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  1. Advancing Regulatory Science

Creating a Framework for a National Adaptive Platform Trial to Evaluate Pediatric Medical Devices

CERSI Collaborators: Christopher Almond, MD, MPH (Stanford; Principal Investigator); Selena Gonzales, MPH (Stanford)

FDA Collaborators: Vasum Peiris, MD, MPH (CDRH; Principal Investigator); Nicole Ibrahim, PhD (CDRH/OPEQ/OHT2); Nicole Gillette, (CDRH/OPEQ/OHT2); Minerva Hughes, JD, PhD (CDRH/OCEA, posthumously); Erika Torjusen, MD, MHS (FDA/OC/OCPP/OOPD)

CERSI Subcontractors: Roger Lewis, MD, PhD (UCLA); Juliana Tolles, MD, MPH (UCLA)

Project Start Date: September 1, 2020

Regulatory Science Challenge

Medical device development and innovation for pediatric populations lags far behind adult populations. Many devices, including life-saving devices, indicated for adult populations are adapted, and, by necessity, used in pediatric care as directed by clinicians. Without the benefit of evidence generated to support the labeled indications, many pediatric patients are routinely cared for with devices whose safety and effectiveness for their care has not been evaluated by the U.S. Food and Drug Administration (FDA). By developing more efficient and cost-effective methods to generate basic safety and effectiveness information regarding medical devices indicated for pediatric populations, investigators will take a critical step toward lowering the barriers for pediatric device development, improving safe use, and addressing this long-standing public health concern.

One possible solution is to use a newer type of clinical trial design called the ‘adaptive platform trial’ (APT), which has the potential to be more efficient, less costly, and safer for human subjects compared to traditional study methods. While APTs have been endorsed by statistical societies and reviewed by the FDA, they have limited acceptance by institutional review boards and limited use by device developers. This CERSI research project seeks to improve understanding of APTs as an option for pediatric medical device development.

Project Description and Goals

The goals of this project are to clarify and summarize the potential benefits of conducting APTs for pediatric medical device development, discuss the challenges that need to be addressed to conduct successful APTs for children, and design a real-world example of an APT involving a pediatric medical device. The broader goal is to generate a guide to help pediatric clinicians, device developers, and other stakeholders assess how APTs may facilitate pediatric device development.

Developing an APT requires multiple steps, where each step is dependent on the last. Thus, this project will go through the following phases:

Step 1: With input from researchers around the country and the FDA, investigators will review existing publications and evidence to identify the advantages and challenges of using APT principles and infrastructure for pediatric medical device evaluation compared to conventional trial designs.

Step 2: Investigators will conduct an overall assessment and address the challenges associated with using APTs for pediatric device evaluation.

Step 3: Finally, investigators will collaborate with pediatric clinical and trial experts at children’s hospitals to apply their findings to create a compelling illustration of an adaptive platform trial using a real-world example drawn from pediatric heart failure practice.

A manuscript of the findings from this project has been developed for potential publication in an academic or scientific peer-reviewed journal. The manuscript is currently undergoing review and editing prior to potential submission to a journal for publication.

 

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