Enhancing Drug Development for Paediatric Pulmonary Hypertension-An Integrative Perspective
Abstract
As with adult pulmonary hypertension (PH), high morbidity and mortality persist with diverse types of paediatric PH. Despite major advances in pharmacologic therapies based on extensive studies in adult PH, few drugs have been comprehensively studied in neonates, infants, and children, leaving current paediatric PH care largely dependent on small observational studies and extrapolation of evidence from adult clinical trials. Challenges in developing successful clinical trials in children include the need to define distinct disease phenotypes with well-characterised natural history and outcomes, the lack of established age- and disease-specific study endpoints, small and heterogeneous paediatric populations, and the common off-label use of PH-targeted drug therapies without regulatory approval. From a regulatory perspective, sufficient studies of safety, pharmacokinetics, and pharmacodynamics in neonates and young children are often lacking, and the potential role for bridging biomarkers has been underexplored. Additional opportunities include developing innovative trial designs, employing real-world data from existing registries, and fostering collaborations among sponsors, regulatory authorities, physicians, patients, and their families. By reducing reliance on off-label drug use and leveraging paediatric PH registry data, this approach offers a path toward more effective and evidence-based treatment protocols for paediatric patients. This review provides an overview of…
Read the paper
Related studies
- Safety and efficacy of COVID‐19 vaccines: A systematic review and meta‐analysis of controlled and randomized clinical trials
- Accuracy Comparison between Robot-Assisted Dental Implant Placement and Static/Dynamic Computer-Assisted Implant Surgery: A Systematic Review and Meta-Analysis of In Vitro Studies
- Determinants of passive antibody efficacy in SARS-CoV-2 infection: a systematic review and meta-analysis
- Cleaning efficacy of EDDY versus ultrasonically-activated irrigation in root canals: a systematic review and meta-analysis
- Bonding Efficacy of Universal Resin Adhesives to Zirconia Substrates: Systematic Review and Meta-Analysis
- In vitro antimicrobial activity of extracts and essential oils of Cinnamomum, Salvia, and Mentha spp. against foodborne pathogens: A meta-analysis study
Go deeper in Evidence Hub
Evidence Hub adds a study data sheet (design, sample size, evidence level, key numbers), an AI summary and a chat grounded in the paper's text.