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New network will advance treatments for children

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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.

$250K awarded to six winners presenting innovative pediatric medical devices

SZI Symposium Winners

Six companies presenting innovative medical device solutions that address significant unmet needs in pediatric health were awarded a total of $250,000 in grant money yesterday in San Jose, Calif. at the Fifth Annual Pediatric Device Innovation Symposium, organized by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Health System.

The “Make Your Medical Device Pitch for Kids!” competition is sponsored by the National Capital Consortium for Pediatric Device Innovation (NCC-PDI), an FDA-funded consortium led by Children’s National and the A. James Clark School of Engineering at the University of Maryland. Four companies were awarded $50,000 each and two were awarded $25,000. The six winners were selected from a field of twelve finalists. A record 98 total submissions from five countries were received for the competition this year.

“To improve care for children, it is imperative that we recognize and encourage relevant new solutions in pediatric medical devices, especially in light of the challenges innovators face in addressing this specialized market,” said Kurt Newman, M.D., president and CEO of Children’s National. “Children’s National is committed to fostering collaboration among innovators, clinicians, policy makers and investors to advance pediatric device development for the benefit of children everywhere.”

This year’s winning innovations receiving $50,000 awards are:

  • CorInnova, Houston, Texas – soft robotic, non-blood-contacting biventricular cardiac assist device for the treatment of heart failure in children
  • Green Sun Medical, Fort Collins, Colo. – novel device that provides necessary pressure for the correction of spinal deformity while providing real-time feedback to clinicians
  • Hub Hygiene and Georgia Institute of Technology, Atlanta, Ga. – low-cost, single-use cleaning technology to prevent central line-associated blood stream infections (CLABSI), a hospital-acquired infection by pediatric ICU patients
  • NAVi Medical Technologies, Houston, Texas – device to provide accurate information about the localization of an umbilical venous catheter (UVC) used in critically-ill newborns to reduce the risk of catheter malposition

Winning innovations receiving $25,000 awards are:

  • Prapela, LLC, Boston, Mass. – novel “baby box” that will allow for a non-pharmacological approach to help drug-exposed infants relax and sleep during withdrawal and post-withdrawal care
  • X-Biomedical, Inc., Philadelphia, Pa. – portable surgical microscope for use in surgeries for treatable causes of blindness in low-income countries and under-resourced setting

“We are honored to recognize these outstanding innovations with this funding,” said Kolaleh Eskandanian, Ph.D., executive director of the Sheikh Zayed Institute and NCC-PDI. “We are even more excited about welcoming this new cohort of companies to our family of pediatric device startups and entrepreneurs. Together we can move the needle a bit faster and safer to bring pediatric products to market.”

She added that in addition to the financial support and consultation services through NCC-PDI, the awardees can leverage the validation received through this highly competitive process to raise the additional capital needed for commercialization. Since inception in 2013, NCC-PDI has supported 67 pediatric devices and the companies and research labs owning these devices have collectively raised $55 million in additional funding.

The twelve finalists each made five-minute presentations to the symposium audience and then responded to judges’ questions. Finalists also included Anecare, LLC, Salt Lake City, Utah; ApnoSystems, Buenos Aires, Argentina; Deton Corp., Pasadena, Calif.; Kite Medical, Dublin, Ireland; Moyarta 2, LLC, The Plains, Va.; and Oculogica, Inc., New York, N.Y.

Serving on the distinguished panel of judges were Susan Alpert, M.D., of SFA Consulting, a former director of the FDA Office of Device Evaluation and former senior vice president and chief regulatory officer of Medtronic; Charles Berul, M.D., co-director, Children’s National Heart Institute; Andrew Elbardissi, M.D., of Deerfield Management; Rick Greenwald, Ph.D., of the New England Pediatric Device Consortium (NEPDC); James Love, J.D., of Oblon; Josh Makower, M.D., of NEA; Jennifer McCaney, Ph.D., of MedTech Innovator; Jackie Phillips, M.D., of Johnson & Johnson; and Tracy Warren of Astarte Ventures.

The pitch competition is a highlight of the annual symposium organized by the Sheikh Zayed Institute at Children’s National, designed to foster innovation that will advance pediatric healthcare and address the unmet surgical and medical device needs for children. New this year, the symposium co-located in a joint effort with The MedTech Conference powered by AdvaMed, the premier gathering of medtech professionals in North America.

Keynote speakers at the event included Daniel Kraft, M.D., faculty chair of Medicine & Neuroscience, Singularity University and executive director, Exponential Medicine; Vasum Peiris, M.D., chief medical officer, Pediatrics and Special Populations, FDA;  and Alan Flake, M.D., director of Center for Fetal Research, Children’s Hospital of Philadelphia.

Panel discussions focused on gap funding for pediatric innovation, the journey from ideation to commercialization, and the pediatric device needs assessment in the future regulatory environment.

It took an act of Congress to save lives

Boosting research and innovation to find cures and develop new medical devices for children and adults who carry childhood and rare diseases will transform our health system and save lives.

Until now, medical research and innovation have been severely limited in the U.S. by regulations and lack of funding. On behalf of healthcare systems and medical innovators across the U.S., we applaud the House and Senate for their tremendous bipartisan effort to pass the 21st Century Cures Act that will transform our health and research system and enable us to more effectively fight diseases.

We are encouraged by the provisions in the act that break down regulatory barriers and expedite the approvals of drugs and devices. We are particularly excited about the provisions to increase funding to the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), as well as the establishment of precision medicine, the cancer moonshot initiatives and new programs that will improve our mental health system and fight the worsening opioid epidemic. Boosting research and innovation to find cures and develop new medical devices for children and adults who carry childhood and rare diseases is at the core of our mission at Children’s National. Our researchers are working to find new biomarkers, map the human genome, develop medical devices for children and personalize medicine to make treatment and cures more targeted and effective. They are also studying pain and looking at new ways to detect the presence of opioids and cannabinoids. Thanks in large part to funding from the NIH, institutions like ours are able to continue groundbreaking biomedical research. This legislation brings hope to our children and their families, especially those who volunteer to participate in research, that our scientific breakthroughs will be translated to drugs, therapeutics and medical devices safer and faster.

Another victory for all of us in the pediatric medical device field is the expansion of the Humanitarian Use Device program to include devices used by up to 8,000 individuals rather than the current 4,000 individual cap. The hard cap at 4,000 individuals was excessively restrictive and was a significant disincentive blocking the development of devices for rare diseases and conditions, especially those affecting children. The 4,000 limit was also an obstacle for the development of diagnostic devices, since the FDA interprets the limitation to apply to the number of patients that would receive the diagnostic test, rather than the number of individuals affected or manifesting the rare disease.

Currently, medical device development for children lags woefully behind adults. Children have medical device needs that are considerably different from adults. The subtleties of developing devices for pediatric patients are fundamentally different than those for adults. The challenges include small markets, scarce financial incentives, regulatory issues, and the procedural dissimilarities of premarket clinical trials and post-market surveillance. The lack of available pediatric devices often forces clinicians to treat pediatric patients by using or modifying adult devices, adjusting implants designed for other purposes, and using implants designed decades ago. Because devices are being used “off-label,” clinicians and regulators are not able to collect information on their effectiveness. This act promises a faster regulatory approval process, which increases the enthusiasm of the venture community in investing in drug and device development, which in turn can help startup companies in the field secure private capital.

Thank you to everyone who worked tirelessly to create this bill and to those who lobbied on its behalf. It’s efforts like the 21st Century Cures Act, that break down regulatory barriers and provide the resources to expedite the approvals of life-saving drugs and devices, that save children’s lives.

About the Author

Kolaleh Eskandanian, Ph.D.
Executive Director
Sheikh Zayed Institute for Pediatric Surgical Innovation
Research interests: device development, entrepreneurship, innovation in health care