Identified research priorities

Research priorityAction steps
Identifying young patients at highest risk for progression to severe diseaseEstablish a European real-world paediatric severe asthma cohort with longitudinal follow-up (into adulthood), building on existing initiatives and infrastructures (e.g. SPACE, UBIOPRED, GAN, PERMEABLE, COBRAPed, VIRASTHMA2 and the Danish National Database for Severe Asthma)
Identifying paediatric responders and nonresponders to biologicalsStandardise a definition of a responder/nonresponder
Large-scale collaboration to include paediatric patients with severe  asthma/allergy
Collaborative study efforts using joint research/clinical protocols (e.g. pragmatic real-world studies and comparative studies with biologics)
Assess whether incorporation of identified predictive biomarkers in clinical decision-making models improve clinical outcomes and are cost-effective
Understanding phenotypes of severe disease, and differences between adult and paediatric severe disease phenotypesIn-depth phenotyping of severe asthma/allergy patients combining clinical characteristics and -omics data (e.g. UBIOPRED, COBRAPed, SysPharmPediA, SPACE, PERMEABLE and VIRASTHMA2) in combination with validation in adult cohorts (e.g. SHARP and UBIOPRED)
Long-term follow-up of severe paediatric asthma into adulthood
Long-term efficacy and safety of biologics useEstablish a European real-world paediatric cohort with patients on biologics of choice and/or switchers with longitudinal follow-up (assess the maintenance dosage, long-term follow-up on overall health and QoL of patients)
Understanding molecular mechanisms to accelerate drug development using ex vivo translational modelsEstablish collaborations with immunologists and molecular biologists to ensure hypothesis/outcome validation in preclinical disease models
Implement the latest molecular biology techniques (i.e. CRISPR-Cas9 and single-cell sequencing assays) both to validate existing outcomes based on associations and to discover novel cell (sub)types that mediate underlying inflammatory processes
Develop noninvasive techniques to explore pathophysiological mechanisms
Understanding patient and caregiver perspectives on biologics usePerform qualitative studies on patient and caregiver experiences
Establish a European patient and caregiver advisory board specific for biologics use (in collaboration with European Lung Foundation and national patient organisations)
Develop and apply a platform for children and parents’ involvement
Harmonising treatment protocols across EuropeSystematically assess which differences exist within the European countries
Develop online educational programmes and regularly update these programmes based on novel scientific evidence
Applying a precision medicine approach in severe paediatric asthmaEvaluate the added value of biomarker and -omics data for individual treatment selection
Combine disease history data, clinical measures, and biomarker data into useful disease score models

SPACE: Severe Paediatric Asthma Collaborative in Europe; UBIOPRED: Unbiased Biomarkers in Prediction of Respiratory Disease Outcomes; GAN: Global Asthma Network; PERMEABLE: Personalized Medicine Approach for Asthma and Allergy Biologicals Selection; VIRASTHMA2: Inflammatory and Immune Profiles During a Severe Exacerbation in Preschool Asthmatic Children; COBRAPed: Pediatric Cohort of Bronchial Obstruction and Asthma; SysPharmPedia: Systems Pharmacology Approach to Difficult-to-Treat Pediatric Asthma; SHARP: Severe Heterogeneous Asthma Registry, Patient-Oriented; QoL: quality of life.