pediatric cardiac and vascular MRI coil

Overcoming real and perceived barriers to pediatric device innovation

pediatric cardiac and vascular MRI coil

A working group of innovators, engineers and clinicians from Children’s National Hospital and other institutions came together to address the real and perceived barriers to the creation of pediatric devices, such as this pediatric cardiac and vascular MRI coil developed by Inkspace Imaging.

A working group of innovators, engineers and clinicians from Children’s National Hospital, Children’s Hospital of Orange County and other institutions came together to address the real and perceived barriers to pediatric device innovation through opportunities that may change the return-on-investment in this market and improve health outcomes.

The new report, published in the Journal of Translational Engineering in Health and Medicine, mentions barriers that impede the advancement of pediatric devices, including excessive limitations for testing and validation, lack of incentives, inadequate research models and inconsistent pediatric-related knowledge among companies, regulatory experts, ethic review panels and government reviewers.

To remove the real and perceived barriers fostering excessive limitations, the researchers suggest reducing the perceived risk by establishing guidelines that standardize the review process and ethical research models, incentivizing small companies to participate in pediatric device innovation.

To increase incentives, the researchers call for the development of pediatric versions of adult devices with or without enforcement of regulations, develop incentives for small, medium and large-size companies, extend patent protection and more.

Since science corroborates the device’s effectiveness and safety, tailored pediatric research models are needed to help advance pediatric device innovation. While randomized trials in well-defined cohorts are commonly used in the scientific quest, they do not reflect the clinical practice in the pediatric devices field.

“Overcoming this barrier will require greater opportunity for creativity in the design of clinical trials, including delayed entry, intent-to-treat analysis, personalized outcome measures, and post-hoc subgroup analysis,” said Terence et al. “Effective research for pediatric device innovation will require greater ability to rely on ‘real-world’ data from post-market use of the proposed device or similar devices.”

On the education side, stakeholders, such as device sponsors and researchers, must receive an improved education on pediatric devices, according to the authors. Pediatricians should also be added to the regulatory review panels and advisory boards, so decision-makers can receive the pediatric perspective and fold it into their considerations.

“The consensus outcome of this meeting is that there are multiple opportunities, and a flexible combination of new programs and regulatory changes can be created to benefit the multiple stakeholders in pediatric device development,” said Terence et al. “An essential component will be building a cadre of experts with the development, regulatory, and clinical expertise to support all innovators.”

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National Hospital adds that supporting and expanding pediatric innovation is a key focus of the new Children’s National Research & Innovation Campus, the first-of-its-kind focused on pediatric health care innovation, with the first phase currently open on the former Walter Reed Army Medical Center campus in Washington, D.C. With its proximity to federal research institutions and agencies, universities, academic research centers, the campus provides a rich ecosystem of public and private partners which will help bolster pediatric innovation and commercialization.

Authors from Children’s Hospital Orange County include Terence Sanger, M.D., Nadine Afari, M.S., Anthony Chang, M.D., William Feaster, M.D., Sharief Taraman, M.D., Debra Beauregard, Brent Dethlefs, Tiffani Ghere, R.D., C.S.P., Mustafa Kabeer, M.D., and George Tolomiczenko.


New network will advance treatments for children


Three leaders from Children’s National Health System are among the investigators of a new FDA-funded program created to launch a global clinical trials network. The initial $1 million grant from the Food and Drug Administration (FDA) establishes a network among the Institute for Advanced Clinical Trials for Children (I-ACT for Children), the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) (affiliated with Children’s National), PEDSnet, the James M. Anderson Center for Health Systems Excellence and the Critical Path Institute, to address the unmet medical needs of children by improving quality and efficiency in developing innovative pediatric drugs and devices.

Along with the fiscal 2017 funds, there is a potential for $1 million in funding each year for an additional four years to I-ACT for Children, contingent on annual appropriations and the availability of funding. I-ACT for Children is a new independent, nonprofit organization that works to improve the planning and completion of pediatric clinical trials. PEDSnet and the Anderson Center will serve as the network’s data and learning core, while the Critical Path Institute will serve as the regulatory science core and NCC-PDI will serve as the medical device core.

From Children’s National, the investigators include: Peter Kim, M.D., Ph.D., vice president of the Sheikh Zayed Institute for Pediatric Surgical Innovation; Kolaleh Eskandanian, Ph.D., executive director of the Sheikh Zayed Institute and NCC-PDI and Johannes van den Anker, M.D., Ph.D., division chief of Clinical Pharmacology and vice chair of Experimental Therapeutics.

“We are pleased that this grant addresses innovative reengineering of the pediatric device trials system,” says Eskandanian. “In contrast with drug trials, device trials are generally less optimally understood in academic medical centers and clinical sites.”

She explains that children have medical device needs that are considerably different from adults. Designing devices for children requires considerations such as growth and development, anatomical and physiological differences. Often, the lack of available devices for children forces clinicians to use an adult device off-label or to improvise. Off-label use may be the only option, but such use can bring risks of serious adverse events that could be avoided if there were more FDA–approved pediatric devices.

“Thanks to partnership with I-ACT we will be able to address the pressing need to improve clinical trials and post-market monitoring of pediatric devices,” says Eskandanian.

Leading the network as principal investigator is Edward Connor, M.D., president of I-ACT for Children and an emeritus professor of Pediatrics, Microbiology, Immunology, and Tropical Medicine at George Washington University School of Medicine and Children’s National.

Work has been initiated to integrate network components and engage public and private shareholders. Next steps include selecting priority projects for implementation in 2018 and beyond, and scaling the network in North America and abroad.

Funding for this work was made possible, in part, by the Food and Drug Administration through grant 1 U18 FD 006297. Views expressed in written materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does any mention of trade names, commercial practices, or organization imply endorsement by the United States Government.