Jeffrey Shuren

Need for a National Evaluation System for Health Technology

Federal regulatory frameworks governing medical products are designed to (1) provide evidence that a product benefits patients when used as intended and should be available despite accompanying risks and (2) ensure timely access to needed therapies and diagnostics. Historically, policy makers and product developers have viewed these objectives as being in tension. However, ensuring safety, expediting patient access, and enabling innovation can be complementary goals within a regulatory framework for medical devices. The US standard for marketing a medical device is “reasonable assurance of safety and effectiveness” (RASE).1 Generally, clinical studies must be conducted to demonstrate RASE for both high-risk and innovative lower-risk devices and US patients and clinicians have greater assurances that the benefits of devices outweigh the potential risks. In contrast, other countries apply a standard of safety and performance with limited clinical data. The greater evidentiary burden of RASE may create disincentives for manufacturers to bring important medical devices to the United States or may delay access to devices. For example, the first transcatheter aortic valve replacement device was available for clinical use in Europe several years before it was available in the United States. However, there are examples of unsafe and ineffective devices that never made it to the US market; these can be found in a report2 from the US Food and Drug Administration (FDA). A key dilemma for device regulation is how to ensure timely access while also providing evidence to guide safe and appropriate use. When a device is approved for the US market, residual uncertainty about benefit and risk is typically addressed through postmarket evaluation. Premarket studies often do not fully reflect how a device will be used in practice, and participants enrolled in such studiesmaynotrepresenttheentirespectrumofpatients likely to receive the device. The effects of operator experience, user learning curves, or skill level of the individual who implants the device and the supporting team also cannot be assessed until the device is in wider use. However, current approaches to postmarket evaluation have limitations. Even though the FDA can require device makers to perform postmarket studies, patients have few incentives to enroll in a study once a device is marketed, and many FDA-mandated postmarket studies for devices have been delayed, scaled back, or never finished. Generally, if the company makes a good-faith effort in performing postmarket studies, there are no penalties. Furthermore, reporting of adverse events and device malfunctions currently depends on clinicians identifying and reporting a possible association; therefore, underreporting is likely common. Spontaneous reporting also fails to capture numerators and denominators that allow reliable risk estimation. Safety issues are therefore often not identified until many patients have been exposed to risks, leading to greater potential for avoidable harm as well as greater liability and loss of consumer confidence in the manufacturer. Spontaneous reporting is not systematic and can be biased by extraneous factors such as news reports. Other safety issues also depend on companies appropriately assimilating and reporting data. However, a strategic approach to linking and using clinically based data sources, such as registries, electronic health records (EHRs), and claims data, could potentially reduce the burdens of obtaining appropriate evidence across the life cycle of a device. By leveraging clinical data and applying advanced analytics and flexible regulatory approaches tailored to the unique data needs and innovation cycles of specific device types, a more comprehensive and accurate framework could be created for assessing the risks and benefits of devices.

An FDA Viewpoint on Unique Considerations for Medical-Device Clinical Trials

Medical devices range from something as simple as an elastic bandage to something as complicated as a deep-brain stimulator. This review describes the FDA approach for understanding and approving medical devices.

Reduced Leaflet Motion in Bioprosthetic Aortic Valves — The FDA Perspective

The incidence and mechanism of reduced leaflet motion in bioprosthetic aortic valves and the accompanying risks cannot yet be fully characterized. Whether it is clinically meaningful or merely an advanced-imaging phenomenon demands additional investigation.

FDA Regulation of Mobile Medical Apps

Mobile apps are increasingly used in health care to promote wellness, treat and diagnose disease, aid clinical decision-making, and manage patient care in hospitals and homes. Historically, health care has been slow to implement disruptive technology tools that have transformed other areas of commerce and daily life. One factor that has been cited is uncertainty surrounding regulation that accompanies medical products, and how US Food and Drug Administration (FDA) regulations may apply to software platforms. There also are questions in the marketplace about the clinical validity and utility of certain mobile tools. Efficient regulation can help promote adoption of mobile medical apps. FDA determination that a product developer or manufacturer has met the high regulatory standard for demonstrating clinical benefit and safety (when agency clearance or approval of the app is required) can increase consumer confidence in that technology.1 In these cases, FDA regulation also can help patients, payers, and investors better understand the performance characteristics of high-quality software products, encouraging a “race to the top” in medical app development.

The FDA and Genetic Testing

This letter provides a response by the Food and Drug Administration to the Special Report, in this issue of the Journal, regarding FDA premarket review of genetic tests.