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(Circulation. 2000;101:e9042.)
© 2000 American Heart Association, Inc.

Cardiovascular News

First Food and Drug Administration Approval Under Humanitarian Device Exemption of a Septal Occluder for Fenestrated Fontan and Muscular Ventricular Septal Defects

Carlos E. Ruiz, MD, PhD; E. H. Austin, III, MD; J. P. Cheatham, MD; D. A. Danford, MD; L. A. Latson, MD; J. Mayer, MD; C. E. Mullins, MD; S. Sanders, MD

From the Department of Pediatric Interventional Cardiology, Rush-Presbyterian–St. Luke’s Medical Center, Chicago, Ill.

Correspondence to Carlos E. Ruiz, MD, PhD, Department of Pediatric Interventional Cardiology, Rush-Presbyterian–St. Luke’s Medical Center, 1653 West Congress Parkway, Room 736MU, Chicago, IL 60612-3833.

The difficult process of obtaining Food and Drug Administration (FDA) approval for pediatric-specific devices and drugs is one of the most important issues confronting Pediatric Cardiology (and Pediatrics in general). It has forced our profession to continually administer drugs and implant devices "off-label" (for purposes and age groups other than those for which they were tested and approved). Consequently, it is possible that important risk factors in the pediatric population, which has far different dispositions than the adult population, may be overlooked. This problem has deteriorated further as the pace of technological change has continued to accelerate and our ability to distribute clinical results has improved.

Accordingly, a group from the Society for Cardiac Angiography and Interventions, Pediatric Section, together with representatives from the American Heart Association, the American College of Cardiology, the American Academy of Pediatrics, the Society of Thoracic Surgeons, and the American Association of Thoracic Surgeons, is working with the FDA to find a solution for this critical problem. This group has met several times and has proposed the formulation of "objective performance criteria." These will be the minimum standard criteria required to establish the safety of any pediatric product for FDA approval. Because Pediatric Interventional Cardiologists deal with a smaller volume of patients with a broader spectrum of disease than our Adult Cardiology colleagues, our ability to use the same study designs and outcome measures as trials in adult patients is restricted. The research and development costs for manufacturers of pediatric products may exceed market returns due to the small and diverse patient population. The goal, therefore, is to encourage industry to manufacture pediatric cardiovascular devices, while ensuring the highest safety and efficacy standards, without the burdens of traditional clinical trials.

The FDA has also been working more closely with industry to achieve disease-specific approvals. This effort is most recently represented by the FDA approval of the CardioSEAL Septal Occluder under Humanitarian Device Exemption regulations. The CardioSEAL is now approved for use in patients with a fenestrated Fontan defect or muscular and postoperative residual ventricular septal defect that requires closure because of an increased risk for open-heart surgery.

These events demonstrate the new cooperative nature of the emerging relationship between physicians, the FDA, and industry to address specific pediatric problems, a relationship that we hope will continue to evolve. However, the first significant step has been accomplished.

Nonetheless, it is appropriate at this time to congratulate and thank those within our profession who have given so much of their time and effort to this project. We would especially like to thank the FDA and industry for their efforts to find a common ground for legitimately bringing life-saving pediatric products to our pediatric patients. We look forward to reporting the future progress of our joint effort to provide appropriate medical devices for pediatric patients worldwide.

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