For the 1.2 million Americans living with Type 1 diabetes, near constant monitoring of their body’s blood sugar level and correctly dosing the right amount of insulin are critical to their long-term health and their very survival.
Recognizing the tremendous need and opportunity to help this community and their caregivers, Food and Drug Administration employees Courtney Lias and Stayce Beck helped manufacturers receive quicker-than-expected regulatory approval for a groundbreaking medical device that assists people manage this serious disease.
Beginning in 2012, Lias and Beck spent several years transforming a slow and cumbersome process to review and approve breakthrough medical devices like the artificial pancreas. Along with providing clear guidelines to industry and setting performance and safety standards, they fostered discussions between government and private researchers, sponsored public forums and found ways to shorten clinical studies—making sure also to protect patient health.
The result was the first artificial pancreas, developed at least three years earlier than expected. Medical experts and patient advocates describe the device, approved by the FDA in September 2016, as “life-changing.”
“This is an incredible milestone,’ said Aaron Kowalski, chief mission officer for JDRF, an advocacy group for people with Type 1 diabetes. “To have a system automatically dose insulin is massive. And it opens the door to tons of possibilities.”
Type 1 diabetes is typically diagnosed in children and young adults. In people with this condition, the pancreas does not produce enough insulin, a hormone in our bodies that converts blood sugar to energy. People with Type 1 diabetes must constantly monitor their insulin levels, typically by multiple daily finger sticks and insulin injections. Missteps in this care can lead to long-term health complications and even death.
The new artificial pancreas system, made by medical device company Medtronic, for the first time automates some of the complicated steps needed to keep blood sugar under control. The system includes a glucose meter with a tiny electrode under the skin and an insulin pump that users attach to their body with a small catheter and infusion patch. The device measures a patient’s glucose levels every five minutes and either administers or withholds insulin as needed, helping people maintain glucose levels within the normal range most of the time.
Before Lias and Beck got involved, both the industry and patient advocacy groups publicly voiced concerns that FDA’s review process caused unnecessary roadblocks and was standing in the way of developing this technology and delivering it to those in need.
Lias and Beck changed this dynamic by treating all those involved—the device companies doing the research, the doctors, the hospitals and the advocacy groups—as partners, not adversaries, as they worked to improve and speed up the process.
“They used to see the FDA as a barrier and now I believe they see us as an ally,” Lias said.
Lias led FDA’s outreach to medical device companies working on this and other new diabetes technologies. She made sure manufacturers had a clear understanding of the documentation needed, how the clinical trials should be designed and how the patient testing of the device could be made safer.
Beck, described as a champion of the diabetes community, brought the voice of these individuals to the evaluation and approval process, and built relationships with other interested parties.
“We call Stayce the ambassador,’’ said Katherine Serrano, deputy director of the FDA’s Division of Chemistry and Toxicology Devices. “She’s really talented at getting groups of people together. She calms them and they really feel like she listens to them.”
Mark O’Donnell, vice president of regulatory affairs at Medtronic Diabetes, said Lias and Beck “created an environment where the goals were very clear of what FDA was looking for.’
O’Donnell also stressed that the expedited approval process did not sacrifice patient safety. “It was not a shortcut process,” he said. “It was a way to efficiently get all the information that FDA needed to ensure the safety and effectiveness of this device.”
Prior to their intervention, clinical investigators had to go through multiple rounds of reviews before they could start a clinical trial on systems like the one that Medtronic developed. Lias created a special team and timeline to expedite the clinical trials. The FDA team also worked with Medtronic to speed up the process by reviewing the device component by component, rather than wait until the entire system was complete.
“The main thing they did was listen,’’ said Dr. William Maisel, deputy director for science at the FDA’s Center for Devices and Radiological Health. “They worked to really understand the clinical environment and home care environment in which the product would be used. By listening, they were able to ensure that appropriate safety features were incorporated into the device.”
Kowalski said JDRF, the diabetes advocacy group, is funding work to integrate the new system into cell phones and make the device even smaller, and the effort led by Lias and Beck was an important step forward.
This first artificial pancreas system is “the biggest step forward since insulin, said Kowalski. “This work is going to help millions of people with diabetes.”