Drugs

FDA Posts Alopecia Complex Case Study

FDA has posted a complex innovative trial case study that evaluated the benefit and risk of a higher dose than previously evaluated in a clinical trial of a drug to treat alopecia areata. The case study says the agency considered these trial design features innovative:

using external and synthetic placebo data;
Bayesian longitudinal data monitoring to generate synthetic placebo data;
Bayesian parametric modeling to analyze primary and secondary endpoints; and
using propensity scores for causal inference.

The post says that the benefits of the trial design are:

using external and synthetic placebo data allows all patients to receive active treatment, rather than randomly assigning additional patients to placebo, since in previously completed alopecia areata trials, the placebo response rate has been consistently low and stable in the severe patient population; and
without a concurrent placebo arm, the number of patients needed for the trial was reduced.

The case study lists considerations for the proposed design, discusses simulation studies the sponsor conducted, and raises points for consideration about whether minimizing the exposure of subjects to ineffective treatment in this way provides enough evidence to characterize the benefit and risk of the new dosing regimen.

FDA says it uses complex innovative trial case studies to showcase how sponsors can incorporate novel methodologies into their drug development plans. “These case studies serve as educational tools,” the agency says, “providing sponsors with practical illustrations of innovative study designs and their applications in various disease areas.”

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