FDA to Shift Clinical Evidence for Medical Devices Toward Postmarket

Advances in technology and data collection can help regulators and other parties keep better track of the safety and performance of medical devices once they are on the market, opening the door to potentially faster product development. This was the message delivered at a recent conference hosted by the University of Maryland’s Center of Excellence in Regulatory Science and Innovation (M-CERSI)-Building the National Evaluation System for Medical Devices: Using Real World Evidence to Improve Device Safety and Effectiveness.

At the moment, clinical trials and clinical practice are “parallel worlds,” according to Jeffrey Shuren, MD, director of the Food and Drug Administration (FDA)'s Center for Devices and Radiological Health (CDRH). But with the data available in electronic health records (EHRs), insurance claims forms, patient registries, and other databases, the FDA believes that creating a system to collect, aggregate, and analyze this valuable information could bring postmarket data into the realm of clinical evidence. Clinical trials are considered the gold standard for ensuring the safety and effectiveness of medical devices on the market; however, high costs, inefficient data generation, and other burdens associated with the regulatory process have created disincentives for companies to study their devices in the United States and are driving away innovation, as reported by a number of conference presenters.

“We face a critical public health challenge. The U.S. regulatory standard for market approval protects patients by setting a high public health bar but imposes costs that make the U.S. marketplace less attractive for innovators thereby delaying patient access to important technologies,” Shuren said in an earlier presentation. “The solution is to reduce the time and cost of the total product life cycle (device development, assessment, review, manufacturing, monitoring, and reimbursement) without compromising the reasonable assurance of safety and effectiveness standard.”

This focus on faster, less expensive product development coincides with a move by the U.S. Senate to create a “breakthrough pathway” for FDA approval of medical devices. The Advancing Breakthrough Devices for Patients Act of 2015 would allow shorter or smaller clinical studies and quicker measures of success to serve as sufficient premarket evidence for the approval of devices with the potential to "reduce or eliminate the need for hospitalization, improve patient quality of life, facilitate patients' ability to manage their own care (such as through self-directed personal assistance), or establish long-term clinical efficiencies."

Following approval by a bipartisan Senate committee, some medical-device safety experts have expressed concern that the bill “sets a low bar to qualify for ‘breakthrough’ status’” and “lowers standards for safety and effectiveness,” as articulated by Diana Zuckerman, president of the National Center for Health Research (NCHR) in Washington, a medical research and advocacy group, in The Wall Street Journal.

“We are concerned that the focus of these bills is on getting medical products to market more quickly, instead of making sure that they are safe and effective,” NCHR and 13 other medical safety groups wrote in a letter to the members of the Senate Committee on Health, Education, Labor, and Pensions Committee.

According to presenters at the M-CERSI conference, establishing a national device evaluation system is a way to strike the balance between faster, cheaper research and long-term, real-world date to assure safety and efficacy. The system, one of CDRH’s strategic priorities for 2016–2017, is envisioned to pool real-world data from disparate sources to provide meaningful information for not only regulators, but also for patients, physicians, payers, and manufacturers. So far, the FDA has invested $20 million to develop a way to link complementary registries and other sources of data, such as EHRs, the Centers for Medicare & Medicaid Services, insurance claims, and manufacturer databases, to facilitate the evaluation of medical devices over their total product life cycle.

A number of systems, such as the Medical Device Epidemiology Network Initiative (MDEpiNet), Sentinel, and the National Patient-Centered Clinical Research Network (PCORnet), provide this information on a smaller scale. However, this has created a “disparate, uncoordinated system of data,” according to Gregory Daniel, PhD, deputy director of the Duke-Margolis Center for Health Policy. A national evaluation system would build a coordinated approach and structure to integrate these data, he continued.

“When projects such as Sentinel and the National Medical Device Evaluation System are linked with the many complementary initiatives under way at our sister agencies and at organizations outside of the government, we can (and I believe in short order will!) build a robust foundation for a system in which both private and public sectors can produce much more useful knowledge at a fraction of the cost such efforts have previously required,” FDA Commissioner Robert M. Califf, MD, wrote in a FDAVoice blog post.

The feasibility of using such an approach to streamline clinical investigations is being investigated in a number of test cases. According to Danica Marinac-Dabic, MD, PhD, director of CDRH’s division of epidemiology, researchers are using national registries to conduct 15 post-approval studies, one continued access study, and seven postmarket surveillance studies, as well as support one label extension, paving the way for “faster, less expensive regulatory decision-making based on the best available data from clinical settings.” The FDA’s goal is to gain access to 25 million electronic patient records from national and international clinical registries, claims data, and EHRs by the end of 2016. It is also aiming to increase the number of pre- and postmarket decisions that leverage real-world evidence by 40 percent during that same timeframe.

Source: Association for the Advancement of Medical Instrumentation (AAMI)