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FDA News: Issue 27, August 2021

Author: [AUTHOR] Published on 8/13/2021 5:00:00 AM


FDA/M-CERSI Announce a Virtual Workshop Entitled “Advancing the Development of Pediatric Therapeutics Complex Innovative Trial Design” on September 1 & 2, 2021

For nearly two decades, findings from the Action to Control Cardiovascular Risk in Diabetes (ACCORD) clinical trial have informed guidelines for treating type 2 diabetes (T2D). The trial enrolled >10,000 T2D patients with elevated cardiovascular disease (CVD) risk and investigated whether moderate or intensive treatment to lower blood glucose levels was more effective in preventing or delaying related events, including myocardial infarction, stroke and even death. Surprisingly, the intensive treatment arm of the trial was terminated early due to a significant increase in CVD-related and overall mortality. However, using a novel computational implementation of dynamic time warping, we found evidence of a previously unrecognized genetic subtype of patients with T2D that demonstrated a 50% reduction in CVD risk when treated intensively compared to standard glycemia treatment.

Using a genome-wide association analysis, we identified the MAS1 gene as a candidate for the modified risk observed in this subtype. Furthermore, we developed a polygenic score that predicted membership of this subtype with 95% balanced accuracy in a withheld test set. Overall, we found evidence of a T2D clinical group in ACCORD that benefited from intensive glycemia treatment, and membership in this group could be predicted using genetic variants. This webinar will discuss how this study generated new hypotheses with implications for precision medicine in T2D and represents an important development in this landmark clinical trial warranting further investigation.

Learning Objectives: How to think about and analyze data from clinical trials to identify patient populations most likely benefit from specific intervention. Using computational approaches that leverage longitudinal data can provide new insights into complex diseases.

The U.S. Food and Drug Administration (FDA) Division of Pediatrics and Maternal Health and Complex Innovative Trial Design Pilot Meeting Program in the Center for Drug Evaluation and Research (CDER) and the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) will host a two-day virtual public workshop entitled “Advancing the Development of Pediatric Therapeutics Complex Innovative Trial Design”. The purpose of this workshop is to discuss opportunities for leveraging complex and innovative trial designs, understand the challenges with their applications, and develop solutions on how challenges in the designs can be overcome. The workshop will specifically focus on two topics of interest: bridging biomarkers in pediatric extrapolation and Bayesian techniques in pediatric studies. In addition, the workshop will allow for an open dialogue around the use of these approaches among regulators, industry, academia, and patient organizations.

The date, time, and location of the workshop are as follows:
Date: September 1 & 2, 2021
Time: 10:00 AM - 3:00 PM ET both days
Location: This public workshop will be held in virtual format only.
Agenda and Registration: To access the agenda and registration for the public workshop, visit the following website: https://cersi.umd.edu/event/16424/advancing-the-development-of-pediatric-therapeutics-adept-7. Registrants will receive confirmation when they have been accepted. There is no registration fee to attend this public workshop.
Webcast Link: https://cersi.umd.edu/event/16424/advancing-the-development-of-pediatric-therapeutics-adept-7

If you have any additional questions about this workshop, please contact Lily Mulugeta at yeruk.mulugeta@fda.hhs.gov. To learn more about the FDA’s Complex Innovative Trial Design Pilot Meeting Program, visit at the FDA complex-innovative trial design pilot meeting program website.


The Office of Clinical Pharmacology (OCP) is pleased to offer the e-mail subscription service Clinical Pharmacology Corner. This is a free service from FDA to provide occasional updates from OCP regarding newly approved therapies, new regulatory and scholarly publications, upcoming events and other items of interest. Subscribe today at https://public.govdelivery.com/accounts/USFDA/subscriber/topics and select Clinical Pharmacology Corner under Drugs.

We always welcome your thoughts regarding the format, content, and utility of this communication. Comments may be sent via email to ocp@fda.hhs.gov.

This communication was prepared by Office of Clinical Pharmacology, Office of Translational Sciences, CDER, FDA.

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