OVERVIEW

Regulatory Considerations in Development of NEC Therapeutics is organized by NICUniversity.

Description:
Dr. Baer discusses the benefits of FDA approval for pediatric and neonatal therapeutics, including established effectiveness, pre- and post-marketing safety assessments, and defined clinically-meaningful endpoints. In the case of NEC, prevention of disease is a clinically-meaningful outcome. She also provides information on drug development tools that are relevant to NEC research, including clinical outcome assessments, biomarkers (e.g. for prediction of a patient’s risk), and animal models. Finally, potential clinical questionsare considered for the next phase of NEC research:

• What non-pharmacologic strategies for NEC prevention are most effective?
• Is there a clinical prediction strategy that can help define at-risk babies, either for non-pharmacologic prevention or pharmacologic prevention?
• What are the most effective treatment strategies for promoting long-term health?

Learning Objectives:
• Review the current understanding of the pathogenesis, prevention and treatment of NEC.
• Establish a transdisciplinary collaboration of stakeholders to accelerate the implementation and standardization of established evidence based practices that can help to reduce the incidence and morbidity of NEC.
• Explore methods for empowering parents/families and supporting their engagement as part of their baby’s care team.
• Serve as a venue for researchers to create new collaborations and lines of investigation, including shared clinical databases and tissue banking.

Additional details will be posted as soon as information is available.