Drugs

PhRMA Rare Disease Day Comments

Pharmaceutical Research and Manufacturers of America (PhRMA) says the topics discussed at the FDA 2/24 Rare Disease Day public meeting were “valuable in generating a shared understanding of the issues facing manufacturers in rare disease drug development, especially regarding the use of natural history studies and registry data.” The association’s 3/29 comment letter says it believes that real-world data and its analysis to generate real-world evidence can be an important tool to inform rare disease drug development, including natural history studies.

PhRMA also said it believes it would be helpful for FDA to emphasize the acceptability of using Bayesian approaches in rare disease therapy development. “Bayesian methods have been proven scientifically and statistically valid, and should accordingly be leveraged as the basis of a substantial evidence determination to support regulatory decision-making, particularly in the context of rare disease therapy development,” the letter says. “PhRMA reiterates its prior recommendations that FDA issue stand-alone guidance addressing FDA’s current thinking on the use of Bayesian methodologies to support FDA regulatory decision-making.”

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