Terapeutic area: Infectious deseases
The Penta ID Network brings together key stakeholders in paediatric infectious disease, including researchers, clinicians, regulatory bodies, the pharmaceutical industry and patients and their families, to plan and carry out high quality clinical research and training.
Terapeutic area: Rare Disease
EURORDIS (European Organisation for Rare Diseases Association) currently represents 676 rare disease patient organisations in 62 countries worldwide, of which 37 are European countries, including 27 EU Member States and is the voice of the estimated 30 million patients affected by rare diseases throughout Europe.
Terapeutic area: Cystic fibrosis
The ECFS (European Cystic Fibrosis Society) is a learned Society of European and international expert in all fields of cystic fibrosis (CF). Mission Statement: The European Cystic Fibrosis Society is an international community of scientific and clinical professionals committed to improving survival and quality of life for people with CF by promoting high quality research, education and care.
Terapeutic area: Paediatric Rheumatology
Istituto Gianna Gaslini represents the Paediatric Rheumatology International Trials Organisation (PRINTO). PRINTO is a non-governmental international network founded by Alberto Martini and Nicolino Ruperto in 1996, and initially included 14 European countries (about 80 countries, nearly 600 centres worldwide with 1700 members today), with the goal to foster, facilitate and co-ordinate the development, conduct, analysis, and reporting of multi-centres, international clinical trials and/or outcome standardisation studies in children with paediatric rheumatic diseases (PRD).
Terapeutic area: Paediatric Oncology
The European Society for Paediatric Oncology (SIOP Europe or SIOPE) is the only Pan-European organisation representing all professionals working in the field of childhood cancers. With more than 1,600 members across 34 European countries, today SIOPE is leading the way to ensure the best possible care and outcomes for all children and adolescents with cancer in Europe.
Terapeutic area: Muscular Dystrophy
TREAT-NMD is a network of world-leading experts for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible.
Since its launch in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide. TREAT-NMD is committed to promoting collaborations between all stakeholders in order to avoid fragmentation and duplication. Its members include clinicians, patients, industry, academic institutions, advocacy groups and regulatory agencies all of whom recognize the advantages of strong interactions in accelerating cutting-edge therapies for otherwise unmet medical needs. TREAT-NMD has a strong track-record in advising the scientific and medical community, as well as providing the pharmaceutical industry with essential go-to resources (including registry management, clinical trial assistance and post marketing services) to advance novel treatments for neuromuscular conditions. The huge success of the TREAT-NMD model is highly relevant to other therapeutic areas, particularly those in rare diseases and paediatric conditions. TREAT-NMD offers tools and resources to facilitate all phases of clinical trials:
Pre-Clinical: TREAT-NMD works to harmonise the tools used in preclinical research. The goal is to identify a limited number of animal models and experimental protocols that are considered appropriate for use by research groups around the world, thereby improving the comparability of the preclinical work and accelerating the development of new drugs into the clinic.
Phase II: The TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary group of internationally recognized academic and industry drug development experts as well as representatives of patient foundations and regulatory experts. The committee meets twice a year to review applications and provide guidance on the translation and development path of therapeutics programs in rare neuromuscular diseases. The confidential, comprehensive reviews and resulting recommendations emphasize a rigorous, milestone-driven approach enabling optimal use of funding and resources. To date TACT has reviewed over 40 applications.
Phase I-Phase III: TREAT-NMD coordinates global registries for several neuromuscular diseases. TREAT-NMD Global Registries collect, as a minimum, a standardized core set of information that is not only useful for planning clinical trials, but also for recruiting patients into them. The TREAT-NMD Global Registries are governed by a Charter and the TREAT-NMD Global Database Oversight Committee (TGDOC). There have been nearly 20 global enquiries to the registries including feasibility studies, academic research and patient recruitment for trials. In addition, TREAT-NMD has established and manages a Care and Trial Site Registry (CTSR) in order to identify those sites capable of recruiting enough patients and offering a specific standard of care and experience in clinical trials. The CTSR provides information on personnel, facilities and patient population in order to help pharmaceutical industry and clinical investigators select trial sites, and to help to identify potential partners for upcoming research projects.
Phase IV: As the first therapies for neuromuscular diseases come to market TREAT-NMD also has a key role to play in Post Marketing Surveillance (PMS). The TREAT-NMD Executive Committee (including patient representatives) are in active consultation with the stakeholders to ascertain the feasibility of developing a disease specific PMS platform. Networking and communication is a vital role of TREAT-NMD which has a global membership. A monthly newsletter is sent to over 3500 subscribers and is read by academics, patients, professionals, industry and other key stakeholders. TREAT-NMD has close to 10 years’ experience in the neuromuscular field and has a strong track record in managing international networks with a variety of stakeholders.
Terapeutic area: Chronic Kidney Disease
The European Study Consortium for Chronic Kidney Disease Affecting Pediatric Patients (ESCAPE Network) was established in 1989 by a group of centres dedicated to promoting clinical research in paediatric nephrology.
Terapeutic area: Paediatric Psychopharmacology
The European Child and Adolescent Psychopharmacology Network (ECAPN) is a well-established network of child and adolescent psychiatrists representing several EU countries, all of whom are experts in paediatric psychopharmacology.
Terapeutic area: Global Research Network
The Gianni Benzi Pharmacological Research Foundation (FGB) is a registered not-for-profit Scientific Research Organisation, promoting research in biological, medical and pharmaceutical areas, with particular reference to patients affected by rare diseases and other populations with specific therapeutic needs, including elderly and children.
Terapeutic area: Global Research Network
The European Clinical Research Infrastructure Network (ECRIN, www.ecrin.org) is a sustainable, non-profit, distributed infrastructure with the legal status of a European Research Infrastructure Consortium (ERIC). ECRIN provides support for the development and implementation of multinational clinical research projects in Europe.
Terapeutic area: Hereditary Metabolic Diseases
METABERN: The European Reference Networks (ERN)is the most advanced patient-centered program launched from the European Commission to network the most acknowledged clinical and research centers of excellence in Europe.
Terapeutic area: Global Research Network
Arsenàl.IT is the Veneto’s Research Centre for eHealth and the voluntary consortium among all the 13 Health Authorities and Hospital Trusts of the Veneto Region, funded in 2005. It aims at enhancing the quality of the clinical processes and assistance offered to citizens with an inter-enterprises view.