The paediatric respiratory community in the UK is to address “age discrimination” against children with severe asthma, announcing plans to undertake a pragmatic clinical trial that will compare biologics in children with severe therapy-resistant asthma (STRA) and assess the benefits of specialist paediatric severe asthma centres.
The year long non-inferiority TREAT trial, to be funded by the National Institute for Health Research, will compare the efficacy of mepolizumab and omalizumab in children with STRA, determining which biologic is best for each individual child, with asthma attacks as the primary outcome. It will also investigate biomarkers of response in children.
Writing in the Lancet Respiratory Medicine, the experts led by Professor Sejal Saglani from Imperial College London warn of unmet therapeutic needs in children with STRA, highlighting “stark differences” in their health care compared to that of adults with severe asthma.
“Unlike adults, children with severe asthma are not treated at specialist centres, clinical trials of novel therapeutics are not tailored for children, and novel biologics are being approved without evidence of efficacy.”
They point out that without specialist services – which are associated with improved quality of life and asthma control as well as a reduction in healthcare use – children could be exposed to potential harm associated with inappropriate prescribing of biologics, or they could be denied an appropriate therapy.
“Importantly, we will aim to demonstrate the advantages of specialist paediatric severe asthma centres and thereby encourage childhood STRA to also be commissioned as a specialist service,” they said of the new trial.
“This framework should also be attractive for the pharmaceutical industry to engage with paediatricians to design future trials specifically for children and enable efficient recruitment and regulatory approval,” they added.
Key concerns over biologics
The experts say children with STRA and refractory difficult asthma should be considered for biologics, but flag three key concerns: first, that the appropriate studies are not being done in children; second, that drug development is aimed at therapeutic targets from adult models of disease; and finally, there are very little data to guide choosing the optimal biologic for individual patients.
They say the decision by the European Medicines Agency (EMA) last year to licence mepolizumab for use in children aged 6–17, despite inadequate efficacy data in adolescents and complete absence of efficacy data in those aged 6–11 years, is of huge concern.
“We are concerned that, having achieved approval for a paediatric licence by extrapolation of adult data for one biologic, the pharmaceutical industry will adopt the same approach for the many other biologics being approved for adult severe asthma.”
Children with severe asthma cannot continue to be treated as second-class citizens, they said.