Fondazione Gianni Benzi Onlus, at the initiative ofAssociazione Bottega del Sorrisoand IRCCS Istituto Tumori "Giovanni Paolo II” Bari, organised the meeting “I tumori rari: sfide ed opportunità”, held on 23 February 2023 at Istituto Tumori Bari. The meeting, focused on patients with rare tumours, including paediatric patients, underlined the need to establish a network for patients and their needs by applying a multispecialistic approach for a more effective and prompt diagnosis and treatment. Organising the healthcare assistance according to this model might ensure the access to treatment in Apulia region to patients, reducing the so-called health tourism.
PUBLICATIONS Our recent publications and other editorial activities
MOVING TOWARDS A QUESTION-CENTRIC APPROACH FOR REGULATORY DECISION-MAKING IN THE CONTEXT OF DRUG ASSESSMENT
A new article was recently published: “Moving Toward a Question-Centric Approach for Regulatory Decision Making in the Context of Drug Assessment”. Fondazione Gianni Benzi cooperated in this work focused on the regulatory decision-making process aimed to put medicines on the market. The most intuitive question to establish the efficacy and safety of medicinal products for market access is the benefit/risk balance. However, there is currently no systematic approach in the regulatory setting to assess and establish the acceptability of alternative methods and data sources. This paper discusses the value of explicitly redefining and restructuring the regulatory scientific decision making around the scientific question to be addressed.
Within the EU funded project PedCRIN, an easy-to-use tool has been developed to guide investigators and sponsors in managing specimens and associated data in compliance with the applicable European rules in the context of paediatric clinical trials.
ACROSS THE WORLD Updates and latest news across the world
EMA HIGHLIGHTS 2022
The European Medicines Agency has published the Human medicines highlights of 2022. In 2022, EMA recommended 89 medicines for marketing authorisation. Most of them covers the oncology area followed by neurologic and endocrinologic diseases. Six are Advanced Therapy Medicinal Products and 21 were designated as orphan medical products. Ninety extensions of indication were recommended in 2022, including 37 for paediatric use. Five medicines were authorised under exceptional circumstances. All of them are intended for children. This year, 8 PRIME-designated medicines were recommended for approval, including 3 addressing paediatric diseases.
TheInterdisciplinary Scientific Committee(ISC) is a multi-stakeholder, multi-disciplinary group of experts in rare diseases medical research with a strong expertise in data sharing, registries, biobanks and natural history studies. ISC has one opening for a new member. Interested candidates are invited to send their resume, biosketch and letter of motivation to scisec-irdirc@ejprarediseases.org before the 2nd of April, 2023.
The European Medicines Agency recently launched a pilot to support the translation of basic research developments into medicines that could make a difference in patients’ lives. The pilot is open to academic sponsors and non-profit organisations who are developing advanced therapy medicinal products (ATMPs). They are a major contributor to the development of ATMPs and diagnostic and delivery devices, but experience has shown that navigating regulatory requirements can be challenging.
The European Medicines Agency offers consultations in parallel with the European Network for Health Technology Assessment (EUnetHTA) 21 consortium. This aims to allow medicine developers to obtain feedback from regulators and health technology assessment bodies in Europe on their evidence-generation plans to support decision-making on marketing authorisation and reimbursement of new medicines at the same time.
The objective is to generate robust evidence that satisfies the needs of decision-makers.
To this end, a guidance was published to describe the process, timelines and actions for each party undertaking a parallel EMA/EUnetHTA 21 Joint Scientific Consultation.
We are looking for new people to be included in our staff to collaborate to research activities in the following areas:
- Research activities within collaborative European/national projects - Registries, observational studies and secondary use of data - Support and coordination of scientific groups within paediatric/rare disease Networks and Research infrastructures in the biomedical field
- Bioinformatics and biostatics - Bibliographic search and systematic reviews
- Data extraction from national and international databases, data analysis and related reports
- Preparation of applications for funding in response to European/national calls for proposals
