Drugs

BIO Backs Rare Disease Endpoint Pilot

The Biotechnology Innovation Organization (BIO) says that FDA’s new Rare Disease Endpoint Advancement Pilot Program will provide greater clarity on how to develop novel endpoints, and it will help identify current limitations on regulatory flexibility involving the acceptance of such endpoints.

In just-posted comments to FDA, BIO says it agrees with the agency that pilot applications should include a detailed plan for patient engagement. BIO further recommends that FDA consider selecting applications for the pilot which feature the following characteristics:

Programs addressing areas that have not benefited from high engagement
Rare diseases with notable failures due to endpoint issues
Situations where the ability to employ regulatory flexibility is high and where biomarker data may support a clinical outcome trending in the right direction

Additionally, BIO says FDA should identify early any operational or process challenges to ensure that they can be mitigated at an early stage, rather than in the post-pilot analysis. “Providing this high-level information early on in the pilot could help sponsors adjust their applications to better meet FDA application expectations,” BIO says.

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