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Neonatal Drug Development Could Benefit From Rare Disease Experience

 
• By Derrick Gingery

Early parent involvement in drug development and clinical trial design could improve recruitment and outcomes, stakeholders argue.

Neonatal ICU
Parents may be more interested in therapies that could help babies leave the NICU sooner, rather than an improvement in a clinical score. • Source: Shutterstock

The successful playbook for rare disease drug development, including involving patients and advocates in early stages, could help boost efforts to bring new neonatal products to market.

Neonatal drug development has fallen behind despite legislation to incentivize pediatric research. The Best Pharmaceuticals for Children Act (BPCA) and...

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