Neonatology
Global Neonatal & Children's Health
Cross-Disciplinary Pathway
Clinical Research Pathway
Academic and Research Skills
Jonathan Davis, MD
Chief of Newborn Medicine
Pediatrics
Tufts Children's Hospital
Boston, Massachusetts, United States
Thomas Miller, PhD
VP & Global Head, Pediatric Clinical Development
Pediatric Clinical Development
Bayer
Yardley, Pennsylvania, United States
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.
Presenter: Susan McCune, MD – Office of the Commissioner/ FDA
Presenter: Janet Soul, MDCM, FRCPC – Boston Children's Hospital
Presenter: Thomas Salaets, MD PhD – KULeuven
Presenter: Kanwaljit Singh, MD, MPH – Critical Path Institute / International Neonatal Consortium
Presenter: Jonathan Davis, MD – Tufts Children's Hospital
Presenter: Lois Smith, MD, PhD – Boston Children's Hospital, Harvard Medical School
Presenter: Gerri Baer, MD – Food and Drug Administration
Presenter: Thomas Miller, PhD – Bayer
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