Drugs

Suggestions for Oncology Accelerated Approval Guidance

The National Organization for Rare Disorders says it appreciates FDA efforts in a draft guidance on clinical trial considerations to support accelerated approval of oncology drugs “since the guidance, when finalized, will likely have far-reaching impacts on rare disease drug development well beyond oncology products.” The comment letter recommends:

providing concrete guidance and best practices for effective confirmatory trial enrollment;
providing more nuanced guidance on the effective and efficient use of single-arm trials and other external controls using real-world evidence;
providing more guidance for the validation of surrogate or intermediate clinical endpoints in rare diseases and the intersections with the Rare Disease Endpoint Advancement pilot program; and
creating an education campaign to demonstrate the history and efficiency of accelerated approval drugs.

Pharmaceutical Research and Manufacturers of America (PhRMA) says the guidance provides “concise recommendations on the design of clinical trials to support accelerated approvals in oncology.” The association urges the agency to consider how single-arm trials, including those using real-world data/real-world evidence and historical clinical trials for comparison, may be used in the context of oncology accelerated approval and address that issue in the final guidance. It further suggests that FDA address considerations for when using real-world data may be appropriate to verify clinical benefit. PhRMA also provides specific comments.

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