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University of California, San Francisco (UCSF)-Stanford CERSI

Tuesday, July 25, 2023

About the Presentation

This CERSI talk focused on regulatory challenges facing pediatric medical devices. In short, very few medical devices were FDA-approved or cleared for children. At the root of the problem was that the costs to develop a pediatric medical device were high while the commercial market was small. Thus, clinicians were forced to improvise—by squeezing an adult-sized device into and infant or child (e.g., adult pacemaker, ventricular assist device) or by using a device with outdated technology like the Berlin Heart VAD. In both instances, numerous studies showed that children had inferior outcomes compared to adults because of the size mismatch or used of dated technology. As a separate issue, because adult devices could be used in children off-label (i.e., without a clinical trial), there was a striking lack of safety and effectiveness data on the devices that were used routinely in children. In this first section, we discussed how pediatrics might use an adaptive platform trial to generate regulatory evidence more efficiently than conventional clinical trials. We explored this idea using ECMO (life-support) as a real-world illustration because ECMO was extremely high-risk and no standalone ECMO circuits had ever been FDA-cleared for children. We explored the potential efficiencies of this approach and how adaptive platform trials could save significant time and money relative to conventional trials. In the second section, we explored the broader context of the financial barriers facing pediatric device development. This discussion reflected the deliberations of a think tank on the pediatric medical device market failure convened in May of 2023 outside Washington, DC. The discussion brainstorms potential market and non-market (policy) based solutions that might address the market failure. In the last section, we discussed the problem of racial bias in FDA-approved pulse oximeters that could lead to serious health disparities in children with darker skin pigment. We reviewed the rationale and design of a CERSI-funded prospective clinical study being conducted at Stanford/Lucile Packard Children’s Hospital that parallels one being conducted at UCSF. We previewed some of the preliminary results, and discussed the challenges of conducting clinical trials involving pediatric medical devices in vulnerable populations.

About the Presenter

Dr. Almond is Professor of Pediatrics at Stanford University and Director of the Heart Failure Service and Cardiac Anticoagulation Service at Lucile Packard Children’s Hospital. His primary clinical and research efforts focus on the clinical evaluation and management of children with end-stage heart failure (HF), VAD support, and antithrombotic therapy for children with heart disease, the leading cause of stroke in children. His primary research interests include risk-stratification of children with end-stage HF, hemodynamic evaluation of children with HF, and the design of FDA trials to evaluate drugs and medical devices intended to treat pediatric heart failure. Dr. Almond has served as PI for several multicenter clinical trials including the FDA trial of the Berlin Heart EXCOR Pediatric VAD, the NHLBI-funded PumpKIN Trial evaluating the Jarvik 2015 LVAD, and the TEAMMATE trial, the first multicenter randomized clinical trial in pediatric heart transplantation evaluating the role of everolimus to prevent long-term transplant complications. Dr. Almond also serves as PI for an FDA-funded prospective clinical study to evaluate racial bias in commercially available pulse oximeters that may be a driver of health disparities. Dr. Almond has a special interest in the design of more efficient (smaller “N”) clinical trials through the development of novel surrogate endpoints and more flexible adaptive clinical trials that leverage incoming trial data to adjust parameters to avoid a failed trial and to protect human subjects from being randomized to inferior therapies. Dr. Almond completed an MPH in statistics and epidemiology at the Harvard School of Public Health and a Medical Device Fellowship in the Division of Cardiovascular Devices at FDA. He loves cycling and running and lives in San Francisco with his golden doodle, Kensi.

Recording

Play recording (55 mins)

For Questions :

Please contact Ruize Li at Ruize.Li@fda.hhs.gov .