A draft framework is in the works at the US Food and Drug Administration (FDA) to create an innovative safety net for pediatric medical devices, Vasum Peiris, Chief Medical Officer for pediatrics and special populations at the Center for Devices and Radiological Health (CDRH), told Focus.

The new framework is “centered around the fundamental issue of safety for children,” Peiris said, posing the question “where can we actually innovate safely?” Pediatric academic medical centers “have the breadth and depth of expertise to be able to innovate in a safe manner.” Partnerships with these centers will seek to create incentives not just to develop pediatric medical devices, but also to consider pediatrics before adult populations.

“If we can actually support technology development that assists the complex needs of children, we will accelerate device development for all," Peiris argued.

The framework is still in the preliminary stages—so much so that when it was mentioned at a session during RAPS’ 2018 Convergence last week, the presentation slide that laid out the current vision was intentionally blurred by Peiris. Yet the hope is that the framework will be “a bit closer to being ready for prime time” in up to a year from now, Peiris said.

NEST

Both internal and external partners are currently being engaged to then begin working out the details of the framework. This is because the process for creating the new nationwide network that can support innovation in pediatric device development, dubbed System of Hospitals for Innovation in Pediatrics (SHIP), will be very similar to the process started about three years ago for the National Evaluation System for health Technology (NEST), Peiris noted.

CDRH joined forces with the Medical Device Innovation Consortium (MDIC) in 2016 for the co-creation of NEST, with the $3 million in initial funding now reaching about $10 million via new awards and the Medical Device User Fee Amendments. But this has yet to be implemented, despite being a priority CDRH set for 2016-2017. Director Jeff Shuren said at the recent MDIC public forum that NEST is on track for launching “version 1.0 by end of 2019-ish.”

In 2017, FDA established the NEST Coordinating Center to ensure NEST “continues to move forward and becomes a sustainable entity that actually adds value to the healthcare system and to the system of medical device innovation,” Peiris said. He anticipates having a similar process of bringing together a variety of expert groups to work out the details of the SHIP.

SHIP

The idea stemmed from an ask from Congress in 2016 to identify barriers to pediatric medical device development, followed by the public meeting FDA held earlier this year on the topic.

According to Peiris, this public meeting confirmed that the issues the agency already expected to hear were focused on three main areas. These relate to optimizing evidence generation, developing regulatory value and simplicity, as well as creating a supportive marketplace.

But the “really unique aspects” of the conversation that emerged during the meeting were the issues surrounding the regulatory environments and economic issues, Peiris noted. His main takeaway, however, relates to the debate on improved return on investment as this is “keeping people from entering the space, innovating in the space and continuing to remain in the space.”

This debate led to a commitment on increasing incentives for sponsors and identifying the appropriate considerations for a new framework that can capture everything around the issue of return on investment, such as the decisions on what type of medical device to invest in.

SHIP would also seek to address the risk that has been historically associated with innovation.

“There is a risk associated with not innovating” as well, Peiris said. This is because with medical device innovation, comes additional opportunities to improve patients’ health, safety and overall quality of life. So the risk is “worth taking when you recognize that it can be done in a safe way and that it can make a significant difference to a patient,” Peiris added.

Further, the SHIP draft framework incorporates several other factors. These include the issue of de-risking the entire experience of innovation in pediatrics from a medical, technological and a business standpoint in terms of having a time-limited period to get products to the market.

It also seeks to harness the potential of a concept called reverse extrapolation. The process of extrapolating adult data for pediatric use is supported in 2016 final guidance on leveraging existing clinical data. But “there is far more potential and perhaps more ease with respect to collecting the data” when extrapolating from pediatrics to the adult market “and maybe enhancing labels from pediatrics to adults versus the other way around,” Peiris argued.

Peiris has already seen “a great deal of support” for the concept “from all the stakeholders” with whom he has engaged, including “a number of people in academics and industry” among others. “It is very reassuring to see that there is potential for the concept, especially because it makes a difference in the health and wellbeing of children,” he added.

Click on this link for more news coverage on RAPS' 2018 Convergence from Regulatory Focus.