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By Isabella Schmitt, RAC, director of regulatory affairs at Proxima CRO
The design and development of pediatric medical devices continues to be a challenge for many reasons. Fortunately, the Food and Drug Administration (FDA) has recently taken notice and begun working on strategies to bring more pediatric medical devices to patients. Today, there are far fewer pediatric devices than adult devices on the market, forcing pediatric specialists to find creative solutions to treat children, often modifying devices made for adults, using adult devices off-label, or seeking regulatory opportunities that afford quicker, but less advantageous, device approvals.
The financial, evidentiary, and regulatory challenges of the market often overshadow even the most basic, universal compassion of caring for sick children. Many sponsor companies simply are not willing to take on the financial risk to develop pediatric indicated devices as the smaller market size for pediatrics ultimately means a smaller return on their investment. The bottom line is that even though there aren’t many competitors in these areas of high need, the ends still don’t justify the means. Compassion isn’t enough to justify the cost, which in many cases can be quite high.
The cost of developing any medical product is significant, but in many adult cases, companies can rely on the large market size for financial benefit and previously cleared devices for ease in obtaining regulatory clearance. In the case of pediatric products, there are often no previously cleared devices, but even when there are, there may be a need to establish clinical safety and effectiveness in the pediatric patient population, specifically. The evidence generation may take the form of a clinical trial, which is one of the highest ticket items of any medical product’s development, and the duration of clinical trials for pediatrics can be lengthy due to enrollment challenges and/or the extensive follow-up timelines to account for child growth. A lengthy clinical trial timeline can delay the regulatory clearance or approval, and sales cannot begin until the product has been successfully reviewed by FDA.
From a regulatory perspective, because so few pediatric devices get the funding necessary to support development beyond the earliest stages, FDA often does not even get a glimpse of these products, resulting in fewer FDA reviewers, FDA clinicians, and FDA subject matter experts being exposed to devices unique to this population. This can cause delays in review timelines or misunderstandings between the companies and review teams.
To compensate for these challenges, companies may need to consider regulatory pathways, such as Humanitarian Device Exemption (HDE), in which the burden of evidence for approval is reduced from that of submission like a pre-market approval (PMA), resulting in quicker times to market and opportunities for profit. However, HDE approvals come with constraints that other regulatory pathways do not, which can limit sales and increase the company’s requirements for oversight. Other options, such as Breakthrough Device Designation and STeP designation, can help speed up the overall regulatory process by allowing for more FDA interaction and priority review timelines. However, these pathways have specific acceptance criteria and not all products will be eligible for these designations.
For many, including Vasum Peiris, MD, MPH, FAAP, FACC, FASE, chief medical officer and director of Pediatrics and Special Populations at the FDA Center for Devices and Radiological Health (CDHR), improving the regulatory process for pediatric medical devices is a matter of health equity. He believes the infrastructure exists within children’s hospitals across the nation to support an increase in the development of medical devices, but more emphasis needs to be placed on supporting innovation and regulation that provides equitable device design and development for all populations.
Pediatric device consortia have been established through grants to support pediatric device development. In the hopes to bridge some of the funding challenges for devices in these indications, these consortia provide funding support for regulatory, clinical, and reimbursement, as well as provide the opportunity for early-stage special meetings within FDA. This much needed support can help companies meet important milestones and obtain key insights into their overall path to market.
Effort is being made to bridge the gap between adult devices and pediatric devices, but we can’t afford to lose momentum. Children deserve the same access to life-changing, and often lifesaving, new devices that is afforded to adult populations.
Isabella Schmitt currently serves as the Director of Regulatory Affairs at Proxima Clinical Research, Inc.