Development of drugs for children is of great importance for medical and ethical reasons. This requires scientific and clinical excellence, educational efforts and competent clinical platforms collaborating in networks to collect pediatric patients in numbers large enough to conduct conclusive studies. Establishment of networks for this mission has been politically promoted in the European Union and the United States.
Background
Less than one third of marketed drugs worldwide have any approved therapeutic indication for children of various ages, and less than half of them have ever been tested in children – for newborns less than ten percent. Children have the same rights as adults to be treated with effective and safe medicines and take part of new therapeutic treatment strategies. American (FDAMA 1997, Pediatric Rule 1998, Best Pharmaceuticals for Children Act 2002) and European legislation (Pediatric Regulation No 1901/2006) have now implemented legal claims to document pediatric effects, side effects and safety of any new drugs or new indications seeking marketing authorization.
The “Conect4Children” initiative (www.conect4children.org, COllaborative Network for European Clinical Trials for Children - “c4c”) is a large collaborative pediatric network that will facilitate the development of new drugs and other therapies for the entire pediatric population. The Conect4Children consortium aims to enhance the competitiveness of Europe as a critical region for developing medicines for children by using existing expertise, patient access and developing common processes to be applied to disease natural history studies, registries, studies of new therapies and comparisons of existing therapies.
The consortium is a novel collaboration between academic and private sectors that includes 33 academic and 10 industry partners from 19 European countries, more than 50 third parties and around 500 affiliated partners. The six-year (extended one year) multidisciplinary project started on May 1st 2018, and is probably the largest EU public-private-partnership consortium known. It is a pioneering opportunity to build capacity for the management of multinational pediatric clinical trials across Europe, bringing together key stakeholders across academia and industry. Strong links with regulators have been established.
The medical and clinical pharmacological rationale: The overall therapeutic result is dependent on the intrinsic features of the drug, its interactions with all biological processes that mediate effects and toxicity and that are part in any step of the uptake, distribution, metabolism, and excretion of the drug. There is a large variation in these processes among children, both within and between age groups, and more so than in adults. This is caused by the effects of the maturation on all these biological processes. The maturation itself may also modulate the expression of genetic traits that are very important for the above processes.
Aims
The project aims to generate a sustainable infrastructure that optimises the delivery of clinical drug trials in children in high medical need areas. This will be done by implementation of a single point of contact (SPOC)* for all sponsors, sites and investigators in each country. Our dept is part of the Swedish SPOC currently located at Queen Silvia´s Childrens´ hospital (Västra Götalandsregionen). The SPOC is responsible for:
- a single national point of contact for all sponsors, sites and investigators.
- efficient trials adopting consistent approaches, aligned quality standards and coordination of sites at national and international level.
- collaboration with specialists and national networks.
- high quality input to study design and preparation through rigorous strategic and operational feasibility assessment.
- promotion of innovative trial design and quantitative science methods.
- education and training platforms to shape the future leaders of pediatric drug development.
- sustainable support for all these activities.
Importance
Clinical trials with medicinal products for pediatric use are one of the most sensitive areas from a medical and an ethical perspective. c4c addresses critical problems with the design, implementation and operational conduct of pediatric clinical trials. Such problems include, fragmented and redundant efforts between sponsors, sites and countries and the paucity of patients available for studies on many pediatric indications, c4c will also address the need for multiple capable sites and expertise. The European network will have a significant impact on how we develop much-needed innovative and improved medicines for babies, children and young people.
A number of clinical PIs at various clinical sites (Third parties) in Sweden collaborate by recruiting and treating pediatric patients according to protocol. Prof Carlo Giaquinto (Padova) and Dr Mark Turner (Liverpool) are coordinators.
Research techniques
Feasibility, trial design, biostatistics, clinical methodologies for assessment of outcome parameters, regulatory affairs, pharmacovigilance.