• 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.

In particular, CRAs and PIs highlighted the strengths and weaknesses encountered in the study conduction with particular reference to the following issues:

• Informed consent procedure

• Planning study visits

• IMPs handling

• Dose adjustments for Safety and Efficacy reasons

• Periodic assessments for Safety

• Data entry in e-CRF

• Collection of Ferritin Samples for centralized evaluations and MRI Scans

• Study documentation

• Pharmacovigilance

• Monitoring activities.

The gathered information have provided updated information on all the centres involved in the study and are essential to identify the main issues to be solved to increase the effectiveness of the study and to ensure patients’ safety and high quality of collected data.

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+).

The 29th February, 2016 marked the ninth international Rare Disease Day coordinated by EURORDIS . The main objective of Rare Disease Day was to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives. The campaign targeted primarily the general public and also sought to raise awareness amongst policy makers, public authorities, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases.

Since Rare Disease Day was first launched by EURORDIS and its Council of National Alliances in 2008, thousands of events have taken place throughout the world reaching hundreds of thousands of people and resulting in a great deal of media coverage. The political momentum resulting from Rare Disease Day also serves advocacy purposes. It has notably contributed to the advancement of national plans and policies for rare diseases in a number of countries. Even though the campaign started as a European event, it has progressively become a world phenomenon, with the USA joining in 2009, and with the participation of a record-breaking 85 countries and regions around the world in 2015.

The theme of Rare Disease Day 2016 “Patient Voice” – paying tribute to the millions of people whose lives are affected by a rare disease and also to their families, friends and loved ones, who stand by them and support them every step of the way – recognized the crucial role that patients play in voicing their needs and in instigating change that improves their lives and the lives of their families and carers. A call to solidarity and to the acknowledgement of rare diseases as a real problem, not to be dismissed under the veil of indifference. In particular, the Rare Disease Day 2016 slogan “Join us in making the voice of rare diseases heard” appealed to a wider audience, those that are not living with or directly affected by a rare disease, to join the rare disease community in making known the impact of rare diseases.
DEEP project welcomed this initiative and has been pleased to contribute to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives. Due to the fragmentation of knowledge, it can take years before a patient affected by rare diseases get diagnosed. DEEP considers the promotion and the coordination of Research extremely important for health care system, and particularly for Rare Diseases, since they represent the tools by which speed up the achievements in research and benefits for patients. This year Rare Disease Day encourages us to continue finding ways to work together to raise awareness on haemoglobinopathies and make the voice of patients affected by thalassaemia and other haemoglobin disorders heard to a wider audience and highlight their needs to equal access to quality health care.

DEEP joined this event by becoming a friend of Rare Disease Day, showing its support on its official website and social networks.

For more info about the rare disease day, please visit http://www.rarediseaseday.org/

The initiative is organised by the H. H. Sheikh Sultan Bin Khalifa Bin Zayed Al Nahyan Humanitarian & Scientific Foundation, a non-profit non-governmental organization dedicated to value substantive efforts and contributions that have been made towards improving quality of life of patients with Thalassemia & other Hemoglobinopathies, by Individuals & Institutions, at National, Regional or International level.

This 2015 edition has been established by H. H. Sheikh Sultan Bin Khalifa Al Nahyan Humanitarian & Scientific Foundation in collaboration with ENERCA partner, Thalassemia International Federation (TIF), and included three categories: International Awards, Arab Awards and National Awards.

The Higher Scientific Committee Members for the International Awards was composed by Dr. Androulla Eleftheriou, Head of Committee, Executive Director of Thalassaemia International Federation (Cyprus), together with Professor Dimitris Loukopoulos (Greece), Professor Suthat Fuscharoen (Thailand), Professor John Porter (UK) and Professor Maria D. Capellini (Italy).

Prof. Amal el-Beshlawy, Professor of Pediatric Hematology at Cairo University (Egypt) and President of the Egyptian Thalassemia Association won the Best Published Research award in the 2015 Arab Awards Category.

The DEEP Consortium is proud of this award received by one of the project partner and congratulated her for the high level of her research and the recognized excellence in the field of Thalassemia.

The DEEP project supports this initiative and will strengthen the collaboration with the Sheikh Sultan bin Khalifa Al Nahyan Humanitarian and Science Foundation, linked by the common interest in employing scientific research to improve the quality of the lives of patients and their families.

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.