Neonatology

Global Neonatal & Children's Health

Cross-Disciplinary Pathway

Clinical Research Pathway

Academic and Research Skills

International Stakeholder Collaboration to Develop Neonatal Therapeutics: How to Play Well in the Global Sandbox

Chair(s)

• 

  Jonathan Davis, MD

  Chief of Newborn Medicine
  Pediatrics
  Tufts Children's Hospital
  Boston, Massachusetts, United States

Panelist(s)

• 

  Thomas Miller, PhD

  VP & Global Head, Pediatric Clinical Development
  Pediatric Clinical Development
  Bayer
  Yardley, Pennsylvania, United States
• RP

  Ronald Portman, MD

  Head, Clinical Pediatric Development
  Pediatric Center of Excellence
  Novartis Pharmaceuticals
  East Hanover, New Jersey, United States
• 

  Mark Turner, PhD, FRCPCH

  Professor of Neonatology and Research Development
  University of Liverpool
• 

  Sandra Beauman, MSN, RNC-NIC

  Manager, Clinical Trials Operations
  Pediatrics
  University of New Mexico Health Sciences Center
  Albuquerque, New Mexico, United States
• 

  Jennifer Degl, MS

  Author/Speaker/Advocate
  Speaking for Moms & Babies, INC, New York, United States
• 

  Deborah Discenza, MA

  CEO/Publisher
  PreemieWorld
  Burke, Virginia, United States

Although the field of neonatology has evolved significantly since patients were routinely administered chloramphenicol and 100% oxygen, major knowledge gaps and unmet therapeutic needs persist. The majority of drugs used to treat critically ill neonates are used off-label, with incomplete information on dosing, safety, and/or efficacy. Despite efforts to design and perform high-quality neonatal clinical trials, optimal treatments for the unique conditions related to preterm birth remain elusive. To begin addressing these unmet needs, the Food and Drug Administration (FDA) and the Critical Path Institute (C-Path) launched the International Neonatal Consortium (INC) in May of 2015. The Consortium convenes academic experts, regulators, nurses, industry sponsors, and patient/parent advocates from across the globe to “forge a predictable regulatory path for evaluating the safety and effectiveness of therapies for neonates.”

In its first 5 years, INC has achieved notable successes through interdisciplinary collaboration, including:

Publication of: “Safety, Dosing, and Pharmaceutical Quality for Studies that Evaluate Medicinal Products (Including Biological Products) in Neonates” to assist investigators and sponsors in designing clinical trials (used in FDA Guidance)

Development of standardized definitions, outcome measures, and master protocols for clinical trials of treatments of retinopathy of prematurity (ROP), bronchopulmonary dysplasia, and seizures in order to obtain consistent, robust, and discriminatory data

Development of a neonatal adverse event severity scale to harmonize adverse event reporting for neonatal clinical trials

Publication of a paper optimizing long-term neurodevelopmental follow-up assessments following trials of medicinal products in neonates

Our Cross-Disciplinary approach will highlight the interaction and collaborative efforts needed by key stakeholders to create tools to streamline research on neonatal therapies.

Presentations:

• 1:00 PM - 1:10 PM US CT

  How Did a Group of Academic Researchers, Families, Nurses, Regulators, and Industry Representatives Team Up to Facilitate Neonatal Research?
  

  Presenter: Susan McCune, MD – Office of the Commissioner/ FDA
• 1:10 PM - 1:25 PM US CT

  Developing Recommendations for Neonatal Seizure Treatment Trial Design
  

  Presenter: Janet Soul, MDCM, FRCPC – Boston Children's Hospital
• 1:25 PM - 1:40 PM US CT

  Developing and Validating a Neonatal Adverse Event Severity Scale
  

  Presenter: Thomas Salaets, MD PhD – KULeuven
• 1:40 PM - 1:50 PM US CT

  Translating Real World Data (RWD) into Real World Evidence (RWE) for Use in Neonatal Clinical Trials
  

  Presenter: Kanwaljit Singh, MD, MPH – Critical Path Institute / International Neonatal Consortium
• 1:50 PM - 2:00 PM US CT

  Translating Real World Data (RWD) into Real World Evidence (RWE) for Use in Neonatal Clinical Trials
  

  Presenter: Jonathan Davis, MD – Tufts Children's Hospital
• 2:00 PM - 2:15 PM US CT

  Development of a Retinopathy of Prematurity Activity Scale and Clinical Outcome Measures for Use in Clinical Trials
  

  Presenter: Lois Smith, MD, PhD – Boston Children's Hospital, Harvard Medical School
• 2:15 PM - 2:25 PM US CT

  Is there a path forward for neonatal therapeutics?
  

  Presenter: Gerri Baer, MD – Food and Drug Administration
• 2:25 PM - 3:00 PM US CT

  Panel Discussion
  

  Presenter: Thomas Miller, PhD – Bayer
• 3:00 PM - 3:30 PM US CT

  Break
• 3:30 PM - 4:00 PM US CT

  Click for Live Q&A: International Stakeholder Collaboration to Develop Neonatal Therapeutics: How to Play Well in the Global Sandbox
  

  Presenter: Belinda Thomas