Drugs

Externally Controlled Trial Guidance

FDA has published a draft guidance, Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products, with recommendations for sponsors and investigators considering the use of externally controlled clinical trials to provide evidence of the safety and effectiveness of a drug product. The document addresses considerations for the design and analysis of externally controlled trials to study the safety and effectiveness of drugs, including a discussion of threats to the validity of trial results from potential bias.

“Although various sources of data can serve as the control arm in an externally controlled trial, this guidance focuses on the use of patient-level data from other clinical trials or real-world data sources, such as registries, as well as electronic health records and medical claims,” the draft says. “The guidance also describes considerations related to communicating with FDA and ensuring access by FDA to data from an externally controlled trial.”

The guidance does not address other types of external controls, such as using summary-level estimates instead of patient-level data. It also does not discuss details of the design and analysis of a natural history study nor the reliability and relevance of various sources of real-world data that could be used in an externally controlled trial. Finally, the document says, the guidance also does not discuss considerations for using external control data to supplement a control arm in a traditional randomized controlled clinical trial.

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