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Involving children in clinical research has long been a topic that is fraught with conflict. Children make up a huge percentage of the patient population for many diseases, including asthma, diabetes and most genetic rare conditions, so it makes sense that they would participate in research to bring lifesaving treatments for these conditions to market.

Yet children must generally rely on adults to make decisions about their care. That includes deciding whether they will participate in a clinical trial, what constitutes a safe and appropriate environment in which to involve children in research, and how to evaluate that child’s consent to participate ethically. For example, if the research causes them pain or fear, if it holds little potential benefit to a child who may receive a placebo, or if it is overly disruptive to their young lives, who determines whether it is too much burden for that child to participate? These are serious ethical considerations that must be factored into the decision to run pediatric trials. Then once a trial begins, developers face ongoing challenges in gathering real world outcomes from these children/young people, assessing their feedback about their condition, and adapting the trial protocols as they mature.

Despite these obstacles pediatric patients play a vital role in the research process. Involving them in clinical trials is the only way that we can increase our understanding of childhood conditions and improve healthcare for pediatric populations. Without their involvement, their physicians can only guess at what drugs and doses are appropriate.

Children should be seen and heard

Nuffield Council on Bioethics in the UK is working to address some of these obstacles. In 2015, the organization published a report on ethical issues related to children and clinical research, arguing that genuine engagement with children, young people and parents throughout the research endeavor is ethically necessary if children are to be involved in the research environment. They recommend that children/young people should be involved not only as participants in clinical trials but also in advising sponsors on all aspects of clinical trials, including study design and protocol development. The report recommended that industry partners should contribute financially to the running costs of initiatives that facilitate involvement of children and young people, such as the network of Young Persons’ Advisory Groups (YPAGs).

To build on this idea of integrating children into the research conversation, the Council hosted a meeting in April, 2016 that brought together young people with representatives from the National Institute of Health Research (NIHR), and experts in drug development, policy making, and bioethics to explore how the life-sciences industry can work more collaboratively with young patients to improve research. Throughout the meeting, participants discussed the benefits of young people’s involvement in the wider research agenda, the challenges to achieving such involvement, and crucially, ways in which we could start tackling those challenges.

The meeting was made up of a number of brainstorming workshops led by NIHR experts, industry professionals and young people with experience influencing how research is carried out. A number of suggestions for future action were put forward in these sessions, including the need for a ‘statement of aspiration’, to which individuals or organizations could sign up that could then inform the development of a position paper setting out guidance on good practice and showcasing what young people can contribute. Participants also discussed the need to factor the child’s input into the protocol and study design to minimize the impact on their lives while maximizing the benefits they can hope to achieve from this research.

The onus now is on industry leaders to further promote this research and to fund ongoing work to better understand the role children can play in clinical trials and how we can protect their interests throughout this process. The challenge is to find an appropriate means to do so that retains the genuine independence of the YPAGs, and also allows for other models of collaboration to exist alongside. Engaging children in the planning of these trials is in everyone’ interest, particularly organizations that are pursuing rare disease studies and diseases with large pediatric populations. By working collaboratively we can overcome the ethical issues that may present barriers to their participation, and best ensure that all of their emotional, physical and medical needs are being met.

 

To learn more about the meeting, please read the “Statement of aspiration: improving research by involving children and young people,” available from the Nuffield Council on Bioethics.