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International Clinical Trials

Promoting Paediatric Research

Vanessa Poustie of the NIHR Medicines for Children Research Network examines the initiative’s key successes and outlines the thinking behind its strategy for the next five years

The creation of the National Institute for Health Research (NIHR) Medicines for Children Research Network (MCRN) is considered by many as the most important initiative to occur in the UK in the development of safe and effective medicines for use in the treatment of children. Forming a key priority area of the infrastructure component of the ambitious and wide-reaching Department of Health strategy for research, Best Research for Best Health, the MCRN was established in April 2005 following an open competitive process. With the key purpose of facilitating the conduct of randomised, prospective trials and other well designed studies of medicines for children, and a primary objective, shared with the other NIHR networks, of building a world-class research infrastructure focused on delivering benefits to children, families and the public, the MCRN was introduced during an exciting period in the history of health research.

BETTER PHARMACEUTICALS FOR CHILDREN

The lack of appropriately tested or licensed medicines for infants and children has long been recognised and the turn of the new century saw a number of significant legislative changes designed to address this unsatisfactory situation, coupled with the realisation that undertaking clinical trials with children is ethical, feasible and necessary. In 2002, the Better Pharmaceuticals for Children Act was introduced into American law, shortly followed by the Pediatric Research Equity Act of 2003, both of which introduced a series of incentives and legal obligations for pharmaceutical companies to conduct trials on the use of medicines for children.

Around the same period, the European Parliament was drafting a similar regulatory framework, which has since become law with the introduction of the EU Regulation on medicinal products for paediatric use in January 2007. Recognising the importance and the implications of these plans and initiatives, the UK Government, working with the Medicines and Healthcare Products Regulatory Authority (MHRA), announced its Strategy on Medicines for Children in August 2004. One of the three main strands of this strategy was to develop a comprehensive research network to meet the needs of the regulatory framework on medicines for children. Almost immediately after this announcement, medicines for children was identified as a priority topic under the auspices of the newly established UK Clinical Research Collaboration.

Following a competitive tender process, the MCRN Coordinating Centre was established within the University of Liverpool Division of Child Health in 2005, under the leadership of Professor Rosalind Smyth, Director of the MCRN. This was closely followed by the appointment of six Local Research Networks (LRNs) located in key paediatric units across England. The dual priorities of the MCRN Coordinating Centre were to develop a portfolio of high quality studies of medicines for children, and to establish the infrastructure with which to support these studies.

The MCRN LRNs are the key vehicle for providing this infrastructure support, and each have a core staff of experienced paediatric research nurses, as well as additional management, administrative and pharmacy support, who work with study teams and local investigators to support a wide portfolio of MCRN studies. The network has extensive knowledge and experience of paediatric research, and supports publicly funded/NHS-sponsored, pharmaceutical/biotechsponsored and investigator-initiated partnership studies in over 100 NHS sites that serve approximately 6 million children. LRNs assist those wanting to conduct studies by improving set-up time and reducing the administrative burden. The networks help with detailed feasibility assessments, assist with site selection and provide support with local financial, ethical and governance arrangements. In addition, LRNs can assist with staff recruitment and training, and facilitate efficient recruitment of participants, monitoring enrolment throughout projects.

CLINICAL STUDIES GROUPS

The strategy for the development of the medicines for children research portfolio commenced almost immediately with the creation of the MCRN Clinical Studies Groups (CSGs). The network now has 13 CSGs, with more in development, which bring together research-active clinicians and other health professionals, to identify research priorities, develop funding proposals and support investigators in preparing funding applications. Consumer representation is provided on each CSG with a number of members being parents of children with relevant conditions, thereby ensuring that studies take into account the views and wishes of children and families. The CSGs are a useful resource for providing access to key opinion leaders and work with both commercial and academic researchers to facilitate the development of robust, feasible studies well suited to the UK clinical environment.

PORTFOLIO EXPANSION

The MCRN portfolio of studies has grown at a rate beyond all expectations, starting with just a handful of studies at its inception but having expanded to almost 200 studies at the present time. We anticipate that growth in the portfolio will lead to a doubling of the number of children and families recruited to MCRN studies over the next five years. Approximately half of the MCRN portfolio studies are commercially sponsored, with the majority of the remainder funded by medical charities and national research councils. Studies within the MCRN portfolio, which sits within the NIHR portfolio, can access NHS research infrastructure support via the MCRN and the Comprehensive Research Network. The MCRN portfolio includes a wide variety of studies, recruiting children from a number of different settings. This presents interesting challenges for the MCRN LRN staff, who may be supporting studies in the community one day, then recruiting very sick children from paediatric intensive care the next.

The involvement of children, parents and families is a central thread running through the activities of the MCRN. High quality research, good rates of recruitment to studies, and improved health outcomes for children depend on listening to the voices of children and young people, as well as their families and carers, and taking account of their experiences, priorities and perspectives. All stages of the research process benefit from consultation with users, and we are adamant that children can make real contributions to decision-making in these areas. The MCRN consumer involvement strategy seeks to involve children and families in all aspects of network activity, and we have received much recognition for our work, which has included collaboration with national research ethics and regulatory authorities, and key national funding bodies, in order to ensure that children’s perspectives are incorporated effectively into every stage of the research pathway.

BENEFITS FOR THE UK

The MCRN has, to date, been very active in promoting the network, and the NIHR, to the pharmaceutical industry, with a view to attracting commercial research back to the UK. The network has been described as the most industry-friendly network and we have been working hard to collaborate with the commercial sector as a way to ensure that industry studies are well-placed to run effectively within the UK. The introduction of the NIHR industry costing template and Coordinated System for gaining NHS Permissions (NIHR CSP) has led to great improvements in efficiency and transparency and is helping to make the UK a more attractive site for undertaking commercial research. This in turn will further the development of safe and effective medicines for children.

The NHS for England Operational Framework for 2009/10 has outlined a national ambition to double the number of participants entered into randomised controlled trials and other well-designed studies within five years. The NIHR and MCRN have a key role to play in achieving this national ambition and have initiated robust performance management processes to ensure delivery of a number of key metrics. The MCRN closely tracks recruitment to portfolio studies on a monthly basis and initiates, in collaboration with the LRNs and study teams, a series of action plans for studies that are at risk of falling behind target. These measures have led to improvements in study delivery and the network is close to meeting its key target of 80 per cent or more studies achieving 100 per cent recruitment within the designated recruitment period.

NEXT PHASE

The MCRN has been actively supporting and developing paediatric medicines research since 2005, and recently underwent a formal review by an international panel commissioned by the Department of Health for England. The international panel recognised and applauded the network for its achievements to date and recommended that its funding be extended for a further five years. As the MCRN enters its next phase of development, the formula of working closely with colleagues with the commercial, clinical and academic sectors to ensure that children’s medicines remains a key priority and receives the focus, infrastructure and support that it needs and deserves looks set to continue.


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Vanessa Poustie is the Assistant Director of the NIHR Medicines for Children Research Network (MCRN), a position she has held since the MCRN was established in 2005. Prior to that, she worked as a paediatric dietician before moving to Liverpool in 1997 to coordinate the CALICO Trial of oral calorie supplements for children with cystic fibrosis. Having completed a PhD which focused on dietary interventions for children with chronic disease, Vanessa worked as a lecturer in the University of Liverpool Division of Public Health, prior to moving to the University of Liverpool Division of Child Health to take up her post within the MCRN.
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