Virtual | Virtual
Event Title
FDA-M CERSI: Advancing the Development of Pediatric Therapeutics Complex Innovative Trial Design Public Workshop
September 1 - 2, 2021
- Date:
- September 1 - 2, 2021
- Day1:
- Wed, Sep 1
- Day2:
- Thu, Sep 2
FDA’s Division of Pediatrics and Maternal Health and Complex Innovative Trial Design Pilot Meeting Program in CDER and The University of Maryland CERSI, are announcing a 2-day public workshop, “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 public workshop will be held on:
Date: September 1 and 2, 2021
Time: 10:00 am to 3:00 pm
The public workshop will be held in virtual format only. Please note that due to the impact of this COVID-19 pandemic, all meeting participants will be joining this public meeting via an online teleconferencing platform.
Webcast:
https://cersi.umd.edu/event/16424/advancing-the-development-of-pediatric-therapeutics-adept-7
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.
Day 1 slides and recording: (list the attached slides and recording link: Day 1:
- Garnett, Christine, Ph.D. Regulatory Perspective: Biomarkers for Pediatric Extrapolation (PDF -703.37 KB)
- Conklin, Laurie, M.D. Modeling and Bridging Biomarkers to Support Pediatric Extrapolation (PDF - 187 MB)
- Fleming, Thomas R. What Criteria Justify Use of a ‘Bridging Biomarker’ for Extrapolation to Pediatric Patients? (PDF- 367.77 KB)
- Hsu, Daphne T. M.D. Case Study in Assessing Disease Similarity:Heart Failure in Adult and Pediatric Patients (PDF - 717 KB)
- Portman, Ron. Leveraging adult data to support a biomarker extrapolation: Entresto (PDF - 1.4 MB)
- Yao, Lynne, M.D. Pediatric Therapeutics Development in the 21st Century: It’s Not Your Mother’s Clinical Trial (PDF - 177.79 KB)
Day 2 slides and recording: (list the attached slides and recording link: Day 2:
- Welcome to ADEPT 7: Advancing Complex Innovative Trial Designs to Accelerate Drug Development in Pediatric Patients
Session 2: Bayesian techniques in Pediatric Studies (PDF - 267.99KB) - Rothmann, Mark. An FDA Perspective: Drug development and decision-making in pediatric settings starts with adult studies* (PDF - 430.41 KB)
- Thomson, Andrew. Bayesian techniques in paediatric development (PDF - 502.53 KB)
- Gamalo, Margaret Ph.D. Assessment of Consistency and Bayesian Approaches for Demonstrating Efficacy in Pediatric Populations (PDF - 457.65 KB)
- Best, Nikky and Hammer, Anne. Bayesian borrowing of adult efficacy data in paediatric drug development: A Case Study (PDF - 329.38 KB)
- Lee, Paul R. M.D., Ph.D. Assessing Disease Similarity in Adult and Pediatric Patients with Multiple Sclerosis (PDF - 122.09 KB)
- Marius Thomas, Dieter Haering, Jun Li. Leveraging external data for efficient pediatric study design in multiple sclerosis (PDF - 628.85 KB)