Drugs

Neonatal Neurodevelopmental Safety Study Guide

FDA has published a draft guidance entitled “Long-Term Clinical Neurodevelopmental Safety Studies in Neonatal Product Development” that provides a framework for neurodevelopmental evaluations that could be useful to assess long-term safety of a medical product intended for use in neonates. The document is focused on long-term evaluations of neurodevelopmental safety. “Although assessments of nephrotoxicity, pulmonary toxicity, and toxicity to other tissues and organs may also be warranted in neonatal medical product development, the approach to those assessments is outside the scope of this guidance,” it says.

FDA says the following are key points to consider:

A controlled study design is recommended to allow for more reliable discrimination of drug or device-related patient outcomes from outcomes caused by other factors
Neurodevelopmenta loutcomes should be evaluated up to at least two years adjusted age; evaluation of some outcomes may also require assessment later in childhood
A general evaluation of all key neurodevelopmental domains is recommended, with additional evaluations based on domains of concern; physical, mental, and social health should also be included
Neurodevelopmental safety evaluations should be well-defined, reliable, and relevant; clinical outcome assessments should include validated tools, when available
Sponsors should ensure reliable and consistent evaluations across study sites
Data should be collected on co-variates that may affect neurodevelopmental outcomes (e.g., perinatal factors, comorbidities, socioeconomic factors, etc.)
Certain objective developmental measures with established reference standards (e.g., growth, vision, and hearing screening) captured during routine care may be used when collected reliably, but general developmental screening and formalized assessments of neurodevelopment are not interchangeable
Long-term functional assessments provide the most meaningful outcome information; in general, adjunctive assessments and biomarker measures (e.g., neuroimaging and neurophysiologic studies) may be useful to support the evaluation of neurodevelopmental safety in certain circumstances, but may not substitute for functional evaluations

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome