Drugs

GAO Report Urges Key Strategies for FDA Rare Disease Efforts

A new Government Accountability Office (GAO) report examining FDA’s programs for advancing rare disease products says training, collaboration with patient advocacy groups, and a multi-layered review process are key strategies the agency should rely on to maintain expertise and consistency in rare disease drug reviews. The report was mandated under the 2023 Consolidated Appropriations Act to examine FDA activities related to rare disease drug development and approval.

GAO says it found that FDA officials rely on a multi-layer review process to help ensure consistency among reviewers in regulatory decision-making. The officials said they are committed to using available flexibilities where appropriate to ensure safe and effective rare disease drugs are approved for marketing, according to the report.

The report also found that since 2019, the agency has launched 18 rare disease-specific programs that focus on advancing understanding of rare diseases, increasing stakeholder engagement, and supporting drug development. Now, FDA is moving forward with a new Rare Disease Innovation Hub, which is designed to enhance coordination between CDER and CBER and streamline the development and approval of rare disease therapies. The hub will also build on existing cross-center collaborations and leverage each center’s specialized expertise.

While it is still early, GAO says the initiative holds promise for guiding the agency’s rare disease activities in a strategic and coordinated fashion. It says it will leverage each Center’s rare disease-focused activities and enhance existing cross-Center collaborations. Before the agency announced its new initiative, the two Centers focused on developing Center-specific goals for their respective rare disease activities. This strategic approach, according to GAO, will ensure that FDA remains at the forefront of addressing the complexities of rare disease drug development while maintaining its commitment to public health and regulatory excellence.

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