Drugs

FDA, EMA Rare Diseases Cluster

FDA and the European Medicines Agency (EMA) are establishing a joint cluster on rare diseases to share scientific evaluation of various aspects of drug development for rare diseases. A document on terms of reference for the cluster says that the aspects include selection and validation of trial endpoints, potential trial designs in small populations, opportunities for regulatory flexibility (approval supported by other than two adequate and well-controlled studies and/or use of a novel endpoint), determination of safety populations, evaluation of pre-clinical data needed to support human trials, and design and conduct of post-marketing studies especially in the case of accelerated approval (FDA) and conditional/exceptional approval (EMA) or breakthrough designation (FDA) and PRIME designation (EMA).

“The main mechanism to achieve these goals will be regularly scheduled teleconferences,” the document says, “including individuals from review divisions and therapeutic teams, for exchange of information and experiences. It may be appropriate to agree to additional in-depth discussions on specific topics during separate ad hoc teleconferences when it might be relevant to include participation of appropriate EMA/FDA staff members.”

Cluster participants are to discuss and determine which topics will be discussed at this cluster and which will be deferred to other platforms/clusters such as pediatric, oncology, and pharmacovigilance-related topics.

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

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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