Pediatric Grand Rounds (CME) - How to Successfully Conduct Pediatric and Neonatal Regulatory Clinical Trials

Session Description:

Most neonatal drugs used routinely in Newborn Intensive Care Units (NICUs) are “off label” and  have variable dosages, formulations and schedules. Randomized Clinical trials (RCT) are challenging to conduct due to small sample size available in training center NICUs, when they are not combined into a cooperative network with each other or with their regional affiliate NICUs with whom they practice. Comparative clinical practice trials have been useful but a “placebo” arm has not been accepted when a referenced validated drug is not available as a comparator.

A Neonatology community consensus to establish a national neonatal regulatory research network that initially focuses on standardizing existing observational health data and applying clinical analysis tools could be the effective catalyst for enabling a true learning healthcare system in neonatology and would complement standard regulatory pediatric clinical studies to achieve safety and efficacy data needed to support indications and labelling for neonates.

A strategy is needed to address the status quo in which parents and neonatologists accept that clinical practice based on opinion and available data from other pediatric or adult studies is sufficient to inform safe and effective NICU care.

Education goals for this session:

• Review the current status of therapies in Clinical Neonatal Intensive Care Practices
• Briefly review the Regulatory Science Requirements for FDA Therapeutic Approval
• Suggest Strategies for Neonatology to collaborate and improve assessment of novel and old therapies
• Review National and Global Initiatives to Advance Neonatal Therapeutic Development

CME Credit

Accreditation

The Stanford University School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Credit Designation

The Stanford University School of Medicine designates this live activity for a maximum of 1.00 AMA PRA Category 1 Credit(s)TM.  Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Cultural and Linguistic Competency

California Assembly Bill 1195 requires continuing medical education activities with patient care components to include curriculum in the subjects of cultural and linguistic competency.  The planners and speakers of this CME activity have been encouraged to address cultural issues relevant to their topic area. The Stanford University School of Medicine Multicultural Health Portal also contains many useful cultural and linguistic competency tools including culture guides, language access information and pertinent state and federal laws. You are encouraged to visit the portal: http://lane.stanford.edu/portals/cultural.html