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Newsletter no. 14 - January 2022

WHAT'S NEW
Last updates on our collaborative research projects

INHERENT AT THE ASH ANNUAL MEETING

The 63rd ASH Annual Meeting was 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.   

Discover more - Read the abstract

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 at https://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.

Discover more - Visit the Clinical Trials website

IRDiRC TASK FORCES: CALL FOR MEMBERS

The Therapies Scientific Committee (TSC) of the International Rare Diseases Research Consortium (IRDiRC) is establishing two Task Forces:
- Task Force on Drug Repurposing Guidebook aimed at helping developers identify specific tools and practices of relevance for repurposing projects
- Task Force on Disregarded Rare Diseases to characterize specific commonalities amongst a large group of “disregarded” rare diseases
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 for clinical trials, whilst maintaining the high-level of protection of trial participants, data robustness and transparency

Discover more - Read the ACT EU strategy paper

OXBRYTA RECEIVED CHMP POSITIVE OPINION FOR SCD

The Committee for Medicinal Products for Human Use (CHMP) at the European 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.

Discover more 

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.

Discover more - Read the article

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 the fourth 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.”

Discover more - Visit the EJP RD website

HORIZON EUROPE 2022 CALLS NOW OPEN

Several calls for funding have been recently opened within Horizon Europe, the European funding programme for research and innovation with a budget of €95.5 billion. We encourage our partners to apply to the 2022 calls especially those under the Cluster 1 - Health of the Pillar 2 - Global Challenges and European Industrial Competitiveness.

Deadline for submission is 21 April 2022.

 

 

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