Drugs

Firms Seek Broad Extrapolation for Pediatric IBD Dosing

Joint comments from AbbVie, Genentech, and Johnson & Johnson on a draft guidance about pediatric inflammatory bowel disease (IBD) drug development question why FDA does not allow broader use of extrapolating adult data to determine an appropriate dose for pediatric patients. “We recommend that the agency re-evaluate the need for routinely enrolling 100-120 pediatric study participants (ages 2 to 17 years) over two doses… ‘to inform the risk-benefit assessment’ for approval,” the comments say.

“Modeling and simulation and other appropriate methods can be used to identify the appropriate dose for pediatric patients,” the companies continue. “We recommend FDA encourage use of these tools rather than fully powered clinical studies. Efficacy (benefit) may be extrapolated from adults with little uncertainty, and safety in adults (and often other pediatric populations) will inform the risk in adolescents and children…”

The recently released draft guidance, entitled Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment, provides agency recommendations about the necessary attributes of clinical studies for drugs being developed for the treatment of pediatric ulcerative colitis or pediatric Crohn’s disease, including study population, study design, efficacy considerations, and safety assessments.

“The recommendations for clinical study design in this guidance are based upon the assumption that a robust development program is being conducted in adults and that efficacy data from adults will be available to help inform the pediatric program and to support extrapolation of efficacy,” FDA says. “Sponsors seeking to develop a drug only for pediatric ulcerative colitis or Crohn’s disease patients in the absence of an adult program should meet with the appropriate review division to discuss their proposals.”

Additionally, the comments from the three companies also raise issues with FDA encouraging the inclusion of adolescent subjects in adult ulcerative colitis or Crohn’s disease clinical trials. “It is possible for sponsors to attempt this, but the agency should not expect to see large numbers of adolescents in Phase 3 studies that include placebo, due to unwillingness of sites to potentially expose such subjects to placebo, and the fact that many ex-U.S. countries do not allow enrollment of adolescents until efficacy and safety has been adequately demonstrated in adults,” they write.

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