Medical Devices
Public Workshop - Optimizing Clinical Trial Design for the Development of Pediatric Cardiovascular Devices, September 30th, 2010
Background
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.
Agenda
| Time | Presentation Title | Speaker | Affiliation |
|---|---|---|---|
| 8:00am | Welcome Remarks | Susan Cummins, M.D., M.P.H | Chief Pediatrics Medical Officer 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) CDRH FDA |
| 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) FDA |
| 8:40 | Brief Q&A | Ashley Boam (Moderator) | Chief, Interventional Cardiology Devices Branch (ICDB) CDRH FDA |
| 8:50 | Role for preclinical data in designing clinical trials | Matthew Hillebrenner | Chief, Circulatory Support and Prosthetics Branch (CSPB), CDRH FDA |
| 9:05 | When is a randomized clinical trial appropriate vs. historical control vs. performance goal? | Andrew Farb, M.D. | Medical Officer, DCD, ICDB CDRH FDA |
| 9:20 | BREAK | ||
| 9:35 | How can an RCT be accomplished in pediatric cardiology? | Kathy Jenkins, M.D., M.P.H | Pediatric Cardiology, Children’s Hospital Boston |
| 9:50 | What options exist to address the difficulties with randomizing to surgical controls? | John Laschinger, M.D. | Staff Fellow DCD CDRH FDA |
| 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 | |
| ***LUNCH*** | |||
| 12:20 | Benefits and Drawbacks of Different Clinical Trial Designs | Vandana Mukhi, Ph.D. | Division of Biostatistics, Office of Surveillance and Biometrics (OSB), CDRH, FDA |
| 12:35 | Bayesian and Small “n” trial designs | Gregory Campbell, Ph.D. | Director, Division of Biostatistics, OSB, CDRH, FDA |
| 12:50 | Kimberlee Gauvreau, Ph.D. | Children’s 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 CDRH, FDA |
| 1:30 | Lessons Learned from the Pediatric Heart Network’s Single Ventricle Reconstruction Trial | Richark Ohye, M.D. | University of Michigan |
| 2:05 | Lessons Learned from Investigator-run Trials | Richard Ringel, M.D. | Johns Hopkins Children’s Center |
| 2:20 | Standardization of endpoints | Eric Chen, M.S. | Director, HUD Devices OOPD FDA |
| 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, OSB, CDRH, FDA |
| 3:30 | The IMPACT Registry: Improving Pediatric and Adult Congenital Treatments | Gerard Martin, M.D. | Pediatric Cardiology, Children’s National Medical Center; Chair, IMPACT Steering Committee |
| 3:40 | The challenge of registries, historical and present in a pediatric device trial | Robert Kroslowitz | Berlin Heart, Inc. |
| 3:50 | CCISC database experience | 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 Children’s); Pam Simons (AGA Medical), Fred Tobia (NMT Medical), Tim Rynn (W.L. Gore), and above speakers | |
| 4:45-5:30 | WRAP UP discussion session | ||
Transcript
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
Email: francesca.joseph@fda.hhs.gov
