“Training & education, participation with purpose, and institutions that are designed to work for children”

What we learned from our Putting Paediatrics’ Needs First in Clinical Research webinar.

We invited three luminaries from the medical field, Jenny Preston, Senior Patient and Public Involvement Lead for NIHR Alder Hey Clinical Research Facility at the University of Liverpool, Rhian Thomas-Turner, R&D Lead at Noah’s Ark Children’s Hospital for Wales, and Dr Jo Wilmshurst, Head of Paediatric Neurology at Red Cross War Memorial Children’s Hospital, University of Cape Town, to join Dr Elin Haf Davies to champion the UN International Child Day and tackle three challenging topics at our recent webinar through a series of presentations and a close-out roundtable.

Key themes discussed were

1) why it’s our duty to make the experience of clinical trials easier for children & parents

2) what remote patient monitoring has taught us about paediatric patients’ needs through the last eighteen months

3) how we can work together with all partners to break down the barriers to innovation in children’s medicine

So, what did those discussions entail?

Why it’s our duty to make the experience of clinical trials easier for children & parents

Rhian Thomas-Turner reminded us that, under the UN Convention on the Rights of the Child (CRC) it’s a legal right for children to have access to, and development of, suitable medicines and that we must recognise that the obligations to child health are legal rather than moral or ethical: a recent medical paper cited where, due to scarce resources, boys were prioritised over girls, and the paper referred to the decision as an ethical issue rather than a breach of international law.

There needs to be an interpretive community push through our collective writing and communication to ensure people understand children’s human rights to health. “Too often paediatric medicines need to be adapted in a ward setting, e.g crushing tablets where no liquid suspension is available, so we have to question whether these medicines are appropriate for paediatric needs, and without knowing fully how these medicines act in the body, whether we can suggest they do give children the right to grow and develop (as is their legal right)?”

Trials often have too many obligations on children and their families and we should seek to design trials that leverage technology to create meaningful endpoints for paediatric patients and, where possible, keep them out of a hospital setting and monitor them in their own environment. 

Jenny Preston of University of Liverpool posed questions to ask ourselves when designing paediatric research and trials:

• Are we asking the right research question(s)?
• Are we measuring the right (or most meaningful) outcomes?
• Do our research tools and instruments make sense?
• How child/family-friendly is our study?

We must ask ourselves: can we design better clinical research and trial experiences that recognise children’s other rights under CRC, such as the right to play and the right to education?

What remote patient monitoring has taught us about paediatric patients’ needs through the last eighteen months

The health impact for children from the aftershock of COVID-19 is a concern, from lack of access to immunisation programmes through to delayed diagnosis that would impact interventions. 

Jo Wilmhurst highlighted how COVID-19 caused huge disruption to paediatric care and low-income countries suffered disproportionately as they were less able to access telemedicine and telehealth, leading to deleterious, long-term effect on the health of its children:

“Care Delivery for Children With Epilepsy During the COVID-19 Pandemic: An International Survey of Clinicians” found that, despite telehealth usage surging to over a quarter of appointments (i.e. where patients exclusively used telehealth as channel), 91.5% of respondents reported changes to outpatient care for paediatric epilepsy as the pandemic unfurled, and over 90% could not get access to targeted treatments.One particular concern the paper raised was the long-term implications these disruptions pose to patient care, and another paper, Routine childhood immunisation during the COVID-19 pandemic in Africa: a benefit–risk analysis of health benefits versus excess risk of SARS-CoV-2 infection, put this into stark relief;

“In the high-impact scenario, for every one excess COVID-19 death attributable to SARS-CoV-2 infections acquired during routine vaccination clinic visits, 84 (95% UI 14–267) deaths in children could be prevented by sustaining routine childhood immunisation in Africa.”

That raises a challenging question: under the CRC and children’s rights to medical assistance and health care, is access to technology such as remote patient monitoring and telehealth a legal imperative in instances where no alternative is available?

How we can work together with all partners to break down the barriers to innovation in children’s medicine

There is frequent discussion regarding how regulators, HCPs and drug manufacturers can work together to advance paediatric research & trials, but that omits a rather important stakeholder; the patient and their family. 

As Jenny Preston posited, “If a company was developing a new soft drink, would they bring that product to market without consulting their target audience? The answer, of course, is no they would not, so why should it be any different when designing and conducting paediatric health studies?”

Jenny cited her research that found 83% of young people think they must be involved in decisions that the government makes about healthcare, and 14% believe that young people should lead the discussion on healthcare. 

Children have the right to express views freely in all matters affecting the child as per article 12 of the CRC; we can, should and must work with them to ensure their voices are heard and applied through methods such as Dr Laura Lundy’s model of participation with purpose.

The University of Liverpool & NIHR created the GenerationR Alliance to set up Young People’s Advisory Groups (YPAGs) around the world. Now over 50 strong, these groups get actively involved in the design and delivery of clinical research to ensure that research is relevant to young people and their families. 

“Prioritising child health needs isn’t to take away from the needs of our ageing population but if we don’t address child health now, we won’t have a healthy, prosperous society” Elin Haf Davies

Dr Jo Wilmhurst believes that preventative interventions would significantly impact on the burden of child health through trained and equipped healthcare professionals who advocate, lead, train and educate in their local areas. She cited, The African Paediatric Fellowship Programme, which brings the best doctors in Africa together, trains them, and sends them home to implement what they’ve learned. These doctors have gone to lobby for access to medications, collaborated with international associations, and completed research such as publications and PhD proposals. 

Rhian suggested we improve the discourse around human rights and access to develop suitable medicines for children, saying “What we can use (human rights law) for is to have these conversations: we don’t need to move into hard legislation to change the narrative around access to medicines and development of the right medicines”.

To close, we asked our presenters “What would add the greatest value to child health?” 

What did we learn?

Training and education were referenced consistently by all of the speakers, whether medical training to boost preventative interventions or discourse with the wider community to reiterate that children’s rights are legal rights and need to be respected in developing new and appropriate therapies for children.

To design better experiences in paediatric clinical research we need to use the gravitas of the law to drive participation with purpose and provide training & education so that all parties understand the benefits, not least that better patient engagement and experiences leads to better research outcomes.  

Sign up for our newsletter to receive our latest papers, posters and platform updates direct to your inbox!