WHAT'S NEW Last updates on our collaborative research projects
INHERENT AT THE ASH ANNUAL MEETING
The 63rdASH Annual Meetingwas held as a hybrid meeting at the Georgia World Congress Center in Atlanta from December 11-14, 2021. A poster was presented during the meeting on the International Hemoglobinopathy Research Network (INHERENT). Fondazione Gianni Benzi is actively involved in the network activities, particularly in the ethics working group that aims to guide research activities to be conducted within the network ensuring the compliance with the applicable international and national ethical and legal rules.
ACROSS THE WORLD Updates and latest news across the world
CTIS GOES LIVE
The Clinical Trials Information System (CTIS) has gone live on January 31st, 2022. CTIS is the backbone of the Clinical Trials Regulation that will harmonise the assessment and supervision of clinical trials in the European Union (EU). To access CTIS, sponsors can visit the public Clinical Trials website athttps://euclinicaltrials.eu/home.The “CTIS for sponsors” page provides key information and links for sponsor users. Patients, healthcare professionals and the general public can also access the public Clinical Trials website.
If you are interested, you can submit your application to the Scientific Secretariat (scisec-irdirc [at] ejprarediseases.org)
NEW INITIATIVE LAUNCHED: ACCELERATING CLINICAL TRIALS IN THE EU
The European Commission, the Heads of Medicines Agencies and the European Medicines Agency have recently launched the initiative “Accelerating Clinical Trials in the European Union (ACT EU)” to transform how clinical trials are initiated, designed and run. Building on the application of the Clinical Trials Regulation and the launch of the Clinical Trials Information System on 31 January 2022, ACT EU will strengthen the European environment forclinical trials, whilst maintaining the high-level of protection of trial participants, data robustness and transparency.
The Committee for Medicinal Products for Human Use (CHMP) at theEuropean Medicines Agency (EMA)adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide. Voxelotor is a small molecule which attaches and stabilises haemoglobin, preventing haemoglobin polymerization that causes sickling and destruction of red blood cells in SCD. This situation leads to episodes of vaso-occlusive crisis and haemolytic anaemia.
PUBLICATIONS Our recent publications and other editorial activities
FRONTIERS RESEARCH TOPICS: THE USE OF RWD FOR REGULATORY PURPOSES IN THE RARE DISEASES
Viviana Giannuzzi from Fondazione Benzi is the co-editor of the new Frontiers Research Topic dedicated to Real World Data (RWD) generated for regulatory purposes in the rare diseases setting. Experts such as scientists, economists and experts in social sciences are invited to propose their original research articles, reviews and meta-analyses describing the use of RWD in the orphan medicines Research & Development (R&D) and picture the possible economic, social and political impact. You can submit your abstract by 5 February 2022 and the manuscript by 6 April 2022.
A GUIDE FOR INFORMED CONSENT AND ASSENT FOR CHILDREN
Viviana Giannuzzi, from Fondazione Benzi, cooperated in the article “Informed consent and assent guide for paediatric clinical trials in Europe” recently published. This work collates all the current legal, regulatory and ethical documentation on the informed consent (IC) process. There are still many national legal differences in the requirements for consent and assent documents. Therefore, the aim of this new guide was to provide more practical easy-to-use tool for designing the content of IC and assent documents to enhance the high-quality ethical standards of paediatric clinical trials.
NEW ARTICLE PUBLISHED: MEDICAL DEVICES TAILORED FOR CHILDREN
The article “Medical Device Development for Children and Young People—Reviewing the Challenges and Opportunities” has just been published on Pharmaceutics. Fedele Bonifazi, president of Fondazione Benzi, cooperated in this review aimed to investigate the complexities in the development of Medical Devices (MDs) for children as well as the market need and the regulatory framework related to paediatric MDs. Authors point out that to ensure the targeted development of paediatric MDs, collaboration across multiple professional disciplines is required, facilitated by a platform to foster collaboration and drive innovation.
CALLS FOR FUNDING Funding opportunities within relevant research programmes
EJP RD – JOINT TRANSNATIONAL CALL 2022
EJP RD launched on December 14th thefourth EJP RD Joint Transnational Call (JTC) 2022, aimed at funding multilateral research projects on rare diseases under the EJP-COFUND action. The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project, with the call topic being “Development of new analytic tools and pathways to accelerate diagnosis and facilitate diagnostic monitoring of rare diseases.”
Several calls for funding have been recently opened within Horizon Europe, the European funding programme for research and innovation with abudget of €95.5 billion.We encourage our partners to apply to the 2022 calls especially those under theCluster 1 - Health of the Pillar 2 - Global Challenges and European Industrial Competitiveness.
