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Medical Devices

Public Workshop - Optimizing Clinical Trial Design for the Development of Pediatric Cardiovascular Devices, September 30th, 2010


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.

Date, Time and Location

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

Moscone Center, Room 102
747 Howard Street
San Francisco, CA, 94103

The meeting was not videotaped or webcasted.


TimePresentation TitleSpeakerAffiliation
8:00amWelcome RemarksSusan Cummins, M.D., M.P.HChief Pediatrics Medical Officer
Center for Devices and Radiological Health (CDRH)
U.S. Food & Drug Administration(FDA)
8:10Overview of pediatric device regulatory issuesHina Pinto, M.S.Biomedical Engineer, Scientific Reviewer
Division of Cardiovascular Devices (DCD)
8:20Types of funding/support available through NIH for pediatric device developmentJonathan Kaltman, M.D.Pediatric Cardiologist
NIH-NHLBI-Pediatric Heart Network
8:30Types of funding/support available through FDA/OOPD for pediatric device developmentFrancesca Joseph, M.D.Medical Officer,
Office of Orphan Products Development (OOPD)
8:40Brief Q&A Ashley Boam (Moderator)Chief,
Interventional Cardiology Devices Branch (ICDB)
8:50Role for preclinical data in designing clinical trialsMatthew HillebrennerChief,
Circulatory Support and Prosthetics Branch (CSPB),
9:05When is a randomized clinical trial appropriate vs. historical control vs. performance goal?Andrew Farb, M.D.Medical Officer,
9:35How can an RCT be accomplished in pediatric cardiology?Kathy Jenkins, M.D., M.P.HPediatric Cardiology,
Children’s Hospital Boston
9:50What options exist to address the difficulties with randomizing to surgical controls?John Laschinger, M.D.Staff Fellow
10:05REPEL-CV Adhesion Barrier trialMatthew Hillebrenner 
10:20Consider concomitant non-randomized surgical controlsLarry Latson, M.D.Pediatric Cardiology
Cleveland Clinic
10:35Establishing Performance GoalsZiyad Hijazi, M.D.  Pediatric Cardiology,
Rush University Medical Center
Richard Ringel, M.D.  Pediatric Cardiology
Johns Hopkins
Nusrath SultanaAGA Medical
11:05Q&A and Panel discussion  Panelists: Robert Beekman, M.D. (Moderator), Pamela Simons (AGA) and above speakers
12:20Benefits and Drawbacks of Different Clinical Trial DesignsVandana Mukhi, Ph.D.Division of Biostatistics, Office of Surveillance and Biometrics (OSB),
12:35Bayesian and Small “n” trial designsGregory Campbell, Ph.D.Director,
Division of Biostatistics, OSB,

Tips and tricks for maximizing data in small “n” trials

How to design small and efficient clinical trials

Kimberlee Gauvreau, Ph.D.  Children’s Hospital Boston
Donald Berry, Ph.D.  MD Anderson Cancer Center
1:10Q&A and Panel discussion  Panelists: Greg Campbell (Moderator) and above speakers 
1:40Outside US (OUS) DataBram Zuckerman, M.D.Director, DCD
1:30Lessons Learned from the Pediatric Heart Network’s Single Ventricle Reconstruction TrialRichark Ohye, M.D.University of Michigan
2:05Lessons Learned from Investigator-run TrialsRichard Ringel, M.D.Johns Hopkins Children’s Center
2:20Standardization of endpointsEric Chen, M.S.Director, HUD Devices
2:30Q&A and panel discussion  Panelists: Bram Zuckerman, M.D. (Moderator), Lynn Mahony, M.D. (UT Southwestern), and the above speakers
3:15Post-Market: FDA perspectiveHesha Duggirala, Ph.D.Division of Epidemiology,
3:30The IMPACT Registry: Improving Pediatric and Adult Congenital TreatmentsGerard Martin, M.D.Pediatric Cardiology,
Children’s National Medical Center;
Chair, IMPACT Steering Committee
3:40The challenge of registries, historical and present in a pediatric device trialRobert KroslowitzBerlin Heart, Inc.
3:50CCISC database experienceDonald Hagler, M.D.Pediatric Cardiology Mayo Clinic
4:00Q&A and Panel Discussion Panelists: Charlie Berul, M.D. (Moderator), Thomas Jones, M.D.(Seattle Children’s); Pam Simons (AGA Medical), Fred Tobia (NMT Medical), Tim Rynn (W.L. Gore), and above speakers
4:45-5:30WRAP UP discussion session


Contact Us

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

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