• Challenges and opportunities of conducting clinical trials in paediatrics

• Management of thalassaemia and haemoglobinopathies: an ongoing challenge

The meeting was aimed to raise the attention on the progresses achieved by the clinical research and the challenges to be faced in conducting clinical trials in paediatrics, also highlighting the importance of the international cooperation through European Networks and the global initiatives to create synergies and foster clinical research. The second session was more focused on the new pathways and therapeutic strategies for the management of thalassaemia and haemoglobinopathies and was the occasion to talk about the goals of the DEEP project.

The abstract of the first session are available at the dedicated page.

The abstract of the second session are available at the dedicated page.

Here you can also download the DEEP Open Meeting brochure, which can be browsed even through mobile devices.

The meeting provided an update on the achievements and difficulties encountered toward the implementations of specific project tasks, the reasons for some delays and the mitigation strategies set into place in order to overcome such obstacles.

The meeting opened with a comprehensive overview of the activities performed and the most important achievements carried out, with particular reference to the implementation of the DEEP studies in compliance with the Paediatric Investigation Plan – PIP (EMEA-001126-PIP01-10), as approved and further amended by the Paediatric Committee (PDCO). In particular, the main efforts carried out to implement DEEP-2 recruitment and study conduct were highlighted, that have led to an increase of the number of clinical centres, the check of the recruitment capacity as well as the organisation of study qualification visits.

Moreover, an update of the clinical studies has been provided, with a particular focus on:

• the difficulties to conduct the DEEP-2 trial that has to face a default level of complexity, due to regulatory, methodological, ethical and administrative issues;

• the status of DEEP-3 Study and its updated results;

• the updated overview of the pharmaco-economic study.

Among others, the financial resources reallocation according to the recruitment performance at any involved centre have been discussed during the General Assembly meeting.

The second meeting day provided an update on DEEP-2 study protocol and procedures and was characterised by the organisation of the DEEP-2 Investigator Meeting, held as a guided roundtable where CRAs and Principal Investigators from main recruiting sites have presented and discussed the strengths and weaknesses of their own center.

In the same context of the GA meeting, the 4th Scientific Open Meeting took place on April 15th, entailing two sessions:

-Challenges and opportunities of conducting clinical trials in paediatrics

-Management of thalassaemia and haemoglobinopathies: an ongoing challenge

The meeting represented an excellent opportunity to foster fruitful scientific debates and exchange information. Advancement in the work packages and partner involvements were discussed in order to build a perfect working basis for the forthcoming activities.

All the presentations of the 7^th General Assembly meeting can be downloaded from the “Presentations” dedicated page.

All the abstracts of the DEEP Open Meeting can be found in the dedicated brochure, which can be browsed even through mobile devices.

This year the Scientific Open Meeting will  take its cue from the advancement in paediatric clinical research to address patients needs and will entail the following two thematic sessions:

 –          Challenges And Opportunities Of Conducting Clinical Trials In Paediatrics

–          Management Of Thalassaemia And Haemoglobinopathies: An Ongoing Challenge

The Open Meeting will be the occasion to raise the attention on the progresses achieved by the clinical research and the challenges to be faced in conducting clinical trials in paediatrics, also highlighting the importance of the international cooperation through European Networks and the global initiatives to create synergies and foster clinical research. The second session will be more focused on the new pathways and therapeutic strategies for the management of thalassaemia and haemoglobinopathies and will also be the occasion to talk about the goals of the DEEP project.

In particular, the Open Meeting will be the occasion to discuss these topics and challenges by different stakeholders points of views.

More details on the event and the final agenda are available here and through our social network channels (Facebook and Google+).

After the sad decision to cancel the General Assembly Meeting in Tunis, due to the disorders which have occurred across the country, the 7th DEEP General Assembly (GA) will take place in Padua (Italy) on April 13th -15th, 2016 with the participation of the whole DEEP Consortium as well as of external experts.

Here you can find the final agenda of the Meeting, which can be browsed even through mobile devices.

The meeting will be focused on a general update of the project’s activities, the progresses and achievements of the DEEP project as well as the finalization of the DEEP-2 and DEEP-3 studies, that are of utmost priority. Among others, the financial resources reallocation according to the recruitment performance at any involved centre will be discussed during the meeting.

In the same context of the GA meeting, the DEEP-2 Investigator Meeting and the 4th Open Meeting will take place.

In particular, the Scientific Open Meeting on April 15th, 2016 will entail two sessions:

   –  Challenges And Opportunities Of Conducting Clinical Trials In Paediatrics

   –  Management Of Thalassaemia And Haemoglobinopathies: An Ongoing Challenge

The Scientific Open Meeting will be the occasion to raise the attention on the progresses achieved by the clinical research and the challenges to be faced in conducting clinical trials in paediatrics, also highlighting the importance of the international cooperation through European Networks and the global initiatives to create synergies and foster clinical research. The second session will be more focused on the new pathways and therapeutic strategies for the management of thalassaemia and haemoglobinopathies and will also be the occasion to talk about the goals of the DEEP project.

More details on the event and the final agenda are available here and through our social network channels (Facebook and Google+).

Moreover, Dr. Giannuzzi highlighted how this lack of harmonisation makes more difficult the approach in case of studies involving non-EU Countries, as DEEP, and especially ‘small populations’, such as children and patients affected by rare diseases, which typically are multi-centre and multi-national.

More details on the event and the list of all submitted abstracts are available here.

Donato Bonifazi, DEEP Project Manager presented the project in the context of the international paediatric trials that have to face a default level of complexity, due to regulatory, methodological, ethical and administrative issues, especially when they are registrative and international as DEEP is.

In particular, the specific challenges that DEEP has addressed and is still facing with reference to the recruitment and patient’s retention have been described, since DEEP is a ‘Registrative’ CTs with economic burden, ethical stringent provisions and GCP-ICHE11 obligations that involves not only a paediatric multi-ethnic population with different cultures, laws and children of different ages but also a rare and disperse population involving different Rare Congenital Anaemia.

Moreover, the DEEP multisteps strategy to address these issues has been presented, resulting from the following key aspects:

• the organisation of a ‘trials management plan and infrastructure’ including SOPs preparation, data management, drug management, pharmacovigilance, monitoring, etc;

• the implementation of a unique procedure and a unique CTA ‘package of documents’;

• the developments of a ‘patients tailored approach’ including children, families and association.

To conclude Donato Bonifazi updated on the patients’ recruitment that is currently proceeding successfully thanks to a continuous and coordinated activity of the TMT and the PIs and the continuous discussion of the Protocol-related issues among Principal Investigators, Trial Management Team and all the study key contacts that has led the inclusion of many new centres.

The event was also the occasion to discuss, in the framework of an international multi-stakeholders debate, the present and the future of the paediatric research including the efforts made by the scientific community and the ongoing projects such as the proposed Paediatric Research Infrastructures (EPCTRI) and the global networks.

The choice to select Tunis as venue for the next General Assembly comes from the willingness to raise awareness on the recruiting centres that are the backbone of the recruitment activities and to focus the attention on the Mediterranean region, in line with the Horizon 2020 strategy aimed at strengthening the cooperation with the Mediterranean area through the promotion of joint research projects in the health field in order to promote the development and the Mediterranean integrity itself.

The meeting will be the occasion to talk about the goals and the progress of the project and its contribution to improving of the clinical management of children with transfusion-dependent thalaessemia and other hemoglobinopathies who need chelation therapy.

Tunis will also be the venue to encouraging the project Partners to strengthen the efforts towards recruiting increasing number of patients in the DEEP-2 and DEEP-3 studies in order to ensure finalization of these clinical studies which are very important for the overall success of the DEEP project.

More details on the event and the related agenda will be soon available through our social network channels (Facebook and Google+).

The interviews offer an interesting picture of what the clinical research in Albania has achieved so far and what its legacy will be, in particular in light of a more tight international collaboration within a European research Network, as the DEEP project is.

Massimo Gaiani, Italian ambassador to Albania, opened the interview pointing out the importance of developing strong ties and networks as well as fostering the international cooperation to tackle the competition both at EU and global level.

At this YouTube channel, you can download Massimo Gaiani interview.

Amal El Beshlawy, Professor of Paediatric Hematology at Cairo University (Egypt) and President of the Egyptian Thalassemia Association talked about the cooperation between the EU and the Mediterranean countries, emphasizing the great results achieved in iron chelation therapy using deferiprone.

At this YouTube channel, you can download Amal El Beshlawy interview.

Vitangelo Dattoli, General Director of University Hospital Polyclinic of Bari (Italy), highlighted the success of the collaborative efforts in medicine and research between the two University Hospital Centres: “Mother Teresa” (UHCT) and Polyclinic of Bari.

At this YouTube channel, you can download Vitangelo Dattoli interview.

Etleva Refatllari from University Hospital Center “Mother Teresa” of Tirana (Albania) shared her experience in applying Good Laboratory Standards in a context characterized by the absence of any comprehensive guidance from regulatory and monitoring authorities.

At this YouTube channel, you can download Etleva Refatllari interview.

Gjeorgjina Kuli-Lito, Professor of Paediatrics at the University of Medicine, University Hospital Center “Mother Teresa” (UHCT) presented the critical issues in developing pediatric trials and the main points to be considered to ensure that a clinical trial could be successful.

At this YouTube channel, you can download Gjeorgjina Kuli-Lito interview.

Anila Godo, Professor of Paediatrics and Head of Onco-Haematology Service at the University Hospital Center “Mother Teresa” (UHCT), talked about the solid partnership between Albania and the DEEP consortium, focusing on the positive impact that the project brought to the Albanian pediatric research and on the importance of unifying diagnostic, preventive and therapeutic strategies at international level.

At this YouTube channel, you can download Anila Godo interview.

The Scientific Conference was officially opened by the Chief Executive Officer of CVBF Albania, Donato Bonifazi, the Mayor, Fatos Tushe and the General Secretary of the “Associazione TALASSEMICI”, Mura Zenelaj.

The event was promoted by Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF), Branch of Albania in collaboration with Università Cattolica “Nostra Signora del Buon Consiglio” of Tirana, a private university whose academic activities are implemented in collaboration with important Italian Universities (University of Bari “Aldo Moro”, University of Rome “Tor Vergata”, University of Milan, University of Bologna, University of Palermo, Catholic University of Milan).

On that occasion, the poster of the DEEP project was presented.

More details on the event and the related agenda are available here.

To download the presentations of the scientific conference:

– D. Bonifazi, Consorzio per Valutazioni Biologiche e Farmacologiche

– A. Zaka, Dita nderkombetare e luftes kunder Talasemise (in Albanian)

– A. Ceci, M. Kreka, Nuove terapie farmacologiche nella talassemia

– G. Adorno, La terapia trasfusionale nella talassemia

– F. Brancati, La genetica della talassemia

– N. Capodicasa, La donazione volontaria del sangue

DEEP-2 is, at today, the first paediatric clinical trial conducted in Albania; for this reason Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) in collaboration with University Hospital Center “Mother Teresa” (UHCT) has selected the city of Tirana as venue for the 3^rd edition of the DEEP Open Meeting.

The event represented the occasion to raise the attention on the progresses achieved by the clinical research in Albania and the importance of the European Networks to create synergies to spread excellence and attract European funding. The meeting was accredited by the Albanian National Centre of Continuing Education, QKEV (providing with 5 CME credits of Continuing Education for healthcare professionals) and was structured into 2 thematic sessions focused on the Clinical Research in Albania and Networks and Projects in a Global Context, respectively. All sessions were carried out in English, with simultaneous translation English/Albanian available.

The meeting was opened by Anila Godo, Professor of Paediatrics at University Hospital Center “Mother Teresa” and Head of Onco-Haematology Service (Tirana), Donato Bonifazi, CEO of Consorzio per Biologiche Valutazioni e Farmacologiche and Ogerta Manastirliu, General Director of University Hospital Center “Mother Teresa” of Tirana.

The main contents presented and discussed during the Open Meeting are summarized below.

SESSION I – Clinical Research in Albania

The first session of the Open Meeting represented an opportunity for both European and Albanian key opinion leaders and experts to share their experiences in the field of paediatric clinical research. This session was, indeed, an occasion to discuss about many topics related to the conduction of paediatric clinical trials such as the barriers that researchers encounter to the timely development of safe and efficacious medicines especially for children, the related need to set up infrastructures for each study in each site, the collaborative efforts in medicine and research.
In particular, it was stressed the importance of the international cooperation in the activities performed in clinical research as a way to encourage interdisciplinary participation in developing solutions through high-impact, mission-driven collaborations with leading health providers, governments and educational institutions. In this way, the identification of the essential profiles involved in the concrete conduction of clinical research will be supported by the sharing of an high-quality and effective know-how, starting from the basis up to high specialisations proficiency.

The theme of clinical research was also addressed in light of the recent release of the Albanian Law on Drugs and Pharmaceutical services (31st July, 2014), and with a particular focus on the role of Albanian Universities in supporting clinical research as well as the achievements and challenges of the Continuing Medical Education.

Among the various Albanian personalities who attended the meeting, Prof. G. Kuli-Lito on behalf of the University Hospital Center “Mother Teresa”; the General Director of Health Policy and Planning, G. Bejtja, on behalf of the Albanian Ministry of Health and E. Shehu, Director of National Center of Continuing Education of Tirana.

SESSION II – Networks and Projects in a Global Context

The second session of the DEEP Open Meeting was focused on the importance of the Networks and Projects in a global context and on their impact on clinical research in paediatrics.

Among the several initiatives described at European and global level to promote paediatric research and reduce the extent of the off-label use, a special focus was dedicated to the experience of the University Hospital Center “Mother Teresa” (UHCT) in two EU-funded projects, in which it is involved as partner: the DEEP (DEferiprone Evaluation in Paediatrics) project and in particular the DEEP-2 Study and the GAPP (GAbapentin in Paediatric Pain) project. The international activities promoted in the clinical research field and in collaboration with an international research consortium will, indeed, provide support for aligning Albanian standards and practices with those present in the European Union for establishing working procedures to make innovation feasible.

An important example of European collaboration was also provided by the proposal of a new European Research Infrastructure for Paediatric Clinical Trials (EPCT-RI), as a new instrument identified in the European Research Agenda with reference to facilities, resources and related services used by the scientific community to conduct top-level research in their respective field.

Here you can download all the presentations of the Open Meeting.

More details on the event and the related agenda are available here.