Tag Archive for: Francesca Joseph

Drs. Packer and van den Acker at the Pediatric Device Innovators Forum

Pediatric Device Innovators Forum explores state of focused ultrasound

For children living with pediatric tumors, less invasive and less painful treatment with no radiation exposure was not always possible. In recent years, the development of technologies like Magnetic resonance guided high intensity focused ultrasound (MR-HIFU) and Low intensity transcranial focused ultrasound (LIFU) is helping to reverse that trend.

This topic was the focus of the recent Pediatric Device Innovators Forum (PDIF) hosted by the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) in partnership with the U.S. Food and Drug Administration’s (FDA) Pediatric Device Consortia (PDC) grant program. A collaboration between Children’s National Hospital and University of Maryland Fischell Institute for Biomedical Devices, NCC-PDI is one of five PDCs funded by the FDA to support pediatric device innovators in bringing more medical devices to market for children.

The discussion, moderated by Kolaleh Eskandanian, Ph.D., MBA, PMP, vice president and chief innovation officer at Children’s National and principal investigator of NCC-PDI, explored the use of focused ultrasound’s noninvasive therapeutic technology for two pediatric indications, Osteoid Osteoma (OO) and Diffuse Intrinsic Pontine Glioma (DIPG), and the ways it can increase the quality of life for pediatric patients while also decreasing the cost of care.

The discussion also examined the most common barriers preventing more widespread implementation of focused ultrasound technology, specifically small sample size for evidence generation, lack of funding opportunities and reimbursement issues that can make or break a technology’s chances at reaching the patients that need it.

Karun Sharma, M.D., director of Interventional Radiology at Children’s National, emphasized the potential for focused ultrasound to treat localized pain relief and treat other diseases that, like OO, do not have any other therapeutic alternative

“At Children’s National, we use MR-HIFU to focus an ultrasound beam into lesions, usually tumors of the bone and soft tissues, to heat and destroy the harmful tissue in that region, eliminating the need for incisions,” says Sharma. “In 2015, Children’s National doctors became the first in the U.S. to use MR-HIFU to treat pediatric osteoid osteoma (OO), a painful, but benign, bone tumor that commonly occurs in children and young adults. The trial demonstrated early success in establishing the safety and feasibility of noninvasive MR-HIFU in children as an alternative to current, more invasive approaches to treat these tumors.”

In November 2020, the FDA approved this MR-HIFU system to treat OO in pediatric patients.

Roger Packer, M.D., senior vice president of the Center for Neuroscience and Behavioral Medicine at Children’s National, also discussed how focused ultrasound, specifically LIFU, has also proven to be an attractive modality for its ability to non-invasively, focally and temporarily disrupt the blood brain barrier (BBB) to allow therapies to reach tumors that, until recently, would have been considered unreachable without severe intervention.

“This presents an opportunity in pediatric care to treat conditions like Diffuse Intrinsic Pontine Glioma (DIPG), a highly aggressive brain tumor that typically causes death and morbidity,” says Packer.

Packer is planning a clinical trial protocol to investigate the safety and efficacy of LIFU for this pediatric indication.

The forum also featured insight from Jessica Foley, M.D., chief scientific officer, Focused Ultrasound Foundation; Arjun Desai, M.D., chief strategic innovation officer, Insighttec; Arun Menawat, M.D., chairman and CEO, Profound Medical; Francesca Joseph, M.D., Children’s National; Johannes N. van den Anker, M.D., Ph.D., vice chair of Experimental Therapeutics, Children’s National; Gordon Schatz, president, Schatz Reimbursement Strategies; Mary Daymont, vice president of Revenue Cycle and Care Management, Children’s National; and Michael Anderson, MD, MBA, FAAP, FCCM, FAARC, senior advisor to US Department of Health and Human Services (HHS/ASPR) and Children’s National.

Anthony Sandler, M.D., senior vice president and surgeon-in-chief of the Joseph E. Robert Jr. Center for Surgical Care and director of the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital, and Sally Allain, regional head of Johnson & Johnson Innovation, JLABS @ Washington, DC, opened the forum by reinforcing both organizations’ commitment to improving pediatric health.

In September 2020, the Focused Ultrasound Foundation designated Children’s National Hospital as the first global pediatric Center of Excellence for using this technology to help patients with specific types of childhood tumors. As a designated COE, Children’s National has the necessary infrastructure to support the ongoing use of this technology, especially for carrying out future pediatric clinical trials. This infrastructure includes an ethics committee familiar with focused ultrasound, a robust clinical trials research support team, a data review committee for ongoing safety monitoring and annual safety reviews, and a scientific review committee for protocol evaluation.

The Pediatric Device Innovators Forum is a recurring collaborative educational experience designed by the FDA-supported pediatric device consortia to connect and foster synergy among innovators across the technology development ecosystem interested in pediatric medical device development. Each forum is hosted by one of the five consortia. This hybrid event took place at the new Children’s National Research and Innovation Campus, the first-of-its-kind focused on pediatric health care innovation, on the former Walter Reed Army Medical Center campus in Washington, D.C.

To view the latest edition of the forum, visit the NCC-PDI website.

Panelists at the Pediatric Device Innovators Forum

The recent Pediatric Device Innovators Forum (PDIF) exploring the state of focused ultrasound was held at the new Children’s National Research and Innovation Campus, a first-of-its-kind focused on pediatric health care innovation.

Dr. Kurt Newman in front of the capitol building

Children’s National leaders provide expertise and support to advance SHIP-MD pediatric innovation initiative

Dr. Kurt Newman in front of the capitol building

“Having spent 30 years on the frontlines of pediatric healthcare as a surgeon, I saw so much innovation focused on adult medicine and not on pediatric populations. Instead, we were trying to adapt adult devices for use in children, which is not an effective solution,” says Dr. Newman.

The advancement of children’s medical devices in the U.S. continues to significantly lag behind adult devices for many reasons. A dedicated group of public and private sector healthcare leaders are working together to change that trend. In culmination of its first stage of work, the System of Hospitals for Innovation in Pediatrics – Medical Devices (SHIP-MD) initiative recently held a dynamic 3-day public workshop to further develop this groundbreaking public-private partnership, which is currently in its pre-consortium/conceptual phase.

Children’s National leaders and clinicians were among the pediatric healthcare experts who contributed to robust discussions about how to build and nurture a public-private partnership system that will safely accelerate the advancement of pediatric medical devices.

The workshop was developed and guided by a multi-stakeholder group including the Critical Path Institute (C-Path), the U.S. Food and Drug Administration’s (FDA) Center for Devices and Radiological Health (CDRH), AdvaMed, the American Academy of Pediatrics (AAP) and leaders of pediatric health systems.

Lee Beers

“We must strive to improve medical devices for children, which historically lag five to 10 years behind adults. For many children, that can be a lifetime,” says Dr. Beers.

Reflecting its ongoing commitment to bridging the pediatric innovation gap, Children’s National Hospital experts co-led discussions throughout the program, which explored ways to improve children’s health by transforming the existing medical device ecosystem to stimulate investment and innovation in pediatric devices.

Children’s National Hospital President and CEO Kurt Newman, M.D., and Lee Beers, M.D., medical director for the Child Health Advocacy Institute at Children’s National Hospital served as opening session speakers, providing their insights into the current state of innovation in pediatric devices and why a new approach, such as SHIP-MD, is vitally needed.

“Having spent 30 years on the frontlines of pediatric healthcare as a surgeon, I saw so much innovation focused on adult medicine and not on pediatric populations. Instead, we were trying to adapt adult devices for use in children, which is not an effective solution,” says Dr. Newman. “Children’s National Hospital is proud to contribute to SHIP-MD’s pioneering efforts to address this critical disparity and reform pediatric device development in order to ensure that children, regardless of their age or condition, have access to the life-changing treatments and technologies they need to grow up stronger.”

An op-ed recently penned by Dr. Newman in STAT further explores the importance of public-private partnerships like SHIP-MD that are focused on fast-tracking innovation in medical devices for children.

Beers, who also serves as president of AAP, highlighted the fact that, as medical technology continues to advance, children are not reaping the benefits.

Kolaleh-Eskandanian

“Through the SHIP-MD initiative, we can work to ensure that the discipline of medical device development is equally understood and appreciated by its participating hospitals,” says Dr. Eskandanian.

“We must strive to improve medical devices for children, which historically lag five to 10 years behind adults. For many children, that can be a lifetime,” says Beers. “Much more needs to be done to address the countless hurdles that prohibit children from accessing the technology they need. The disproportionate rate of disease in minority children is another indicator that we must not cut corners as we look to improve pediatric innovation access.”

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National Hospital and principal investigator for the FDA-funded National Capital Consortium for Pediatric Device Innovation (NCC-PDI), co-led the Qualifying Hospital Criteria panel, which addressed the importance of expanding the SHIP-MD network to medical institutions that have the infrastructure for the safe conduct of research.

“Through the SHIP-MD initiative, we can work to ensure that the discipline of medical device development is equally understood and appreciated by its participating hospitals. As champions of pediatric innovation, we must work to provide equitable access to device trials for every patient that qualifies,” says Eskandanian. “The goal of the Qualifying Hospital Criteria group is to introduce criteria that hospitals must meet in order to provide a safe environment to conduct pediatric medical device research and trials.”

Co-leading the Regulatory panel was Francesca Joseph, M.D., FAAP, a pediatrician at Children’s National Hospital and co-investigator for NCC-PDI. This workshop explored opportunities to address regulatory needs by refining current processes and considering new options to promote advancement of pediatric medical devices.

Francesca Joseph

Co-leading the Regulatory panel was Dr. Francesca Joseph, a pediatrician at Children’s National Hospital and co-investigator for NCC-PDI.

In the closing session, Eskandanian and other panel experts recapped the workshop and discussed core factors that will help determine whether or not SHIP-MD’s network is prepared to enter Phase II, the consortium phase. This phase includes the development of a strategic plan that incorporates the short, medium and long-term goals needed to create and implement the framework enabling the official launch of SHIP-MD.

During his talk, Dr. Newman also shared the strategic steps being taken by Children’s National that complement the SHIP-MD initiative in advancing pediatric device innovation. Among these is the creation of the Children’s National Research & Innovation Campus (CNRIC), the first-of-its-kind pediatric research and innovation hub located in Washington, D.C., which includes on-site partners JLABS, Johnson & Johnson Innovation’s life science incubator, and Virginia Tech University. The campus will nurture a rich ecosystem for pediatric innovation in the nation’s capital.