Drugs

Stakeholders Seek Changes in Effectiveness Guidance

Stakeholders are asking FDA to consider making changes to a draft guidance on demonstrating substantial evidence of effectiveness for human drug and biological products. The Biotechnology Innovation Organization says that the agency should combine the draft with existing guidance on effectiveness. It also says the guidance (1) should more clearly indicate FDA flexibility around data and/or evidence from a range of sources for demonstrating substantial evidence of effectiveness; (2) should include a reference to the use of patient experience data in the context of demonstrating substantial evidence of effectiveness; (3) could benefit from examples that are relevant to CBER-regulated products; and (4) should reflect flexibility for the full range of therapies and patients.

Regeneron says drafting of the guidance “is a step in the right direction in enhancing industry understanding of agency expectations with regard to demonstrating substantial evidence of effectiveness and also modernizing the 1998 guidance.” The company says the draft also presents an opportunity to provide more concrete recommendations that may assist sponsors in designing trials and development programs that are more scientifically sound when demonstrating substantial evidence.

Finally, Flatiron Health says the document “provides much-needed agency guidance on the flexibility in the amount and type of evidence needed to meet the substantial evidence standard to ensure patients have timely access to safe and effective drugs, and better reflects the current drug development paradigm to address unmet medical needs, rare diseases, and therapies targeting disease subsets, especially for oncology. This flexibility is important due to ethical concerns and feasibility challenges unique to certain disease areas presenting unmet medical need, as FDA recognizes in the draft guidance.”

All comments also include specific line-by-line recommendations.

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’

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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