If you had it all: designing the ideal paediatric PH clinical trial

13 February 2026

Presented at PVRI 2026 Congress by Megan Griffiths, University of Texas Southwestern

Summary

Pediatric clinical trials encounter major hurdles, especially in drug approvals for children with PH. There is a strong emphasis on the need for flexible regulatory approaches and the incorporation of real-world data. The discussion advocates for pediatric-specific endpoints and highlights the importance of considering children's developmental stages in research. Ethical concerns regarding off-label drug use and the limited treatment options are also addressed, underscoring the need for better access to effective therapies.

 

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Megan Griffiths, University of Texas Southwestern
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