Drugs

CDER Updates Rare Disease Efforts

CDER says it has multiple efforts underway to support rare disease drug development, particularly through data sharing initiatives that are important to advance development in this area. For example, the Center is working with the Critical Path Institute to develop the Rare Disease Cures Accelerator-Data and Analytics Platform, which “provides a database and hub designed to promote the secure sharing of existing patient-level data and encourage the standardization of new data collection,” an online agency post says. “It allows authorized users to access patient-level clinical data to better understand disease progression and disease heterogeneity (or differences among people with the same disease) across rare disease patient populations. In turn, this can inform trial design, selection of endpoints, and other important considerations for drug development.”

Additionally, CDER is working on the Standard Core Clinical Outcome Assessment Grant Program that intends to develop publicly available core sets of clinical outcome assessments that may be used as endpoints in clinical trials. “Some assessments developed through this project include physical function, pain in infants and young children, and communication in children with neurodevelopmental disorders,” it says. “We are also working with investigators from the University of Michigan to develop innovative trial designs for rare diseases with small populations.”

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

Read more

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