Turning ACTION Into Data: Dr. Angela Lorts and the Blueprint for Pediatric Medtech Innovation

For decades, pediatric cardiologists have MacGyvered medical devices built for adults to treat children with congenital heart problems. Since the pediatric market lacks the size of the total addressable market for adults, it’s routinely ignored by the R&D teams of startups and corporations. In 2017, after 11 years in the cardiac intensive care unit at Cincinnati Children’s Hospital Medical Center, Dr. Angela Lorts set out to do something big: persuade clinicians, engineers, FDA staff and parents of patients to combine forces to build a multi-center, data-sharing powerhouse.

The benefits of working in pediatrics typically include a high success rate. Kids heal. Sometimes remarkably fast. It’s different with cardiac disease. “I was always interested in cardiology and critical care,” Dr. Lorts says, “and taking care of the sickest patients.” Of the sickest children waiting for a heart transplant, 10-22% die before an organ match. That first step for Dr. Lorts entailed posing a question to her peers at other top-notch hospitals: Are you facing these problems, too? The consensus was an emphatic “yes.”

In 2018, Dr. Lorts, along with Dr. David N. Rosenthal (Stanford University) helped establish the rules of governance for the Advanced Cardiac Therapies Improving Outcomes Network (ACTION). No single hospital had a sufficient number of patients to produce generalizable knowledge. As a learning health care system, ACTION is committed to multistakeholder collaboration, quality improvement science, and data analytics. Their ethos embraces four core principles of select learning networks: steal shamelessly, share seamlessly, all teach, and all learn. (These principles were shamelessly appropriated.)

The Blueprint for Multi-Center Consensus

That December, ACTION unveiled “The Initial ABCs of Stroke Prevention: Quality Improvement Bundle.” The ABCs included standardized protocols modeled after Boston Children’s to combat the incidence of stroke in pediatric patients across 26 member hospitals. Each ACTION pediatric cardiologist was now helping shape the clinical standards for treating patients across the country. By 2019, ACTION included 40 different centers from multiple countries, as well as an active community of patients and families, clinicians, researchers, industry partners, and regulatory bodies. (Today, it’s 68 centers in eight different countries.) Then, as now, all parties work together to improve pediatric heart failure outcomes.

Transforming Adult Clearances into Pediatric Moats

In just eight years, ACTION’s model has produced remarkable results. For example, ACTION’s registry provided the robust, real-world evidence needed for Abbott and Johnson & Johnson (Abiomed) to secure landmark FDA expanded label indications for pediatric use for the HeartMate 3 and Impella devices, respectively. Likewise, for Berlin Heart®, ACTION ran the investigational device exemption trial for the EXCOR Active Driver for more than four years. With the reach of ACTION’s clinical registry, a company can streamline their study operations and reduce data collections 50-70%, compared to studies supported by traditional contract research organizations. “These clearances led to more efficient insurance coverage and pediatric training. And data was collected to monitor for patient safety and outcomes.” Dr. Lorts said. “It’s a big deal: it makes the use of these devices safer for children.”

This past June, ACTION researchers discovered that 3.3% of children experience myocardial functional recovery while supported on a durable ventricular assist device (VAD). Put another way: a small, but significant percentage of the hearts of the smallest patients have healed so well with the VAD, their doctors can explant the device and forgo a heart transplant. When clinical data scales, so do the insights—and so does the gratitude of patients and their parents.

A New Model for Clinical Champions and Founders

Dr. Lorts is also on the leadership team of the Midwest Pediatric Device Consortium (MPDC), and encourages founders and clinicians to take advantage of MPDC and ACTION. “Both organizations bring physicians and engineers and other stakeholders to the table, including the FDA and patients and families,” she said. “That’s the new model.” When physicians develop a better understanding of what goes on outside the hospital, they become better doctors. When founders confirm with physicians which problems are real and urgent, they produce better devices.

“If startups make something we already have, it’s not helpful,” Dr. Lorts says. “If they make something we’re not going to use, it’s not helpful. So how do we work together as a collaboration with engineers and providers to really figure out the unmet needs and then take them to the next level?”

Clinicians and Specialists:

Your medical expertise is the missing piece in early-stage development. To turn your clinical insights into better devices for kids, partner with MPDC today.

Founders and Medtech Startups:

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