Newsletter no. 20 - May 2023

HIGHLIGHTS

SAVE THE DATE - “PUGLIA E THALASSEMIA - CONSAPEVOLEZZA, CONOSCENZA, CURA”

Coordinamento Thalassemici Regione Puglia is organising the conference "PUGLIA AND THALASSEMIA - Consapevolezza, Conoscenza, Cura" to be held on 10 June 2023 at Hotel Hi-Bari, Bari - Italy. The event, sponsored by Fondazione Gianni Benzi, Associazione Italiana Thalassemici Sez. Prov. Bari and Federazione Nazionale delle Associazioni - UNITED, aims at increasing awareness and knowledge on Thalassaemia enhancing the quality of care and therefore improving patients' lives.

THE EU REFORM TO PHARMACEUTICAL LEGISLATION FOR MORE ACCESSIBLE, AFFORDABLE AND INNOVATIVE MEDICINES

The European Commission has adopted the proposal of the revision of the European pharmaceutical legislation. It includes a proposal for a new Directive and a new Regulation, which revise and replace the existing pharmaceutical legislative framework, including the one on medicines for children and for rare diseases.

We cooperated in the “study supporting the Impact Assessment of the revision of the EU legislation on medicines for children and rare diseases” analysing the impacts of policy options to update the legal framework on medicines for children and rare diseases.

INTERNATIONAL THALASSAEMIA DAY - 2023

May 8^th, 2023 was the International Thalassaemia Day! By maximizing the knowledge and skills of patients with thalassaemia and their caregivers, positive changes in health behaviours, health outcomes, quality of life and disease burden can be achieved. We firmly believe that patients are key actors in the scientific research process and healthcare advancements. We encourage patients’ involvement in this framework to ensure that their health needs are better identified and met. Therefore, we are actively committed in providing patients and citizens with accurate and reliable information on specific topics.

FEDELE BONIFAZI’S SPEECH AT THE WORKSHOP ON ARTIFICIAL INTELLIGENCE IN PAEDIATRICS – BARI

Fedele (Duccio) Bonifazi, president of Fondazione Gianni Benzi, spoke about the upcoming revision of both the Paediatric and Orphan Regulations during the workshop “L’intelligenza artificiale a tutela della salute in età pediatrica. Implementazione di una piattaforma digitale per il design di farmaci pediatrici sicuri” held on 30 March in Bari, Italy. The workshop provided multidisciplinary contributions aimed to encourage the discussion on how the artificial intelligence contributes to the identification and development of safer paediatric medicines.

"HOT TOPICS": NEW SECTION ON THE FGB WEBSITE

“Hot topics” is the new section on our website. It includes relevant contributions and interviews from our partners and collaborators aimed to put the spotlight on specific topics in the field of scientific research, innovation, paediatrics and rare diseases. The first two interviews are available now!

COLLABORATIVE RESEARCH
Last updates on our collaborative projects

RARE DISEASES TRAINING FOR PAEDIATRIC PATIENTS – BARCELONA, 21-23 JUNE 2023

A paediatric expert patient training workshop, organised by the TEDDY European Network of Excellence for Paediatric Research, in collaboration with EURORDIS and the Sant Joan de Déu Research Foundation, within the European Joint Programme on Rare Diseases - EJP RD, will take place in Barcelona from 21 to 23 June 2023. This course aims at making young patients ready to actively participate in scientific research and to play an important role in improving research and healthcare services for all patients by contributing with their own specific experience. Register by 31 May 2023.

PUBLICATIONS
Our recent publications and other editorial activities

NEW ARTICLE ON OFF LABEL USE OF PAEDIATRIC MEDICINES

The article “Off-label is not always off-evidence: authorising paediatric indications for old medicines” has been just published in The Lancet – Child & Adolescent Health! Lucia Ruggieri from Fondazione Gianni Benzi, cooperated, as co-author, in this relevant work. The manuscript deals with off-label use of paediatric medicines and particularly old medicines. Approximately half of all paediatric medicine prescriptions in Europe are off-label. This means that they are administered for an unapproved indication or in an unapproved age group, dosage, or route of administration.

FGB AS CO-EDITOR ON FRONTIERS IN MEDICINE - REGULATORY SCIENCE JOURNAL

Viviana Giannuzzi, head of the research department at Fondazione Gianni Benzi, contributed as co-editor to the research topic “Reviews in: Regulatory Science” on the journal Frontiers in Medicine – Regulatory Science. The editorial article has just been published with relevant world-wide highlights on regulatory science.

ACROSS THE WORLD

Updates and latest news across the world

EMA REGULATORY STRATEGY TO 2025: MID-TERM REPORT

The European Medicines Agency has published a report focused on the mid-term achievements of its Regulatory Science Strategy to 2025. Progress was made in several areas, such as innovation in clinical trials, use of high-quality, real-world data in decision making as well as patient relevance in evidence generation.

TIFLIX: A COMPREHENSIVE, RELIABLE AND ENGAGING PLATFORM ON THALASSAEMIA AND SICKLE CELL DISEASE

The platform for healthcare professionals and patients with thalassaemia and sickle cell disease, called TIFLIX (Thalassaemia International Federation LIbrary eXtended) is now available! This brand-new video library powered by the Thalassaemia International Federation - TIF gathers a wealth of audio-visual educational contents on Thalassaemia and Sickle Cell Disease, presented by top-notch haematologists and other experts in the field.

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