The Heart Valve Collaboratory (HVC) is national, multidisciplinary group that brings together clinicians, scientists, industry partners, and regulators to tackle complex problems in the management of adult and pediatric heart valve disease. The group, which is co-chaired by Martin B. Leon, MD, an interventional cardiologist, chief innovation officer, and the director of the Cardiovascular Data Science Center for the Division of Cardiology at NewYork-Presbyterian and Columbia, convened over two days to discuss unmet needs for children with acquired and congenital heart valve disease.
The HVC recently published a white paper in JACC: Advances identifying several challenges in pediatric valvar diseases, from valve durability to delivery system size in transcatheter interventions to regulatory challenges that have contributed to mixed clinical outcomes for children. Oliver M. Barry, MD, a pediatric interventional cardiologist who was recently named the site director for the pediatric catheterization lab and program at NewYork-Presbyterian and Weill Cornell Medicine, and Andrew Goldstone, MD, PhD, a pediatric cardiothoracic surgeon, surgical director of pediatric heart transplantation, and surgical director of pediatric heart valve transplantation at NewYork-Presbyterian and Columbia, were two of the authors on a new white paper addressing those specific pitfalls and potential solutions.
Below, Dr. Barry discusses the unique challenges faced in the development of technology to manage pediatric valvar heart disease and the “call to arms” for more collaboration and regulatory flexibility.
One of the major challenges in pediatric valve surgery is that the heart valve doesn’t grow with the child.
Unique Challenges
In the U.S., it is estimated that there are more than 130,000 pediatric patients living with pulmonary valve disease. One of the long-standing challenges in developing heart valve technology for children is growth potential. A child in need of a heart valve replacement is growing but the currently available valves are too big for our smaller patients, and they don’t grow with them. However, there is a lot of momentum in this area. Our team at NewYork-Presbyterian and Columbia is involved in a first-in-human clinical trial for a novel pulmonary valve replacement known as the Autus Size-Adjustable Valve, which can be expanded as the child grows using minimally invasive techniques. I’m particularly excited about the idea of evolving these technologies to allow the valves to be placed with cardiac catheterization instead of open-heart surgery.
Resistant valve tissue technology and innovative valve materials and designs are potential solutions that we hope can be developed in the future.
— Dr. Oliver Barry
Perhaps the more difficult issues to address are the durability of valves, which are variable, and the risk of endocarditis and thrombosis. Currently, if one of these valves gets infected, children can get very sick, and we don’t have a great way of preventing that. Resistant valve tissue technology and innovative valve materials and designs are potential solutions that we hope can be developed in the future.
Developmental Hurdles
The smaller number of children with valvar disease compared to adults creates economic barriers to developing pediatric valve technology. The economic incentives are different, and the testing and development can take longer because of the smaller population, adding to the overall costs.
There are also some regulatory processes that make development of pediatric technologies cumbersome, from the engineering process for how the technology is tested to the regulatory benchmarks needed for approval from the U.S. Food and Drug Administration (FDA).
Despite the excellent clinical work in pediatric valvar heart disease, it remains a challenging disease to treat. But I am hopeful that the HVC’s work in identifying unmet needs will spur action toward accomplishing our shared goal of improving care for children with valvar heart disease.
— Dr. Oliver Barry
Call to Action
This white paper is a call to arms to motivate the various stakeholders – pediatric cardiologists, surgeons, industry members, and regulatory agencies – to collaborate and find innovative solutions for our patients. One potential area of collaboration is on the creation of a national pediatric heart valve registry that would allow researchers to pool data and outcomes. FDA officials have been open to that type of collaboration in the past and having a registry could offer some advantages in getting technology through the regulatory process. Despite the excellent clinical work in pediatric valvar heart disease, it remains a challenging disease to treat. But I am hopeful that the HVC’s work in identifying unmet needs will spur action toward accomplishing our shared goal of improving care for children with valvar heart disease.
