratory medicine and clinical practise.
EVALUATION OF THE EFFICACY AND SAFETY OF DEFERIPRONE
The article “Evaluation of the effectiveness and safety of Deferiprone compared to Deferasirox in pediatric patients with hemoglobinopathies (DEEP-2)” was recently published on The Lancet Haematology Journal. DEEP-2 is one of three studies foreseen in the DEEP(DEferiprone Evaluation in Paediatrics) project, in which Fondazione Benzi was involved. The project aimed to integrate the existing information on Deferiprone use in paediatric patients, thus covering the lack of information and providing a valid support to the use of the drug in this population.
ETHICAL, LEGAL AND REGULATORY ISSUES OF PAEDIATRIC TRANSLATIONAL RESEARCH. CALL FOR AN ADEQUATE MODEL OF GOVERNANCE
Fondazione Gianni Benzi collaborated in the article “Ethical, Legal and Regulatory Issues of Paediatric Translational Research. Call for an Adequate Model of Governance” that was published on the European Journal of Health Law.
The lack of paediatric medicines, including innovative and advanced ones, is a long-lasting and well-known problem at European and international level. Despite the existing legal frameworks and incentives, children remain deprived of many therapies tailored for them. The authors clarify the concept of translational research. Moreover, ethical, legal and regulatory issues particularly relevant in a children’s rights perspective are identified and pointed out.
A Call for specialistsandexpertsin Biostatistics and Epidemiology has just been published by the European Medicines Agency (EMA). In addition, a call for specialistsandexperts in Data Sciences is now open as well.
These experts will play a key role in the transformation of the Agency into a modern, data-driven organisation providing efficient services in the delivery of evidence to support benefit-risk decision-making on the development, authorisation and use of medicines.
The deadline for applications is 4 January 2021.
XVII SECOND LEVEL POST-GRADUATE MASTER IN REGULATORY SCIENCE "G.BENZI"
The University of Pavia has launched the XVII second level post-graduate Master in Regulatory Sciences “G.Benzi”. The Master aims to provide with the necessary expertise for new professional profiles in the regulatory activities, that are currently searched for by businesses and authorities working in the healthcare and industrial fields. The deadline for the submission of applications is 26 January 2021. Information on the call and instructions are available here.
Among the candidate treatments, EMA endorses the use of Dexamethasone in COVID-19 patients on oxygen or mechanical ventilation. 
Aliyu Mande, health information officer at Ahmadu Bello Teaching Hospital, Zaria (Nigeria), replied to some questions about the COVID-19 management in his country with a focus on Sickle Cell Disease (SCD) patients. The interview is in the context of the FGB activities
This year the XIII FTC 
COVID-19, in addition to being a threat to all patients especially those suffering from chronic diseases, including hemoglobinopathies, has negatively impacted on the access to treatments. Instead, it is crucial to ensure that patients have access to their care in a safe and continuous way! 
Within the ARISE project, the webinar “Newborn Sickle Cell Disease Screening“ was held on 22 July 2020.
Fondazione Benzi provided with its 
The eighth revision of the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) Guide on Methodological Standards in Pharmacoepidemiology was published on the 
Fondazione Gianni Benzi published a new article on the special issue “Innovation and Healthcare” of the European Journal of Health Law. 
The article “Evaluation of the effectiveness and safety of Deferiprone compared to Deferasirox in pediatric patients with hemoglobinopathies (DEEP-2)” was recently published on The Lancet Haematology Journal. DEEP-2 is one of three studies foreseen in the 
Fondazione Gianni Benzi collaborated in the article “Ethical, Legal and Regulatory Issues of Paediatric Translational Research. Call for an Adequate Model of Governance” that was published on the European Journal of Health Law.
The University of Pavia has launched the XVII second level post-graduate Master in Regulatory Sciences “G.Benzi”. The Master aims to provide with the necessary expertise for new professional profiles in the regulatory activities, that are currently searched for by businesses and authorities working in the healthcare and industrial fields. The deadline for the submission of applications is 26 January 2021. Information on the call and instructions are available 
