Paediatric Clinical Trial Design & Endpoints Workstream

This Workstream aims to improve the health, quality of care, and outcomes for children with PH worldwide by enhancing collaborations among care providers, basic and clinician scientists, experienced pharmaceutical leaders, regulatory experts, patients and their families and other advocates.

Members aim to develop and apply novel strategies for the early recognition and initiation of treatment to reverse, prevent or slow the progression of disease in neonates, infants and children with PVD.  

There are several unique features of paediatric PH which affect:

• developmental biology of the cardiopulmonary system 
• mechanisms and timing of vascular injury during susceptible periods of growth and adaptation 
• differences in genetic/epigenetic mechanisms, maturational changes in vascular function and growth, co-morbidities, and responsiveness to therapeutic strategies 
• developmental differences in pharmacokinetics and pharmacodynamics, and the potential for adverse effects 
• maturational changes in right and left ventricular function 

We have a limited understanding of the disease-specific mechanisms underlying paediatric Pulmonary Vascular Disease (PVD), and this is further complicated by factors like the heterogeneity of conditions within PVD, treatments for paediatric patients being extrapolated from adult data, and a lack of well-informed guidelines and quality endpoints for assessing clinical courses and paediatric responses to therapy. 

The Workstream comprises an interactive team of clinicians, scientists, pharma colleagues, regulatory experts, and patient advocates who are working to to identify key challenges and to develop successful strategies to improve the outcomes of paediatric PH: 

• define precise, age-appropriate, disease-specific endpoints and biomarkers 
• analyse datasets from real-world studies so we can understand long-term outcomes and identify disease severity risk factors 
• explore innovative methods for paediatric trial design beyond standard MRCTs 
• develop strategies to better translate data from adult trials to accelerate the care of children 
• extend novel therapeutic strategies at the interface of clinical and laboratory science to enhance the care and outcomes in paediatric PH. 

The Workstream hosted its first webinar, “Enhancing Drug Development for Paediatric PH: An Integrative Perspective,” in February 2024. 

There are working groups planned for the Workstream, details of these are to be confirmed. 

If you’re interested in joining this Workstream, please get in touch.