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U.S. Department of Health and Human Services

Medical Devices

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Public Workshop - Using Scientific Research Data to Support Pediatric Medical Device Claims, December 5, 2011

The Docket for Public Comment has been reopened and extended to March 5, 2012 to provide additional time for submissions.

The workshop transcript and meeting summary of breakout sessions is now available.

The Food and Drug Administration (FDA) held a public workshop entitled: “Using Scientific Research Data to Support Pediatric Medical Device Claims: A Public Dialogue.” The purpose of the public workshop was to receive public comment on the use of scientific research data, including published scientific literature, to extrapolate effectiveness claims from adults to children and between pediatric subpopulations in order to support and establish pediatric indications for medical devices.

The topics discussed were the ways scientific research data can be used to support pediatric effectiveness claims for medical devices and pediatric device approvals or clearance; the scientific and regulatory limitations and issues of using existing scientific research data to support pediatric effectiveness claims and pediatric indication approvals for medical devices; and methods to overcome the pitfalls and data gaps, including statistical approaches and modeling.

This workshop supports FDA’s efforts to define pathways for approving pediatric device indications by leveraging available scientific research data. An important, but not the only focus, was a discussion of how to determine when it is appropriate to use, and how to use, existing scientific research data, including that in published scientific research, to determine pediatric effectiveness based on a similar course of a disease or condition or a similar effect of a device on adults and similar extrapolation between pediatric subpopulations. We are looking to establish a framework to support the establishment of pediatric effectiveness claims.


Date, Time and Location

This workshop was held December 5, 2011 from 8:30 am to 5:00 p.m. EST at:

FDA White Oak Campus
10903 New Hampshire Ave
The Great Room (Room 1503), White Oak Conference Center, Bldg 31
Silver Spring, MD, 20903

Background

The demand by health care professionals and consumers for safe and effective pediatric medical devices continues to steadily increase. Pediatric medical devices prevent, treat or diagnose diseases and conditions occurring from birth through the 21st year of life, in the pediatric spectrum of infancy, childhood and adolescence. Some devices are designed specifically for pediatric use, while others are adopted from specific adult device applications or produced for more general use.

Childhood, from before birth through adolescence, is characterized by a rapid biologically determined developmental trajectory during which the child undergoes predictable change in every body system. Designing pediatric medical devices can be challenging: children are often smaller and more active than adults, body structures and functions grow and evolve throughout childhood, and children may be long-term device users - raising concerns about device longevity and the impact of long-term exposure to implanted materials. The current medical device market for children has a higher demand than supply. FDA is committed to supporting the development and availability of safe and effective pediatric medical devices.

In 2007 Congress passed the Pediatric Medical Device Safety and Improvement Act. One approach that the Act addresses pediatric device needs is by allowing for a pediatric device approval pathway that permits extrapolation of adult effectiveness data to support a pediatric indication based on similar course of the disease or condition or a similar effect of the device.

Extrapolation of effectiveness across populations (for example, from adults to pediatric patients) simplifies the requirements for establishing a pediatric intended use claim. It reduces the need for and complexity of clinical efficacy studies to establish pediatric claims. This facilitates efforts to address the unmet medical device need for children by making optimal use of what is already known to increase efficiency, and reducing the regulatory burden. Due to the unique safety issues of the pediatrics population, extrapolation of safety is not authorized.

Through this effort, FDA and stakeholders will take steps to increase awareness of a path for approval of pediatric devices that uses extrapolation of existing scientific research data, including from clinical trials or scientific literature. FDA will advance this goal through collaboration with medical device and health care industries, and the healthcare provider, academic and consumer communities.

Additional background about the Pediatric Medical Device Safety and Improvement Act and pediatric medical devices is available.

Workshop Details

Workshop Format

This workshop was structured as plenary overview presentations and topic-focused interactive breakout sessions, intended to foster constructive dialogue between stakeholders with diverse perspectives. Moderators of each breakout group will summarize each group’s observations for presentation to all participants.

Written or electronic comments

In order to permit the widest possible opportunity to obtain public comment, FDA is soliciting written or electronic comments on the workshop topics. Regardless of attendance at the public workshop, interested persons may submit written or electronic comments. Submit electronic comments to http://www.regulations.gov. Submit written comments to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. Duplicate submissions are not required. The deadline for submitting comments related to this public workshop will be reopened in January 2012 and extended to March 5, 2012. This website will be updated when the docket is reopened. Please identify comments with the docket number FDA-2011-N-0754, and if responding to specific topics, please identify the topic you are addressing. Received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday and will be posted to the docket at http://www.regulations.gov.

Agenda

Time Topic
8:30- 8:40 AM Plenary Session: Welcome: Structure of the Day and Logistics
8:40 - 9:00 AM Plenary Session: Overview on the use of Extrapolated Data to establish Pediatric Device Effectiveness Presentation: Susan Cummings
9:00 - 9:30 AM Plenary Session: Topic 1: Defining the Useful Research Data Landscape Presentation: MaryBeth Richey
9:30 - 10:30AM Breakout Session: Topic 1: Defining the Useful Research Data Landscape. Focus Questions: What are the sources of available and useful research data? How might this data be used to establish effectiveness for the pediatric patients or subsets of them? What factors should the FDA consider in using available data sources to establish effectiveness claims?
10:30 - 10:45 AM Break
10:45 - 11:30 AM Plenary Session: Moderators report back from Breakout Session on Topic 1
11:30 AM - 12:30 PM Lunch Break
12:30 - 12:45 PM Plenary Session: Topic 2 : Defining the scientific and regulatory challenges and limitations with use of existing research data and published literature, Presentations: Joy Samuels-ReidMarkham Luke
12:45 - 1:45 PM Breakout Session: Topic 2: Defining scientific and regulatory the challenges and limitations and with the use of existing research data and published literature. Focus Question: Considering the landscape of data sources identified in Breakout Session #1, what are the scientific and regulatory challenges and limitations the FDA must consider when using these data to extrapolate or establish pediatric effectiveness for various medical devices?
1:45 - 2:00 PM Break
2:00 - 2:30 PM Plenary Session: Report back from Breakout Session on Topic 2
2:30 -3:30 PM Breakout Session #3: Overcoming the challenges and limitations: Identifying methods to address the pitfalls and data gaps , including statistical approaches and modeling. Focus Question: Considering the challenges and limitations identified in Breakout Session #2, how might the FDA address these challenges and limitations? How might statistical modeling and limited clinical or other research address these challenges and limitations? Presentations: Patricia Beason, Laura Thompson
3:30 - 4:00 PM Plenary Session: Report back from Breakout Session on Topic 3
4:00 - 5:00 PM Closing Plenary Session: Summary of Dialogue and the Road Ahead.
5:00 PM Adjournment

Transcripts and Archived Webcast

A transcript is accessible at http://www.regulations.gov. It may be viewed at the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD. A transcript will also be available in either hardcopy or on CD-ROM, after submission of a Freedom of Information request. Written requests are to be sent to Division of Freedom of Information (HFI-35), Office of Management Programs, Food and Drug Administration, 5600 Fishers Lane, rm. 6-30, Rockville, MD 20857.

An online transcript is currently available:

Archived webcast is currently available:

Meeting Summary

Contacts for Additional Information

For information regarding the program, contact:

Carol Krueger
Office of the Center Director
Center for Devices and Radiological Health
Food and Drug Administration
10903 New Hampshire Avenue
Bldg 66, Room 5437
Silver Spring , MD 20993
Phone: 301-796-3241
Email: Carol.Krueger@fda.hhs.gov