mariangela

About mariangela

This author has not yet filled in any details.
So far mariangela has created 56 blog entries.

An innovative approach for the patient involvement in the paediatric clinical research: the role of YPAGs

Over the last years, a Europe-wide trend towards a patient-focused approach and the involvement of patients is developing and is influencing the decision-making process related to the clinical research. Patients involvement in paediatric clinical trials is recognized as an important added value at European and international level and an essential contribution to paediatric clinical research. To foster patients’ empowerment, advocacy groups including young patients and healthy youngers, named YPAG (Young Persons Advisory Group), have been established from New Zealand to France, from Great Britain to the United States, with the aim to provide new perspectives and attitudes in the designing and conduction of paediatric clinical trials. Currently, 21 YPAGs exist across the world and work together within the iCAN (International Children’s Advisory Network) consortium to provide a voice for children and families in health, medicine, research, and innovation with a global impact. At European level, eYPAGnet, the European YPAG’s network (member of Enpr-EMA) has been established and is coordinated by the Hospital Sant Joan de Déu (Barcelona) with the mission to improve the capacity of collaboration with the different actors who participate in the research and development process of innovative drugs. YPAGs aim to help the professionals involved in the clinical trials to overcome some though issues of the clinical research. For example, they could help to open and complete on time the trials, or could improve the recruitment of patients to agreed target and the retention of patients to completion. In general, through the YPAGs, the clinicians involved in the trials could meet the needs of the patients, designing the study according to their necessities. In this context, Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) in collaboration with the TEDDY Network and the paediatric [...]

2017-12-24T12:53:58+00:00 24/12/2017|Categories: News & Events|

ICH-GOOD CLINICAL PRACTICE (GCP) TRAINING COURSE

The e-learning “ICH-Good Clinical Practice (GCP) Training Course”, promoted by Consorzio per Valutazioni Biologiche e Farmacologiche in the framework of the GAPP project, is available free of charge at the following link: www.gcptraining.cvbf.net. The course meets the Minimum Criteria for ICH GCP Investigator Site Personnel Training identified as necessary to enable mutual recognition of GCP training among trial sponsors as recognised by TransCelerate BioPharma. It is based on the international E6 ICH Good Clinical Practice (R2), revised in 2016, and is aimed at providing a guide for all individuals that are involved in clinical research and clinical trials. Attending the course, participants will become aware of their roles and responsibilities within a clinical trial and will understand how GCP can be applied practically in the research setting. TransCelerate BioPharma Inc. is a non-profit organization dedicated to improving the health of people around the world by accelerating and simplifying the research and development (R&D) of innovative new therapies. Consorzio per Valutazioni Biologiche e Farmacologiche is recognised as an accredited training provider at international level with the GAPP “ICH-Good Clinical Practice (GCP) Training Course”. More information on the course and registrations are available here.

2018-02-06T13:57:33+00:00 24/12/2017|Categories: News & Events|

The pilot study QuBo was presented at the 14th International Conference on Thalassaemia & Haemoglobinopathies and 16th TIF International Conference for Patients & Parents

From November 17th to 19th, 2017, the 14th International Conference on Thalassaemia & Haemoglobinopathies and the 16th TIF International Conference for Patients & Parents took place in Thessaloniki, Greece. In this occasion, the poster of the DEEP (DEferiprone Evaluation in Paediatrics) project discussing the DEEP-2 study informative materials and the results of the pilot study QuBo (the multicenter observational transversal study for patients involved in the DEEP-2 trial) has been presented. DEEP is a EU-funded project, developed under the TEDDY umbrella, involving EU and non-EU countries (Albania, Egypt, Tunisia), aimed to study deferiprone in children affected by congenital anaemias requiring chronic transfusion therapy and iron chelation, such as the β-thalassaemia major and the Sickle Cell Disease (SCD), and develop a new formulation suitable for children. To this aim, the project performed three multi-national paediatric studies involving more than 700 paediatric patients, 23 clinical sites and 16 partners. In the framework of the DEEP project, the QuBo study has been performed with the aim to investigate the quality, the comprehensibility and thelikeability of the informative booklets for patients involved in the DEEP-2 trial, an efficacy and safety trial to compare deferiprone versus deferasirox in 388 paediatric patients aged 1 month < 18 years from Mediterranean area countries. In particular, three informative booklets and two ad hoc assent forms to empower DEEP-2 minor patients were prepared for different ages in six languages thanks to a collaborative effort involving pharmacologists, paediatricians, child psychologists and illustrators, in an easy-to-manage format, a charming graphic including picture, a simple vocabulary and sentences structures. Two age-tailored questionnaires were developed in the QuBo study and distributed to the patients involved in the trial. The QuBo pilot study has been carried out in Albania [...]

2018-02-06T13:59:40+00:00 24/12/2017|Categories: News & Events|

EMA: Brexit update and adoption of work programme for 2018

The last press release published by EMA (European Medicines Agency) on December 15th, 2017, reports some important highlights from the Management Board meeting about Brexit preparation and the adoption of the work programme for 2018. EMA now has just over 15 months to prepare for the move and take up its new seat in Amsterdam by 30 March 2019 at the latest. A delegation from the Dutch government attended part of the Management Board meeting. The Dutch government has identified a temporary premise to EMA from 1 January 2019 for an interim period until the tailor-made Vivaldi building will be ready. Moreover, the Board adopted the work programme for 2018 and the budget for 2018-2019. EMA’s preparations for the UK’s withdrawal from the EU will impact on the activities of the Agency. In the discussion about the Agency’s work programme, it was highlighted that EMA will be able to maintain its core activities although EMA will have to temporarily reduce or suspend activities in other areas. Moreover, the press release states that the Agency is working hard in collaboration with Member States, stakeholders and the developers to deliver a functional EU clinical trial portal and database to support the needs of EU clinical research, and to have the Clinical Trial Regulation enter in operation as soon as practicable. More information are available here.

2017-12-24T12:15:25+00:00 24/12/2017|Categories: News & Events|

Launch of the Parliamentary Advocates for Rare Diseases

On October 17th, 2017, the Parliamentary Advocates for Rare Diseases, a network of European and national members of Parliament advocating to improve the lives of people living with a rare disease, was launched by EURORDIS, the European Organization for Rare Diseases, in occasion of the event “Juggling Care and Daily Life: the Balancing Act of the Rare Diseases Community”. This launch event was held at the European Parliament in Brussels and included the presentation of the results of the first European survey on the impact of rare diseases on daily life. This survey was carried out by EURORDIS through its Rare Barometer Voices, a community of 30 million people in Europe living with a rare disease and willing to participate in EURORDIS Rare Diseases surveys and studies. In particular, the results of the survey outlined the following aspects: - More than 70% of patients have difficulties with daily activities and tasks such as preparing meals and handling household chores, with motor and sensorial functioning such as visual, hearing and body positioning issues, and with social life as maintaining relations with others. - 30% of carers spend over 6 hours a day on disease-related tasks with over 60% of these carers being women. - Having to reduce or stop professional activity due to illness occurs to 70% of rare disease patients and carers. - Feelings of depression and unhappiness are three times more common amongst rare disease patients and carers compared with the general population. The network of Parliamentary Advocates for Rare Diseases intends to tackle these challenges by fostering cross-border EU collaboration. The network is made up of MEPs (Member of European Parliament) and National MPs who have long supported the cause of rare diseases [...]

2017-12-24T12:47:05+00:00 24/12/2017|Categories: News & Events|

THE 4TH EDITION OF RE(ACT) CONGRESS

From 7th to 10th March 2018, the International Congress on Research of Rare and Orphan Diseases will take place at the Istituto Ortopedico Rizzoli in Bologna, Italy and will bring together world leaders and young scientist, who will share and discuss the results of their studies and cutting-edge research. It will be an occasion to exchange ideas and stimulate learning, networking and interdisciplinary collaboration. Moreover, scientists from different disciplines, such as stem cell researchers, geneticists, biochemists, clinicians and pharmacists will have the possibility to exchange information with patient organizations. The 4th edition is co-organized in collaboration with E-RARE ERA-NET for research programs in rare diseases.. For further information and for registrations click here.

2017-12-24T12:07:46+00:00 24/12/2017|Categories: News & Events|

EU H2020 “SPASD – Strengthening the Capacity of Professionals Working in Schools with Children and Young People with ASD” Project Kick-off Meeting

Bucharest, November 30th, 2017. The new European project SPASD - Strengthening the Capacity of Professionals Working in Schools with Children and Young People with Autism Spectrum Disorder (ASD) - was officially kicked off in Bucharest, Romania, on November 30th, 2017. The project, which is funded under the H2020 Program (207-1-RO01-KA201-037459, Erasmus Plus – Strategic partnership program 2017) will last 24 months, and is led by Romanian Angel Appeal Foundation (RAA), Romania gathering a consortium of 4 partners beyond the coordinator: Universitair Medisch Centrum Groningen/Academisch Ziekenhuis (UMCG), The Netherlands, Consorzio per Valutazioni Biologoche e Farmacologiche (CVBF), Italy; Universitatea Babes Bolyai (UBB), Romania; Fundacja Synapsis, Polonia. SPASD project targets primary, secondary and high school teachers from mainstream schools as being the main actors able to combat early school dropout and to increase school integration of children with Autism spectrum disorder (ASD). The project arose within the EU and national policies promoting equal possibilities in accessing education for all children and the integration of children with disabilities into the general education system providing them with individual and appropriate support. ASD is no longer considered a rare condition and we know now that levels of cognitive functioning of individuals with ASD cover the whole range, from intellectual disability to high functioning. Nevertheless, all the four partner countries (Romania, Poland, Italy, the Netherlands) report difficulties in managing children with ASD in a regular classroom due to teachers’ low competencies in working with ASD students; limited school resources to train staff, provide teaching adaptation tools and educational support services; discrimination. To overcome these difficulties in the view of a special and inclusive education, the project proposes a set of activities that respond to the following objectives: 1. Strengthen the knowledge and [...]

2017-12-24T12:44:34+00:00 24/12/2017|Categories: News & Events|

Save the date – ID-EPTRI Kick-off-meeting, January 15th-16th, 2018, Rome

The recently EU-funded project ID-EPTRI (European Paediatric Translational Research Infrastructure), aimed to design a new Research Infrastructure (RI) in Europe completely dedicated to Paediatrics will be launched on January 15th-16th 2018, at the Ministry of Education, Universities and Research, Sala della Comunicazione, Viale Trastevere, 76/a, Rome, Italy. The project, which is financed under the H2020-INFRADEV-01-2017 programme and will last 24 months at a total cost of 3 million Euro, is led by Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF). EPTRI is a new complementary RI acting as a paediatric common service in the context of the existing RIs intended to link all the available competences and technologies useful to enhance research in paediatric medicines from drug discovery and early development phases to be translated into clinical phases and medicines uses. The ID-EPTRI project aims to design the framework for the new RI providing a Conceptual Design Report (CDR) describing the scientific and technical requirements as well as the key components of the European Paediatric Translational Research Infrastructure. Five technical and scientific domains have been identified within EPTRI structure: 1-Paediatric Medicines Discovery, 2-Biomarkers, 3-Paediatric Pharmacology, 4-Formulation Science, 5-Underpinning Paediatric Studies. The kick-off Meeting will gather a consortium of 26 partners, from 19 EU and non-EU countries, to launch the project activities. In particular, it will be aimed to give all participants a better understanding of the tasks and goals of the project and to illustrate the key elements of the new research infrastructure. The first day will be focused on the global scenario around EPTRI and the role of RIs to strengthening research outcomes to improve patients’ health. The second day will be focused on the five thematic platforms foreseen, the project management and coordination and [...]

2018-01-31T15:34:30+00:00 24/12/2017|Categories: News & Events|

Save the date – TEDDY General Assembly, January 14th, 2018, Rome

TEDDY – European Network of Excellence for Paediatric Clinical Research announces the General Assembly entitled “A new phase for paediatric clinical research: a play role for TEDDY?”, that will be held on January 14th, 2018 at Hotel Ripa in Via degli Orti di Trastevere, 3 Rome – Italy. At the end of the 5-years funding period, the TEDDY Network was further renewed continuing to provide its contribution on a voluntary basis with valuable and internationally recognised efforts offered by its members’ resources, with many partners collaborating on a case by case basis. To consolidate the work carried out up to date as well as to expand the future perspective of the Network, its Boards have taken the initiative to provide TEDDY with an autonomous legal status to valorise the results and continue the activities of TEDDY also in its role of category 1 Network Member of Enpr-EMA (the European Network of Paediatric Research at the European Medicines Agency). A new Statute has also been prepared, allowing the adhesion of natural and legal persons within two categories of members: Ordinaries (they are the natural persons who contribute individually) or Supporters (they are the legal entities that support the activities of the network).   Moreover, some operating groups have been improved and/or created: • Standard Operating Procedures for the conduct of paediatric clinical trials; • Ethic Issues and interactions with Ethic Committees; • Off-label medicines use in paediatrics; • Regulatory procedures for paediatric clinical trials; • Inventory of clinical sites and facilities for clinical trial feasibility; • Pharmacovigilance; • Active engagement of children and adolescents in the themes of clinical research; • Advanced therapies in paediatrics. Join the network to give your contribution by filling in the [...]

2018-01-31T14:59:05+00:00 24/12/2017|Categories: News & Events|

ID-EPTRI approval: a great success for paediatric medicines development

We are very pleased to announce that the ID-EPTRI (European Paediatric Translational Research Infrastructure) project, coordinated by CVBF and submitted within the INFRADEV-2016-2017 single-stage call for proposals, aimed to create the framework for a new paediatric Research Infrastructure (RI)  has been successfully evaluated and granted 3.00 million Euro in funding from the European Commission. […]

2018-02-21T11:18:26+00:00 01/09/2017|Categories: News & Events|