2021 INC Scientific Workshop
C-Path’s International Neonatal Consortium held its two-day annual scientific workshop in a virtual format, October 19-20, 2021.
First day of the meeting focused on the rapidly evolving field of cell and gene therapy in neonatal population. The current landscape of gene therapy clinical trials in neonates was discussed, followed by perspectives from stakeholders including industry, academic, regulator, and parent partners of INC. Subsequent discussion focused on the diagnostic platforms for whole genome sequencing, ethical considerations related to diagnosis of genetic conditions and participation in gene therapy trials.
Second day of the meeting focused on the utility of real-world data to generate real-world evidence as it pertains to neonatal research, and how C-Path’s core competencies of data science and analysis applied to real-world data promises to accelerate our understanding of some of the most challenging conditions in the neonatal population.
Launched on May 19, 2015, INC is C-Path’s ninth consortium – a global collaboration formed to forge a predictable regulatory path for evaluating the safety and effectiveness of therapies for neonates. “By uniting stakeholders from research institutions, drug developers, regulatory agencies, patient advocacy and other organizations,” said Janet Woodcock, Director of the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (FDA/CDER), “INC can develop practical tools that can be incorporated into clinical trials for neonates, which will then lead to more successful, efficient trials and provide this population with better treatments.”
The scientific workshop that led to the formation of the consortium – “Roadmap for Applying Regulatory Science to Neonates” – was held at the FDA on October 28–29, 2014: fda.gov/Drugs/NewsEvents/ucm410863.htm.
INC accelerates the development of safe and effective therapies for neonates. The consortium engages the global neonatal community – families, neonatal nurses, academic scientists, regulators, pharmaceutical investigators, advocacy organizations, and funders – to focus on the needs of the neonate. Through teams that share data, knowledge, and expertise, INC advances medical innovation and regulatory science for this underserved population.