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).1Generally, 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 studies may not represent the entire spectrum of patients 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.
A national evaluation system that engages all stakeholders could enable the FDA to focus efforts on facilitating the development and interpretation of more informative data essential for policy making and clinical decisions for individuals and populations. When issues with medical technologies arise, they could potentially be quickly detected and understood within the appropriate context. Ultimately, these changes could contribute to a much more efficient system that rewards innovation that leads to better health outcomes, creating powerful incentives for continuous improvement and accelerating access to technologies that patients and physicians can use with the assurance of safety, efficacy, and a well-characterized balance of benefit and risk.