The Food and Drug Administration (FDA), with support from the American Academy of Pediatrics (AAP), the American College of Cardiology (ACC), Society for Cardiovascular Angiography and Interventions (SCAI), and the National Institutes of Health (NIH), is announcing a public Workshop on clinical trials for the development of pediatric cardiovascular devices.

Rarely have devices been developed, evaluated and approved specifically for treatment of children with congenital heart disease. The small, heterogeneous population, need for long-term follow-up, lack of market incentive, and misperceptions of regulatory requirements and costs are a few of the issues that make a standard randomized control trial difficult to conduct in pediatric cardiology. The purpose of the workshop is to solicit information from clinicians, academia, professional societies, other government agencies, and industry on various efficient and pragmatic clinical trial designs that are conducive to overcoming the challenges in developing devices for the pediatric cardiology patient population. The information gathered in this and future workshops will help to develop future guidance on optimal designs for pediatric cardiology device trials. The marketing approval of more cardiovascular devices specifically designed and/or labeled for pediatric patients would have a significant impact on public health.

Invited experts will address types of clinical trials with a particular focus on trial designs and statistical analysis methods, as well as alternative sources of clinical data, that can help to address the challenges in this particular patient population. After each section there will be an audience question and answer session and panel discussion allowing workshop participants to interact with the speakers and panelists. A concluding session will allow for additional interactions.

This meeting will be held September 30 th, 2010, beginning at 8:00 a.m. at the following location:

Moscone Center, Room 102

747 Howard Street

San Francisco, CA, 94103

The meeting will not be videotaped or webcasted.

Time Presentation Title Speaker Affiliation
8:00am Welcome Remarks CDRH Pediatrics Champion Center for Devices and Radiological Health (CDRH)

U.S. Food & Drug Administration(FDA)

8:10 Overview of pediatric device regulatory issues Hina Pinto, M.S. Biomedical Engineer, Scientific Reviewer

Division of Cardiovascular Devices (DCD)



8:20 Types of funding/support available through NIH for pediatric device development Jonathan Kaltman, M.D. Pediatric Cardiologist

NIH-NHLBI-Pediatric Heart Network

8:30 Types of funding/support available through FDA/OOPD for pediatric device development Francesca Joseph, M.D. Medical Officer,

Office of Orphan Products Development (OOPD)


8:40 Brief Q&A Ashley Boam (Moderator) Chief,

Interventional Cardiology Devices Branch (ICDB)



8:50 Role for preclinical data in designing clinical trials Matthew Hillebrenner Chief,

Circulatory Support and Prosthetics Branch (CSPB),



9:05 When is a randomized clinical trial appropriate vs. historical control vs. performance goal? Andrew Farb, M.D. Medical Officer,




9:20 BREAK
9:35 How can an RCT be accomplished in pediatric cardiology? Kathy Jenkins, M.D., M.P.H Pediatric Cardiology,

Childrens Hospital Boston

9:50 What options exist to address the difficulties with randomizing to surgical controls? John Laschinger, M.D. Staff Fellow




10:05 REPEL-CV Adhesion Barrier trial Matthew Hillebrenner  
10:20 Consider concomitant non-randomized surgical controls Larry Latson, M.D. Pediatric Cardiology

Cleveland Clinic

10:35 Establishing Performance Goals Ziyad Hijazi, M.D.   Pediatric Cardiology,

Rush University Medical Center

Richard Ringel, M.D.   Pediatric Cardiology

Johns Hopkins

Nusrath Sultana AGA Medical
11:05 Q&A and Panel discussion Panelists: Robert Beekman, M.D. (Moderator), Pamela Simons (AGA) and above speakers
12:20 Advantages and disadvantages of randomized, non-randomized, historical control and single-arm trial designs Vandana Mukhi, Ph.D. Division of Biostatistics, Office of Surveillance and Biometrics (OSB),



12:35 Bayesian and Small "n" trial designs Gregory Campbell, Ph.D. Director,

Division of Biostatistics, OSB,



12:50 Tips and tricks for maximizing data in small "n" trials Kimberlee Gauvreau, Ph.D.   Childrens Hospital Boston
Donald Berry, Ph.D.   MD Anderson Cancer Center
1:10 Q&A and Panel discussion   Panelists: Greg Campbell (Moderator) and above speakers  
1:40 Outside US (OUS) Data Bram Zuckerman, M.D. Director, DCD



1:50 Lessons learned from investigator-run trials Richard Ringel, M.D.   Johns Hopkins
Richard Ohye, M.D. Pediatric Cardiac Surgery,

University of Michigan

2:20 Standardization of endpoints/definitions Eric Chen, M.S. Director, HUD Devices



2:30 Q&A and panel discussion Panelists: Bram Zuckerman, M.D. (Moderator), Lynn Mahony, M.D. (UT Southwestern), and the above speakers
3:00 BREAK
3:15 Post-Market: FDA perspective Hesha Duggirala, Ph.D. Division of Epidemiology,




3:30 IMPACT Gerard Martin, M.D. Pediatric Cardiology,

Childrens National Medical Center;

Chair, IMPACT Steering Committee

3:40 Berlin Heart Robert Kroslowitz Berlin Heart, Inc.
3:50 CCISC Donald Hagler, M.D. Pediatric Cardiology Mayo Clinic
4:00 Q&A and Panel Discussion Panelists: Charlie Berul, M.D. (Moderator), Thomas Jones, M.D.(Seattle Childrens); Industry perspective (AGA Medical, NMT Medical, W.L. Gore), and above speakers
4:45-5:30 WRAP UP discussion session

If you wish to attend this Workshop, you must register by close of business on September 16th, 2010.

There is no fee to register for the Workshop and registration will be on a first-come, first-served basis. Early registration is recommended because seating is limited.

Public comments will be permitted and encouraged during any of the open comment discussion sessions at the meeting.

If you need special accommodations due to a disability please contact Lynn Colegrove, AAP, at 847-434-7820. If you need additional information regarding registration, please contact Francesca Joseph, M.D., Office of Orphan Products Development, Office of the Commissioner, Food and Drug Administration, 10903 New Hampshire Avenue, Bldg. 32, Silver Spring, MD 20993, 301-796-6805, FAX: 301-847-8621,

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For further information about this meeting, contact:

Francesca Joseph

Food and Drug Administration

Office of Orphan Products Development

10903 New Hampshire Avenue, Bldg. 32, Rm. 5277

Silver Spring, MD 20993

Phone: 301-796-6805