- The FDA plans to improve patient access to medical devices by streamlining the agency’s regulatory actions, FDA Commissioner Scott Gottlieb, MD, announced.
In a public statement, Gottlieb reiterated the agency’s commitment to improving patient access to devices and treatments while ensuring patient safety.
Currently, FDA is facing issues with small, iterative improvements to medical devices, Gottlieb said. These improvements are often essential for supporting patient safety.
“Therefore it’s important for the FDA to enable innovators to have the flexibility to efficiently make these kinds of small modifications,” Gottlieb said. “At the same time, the FDA needs to establish modern tools and benchmarks for measuring the safety and performance of devices to make sure they’re delivering the expected benefits to patients.”
FDA created a medical device development tool (MDD) in response. The first MDDT is targeted for developers creating tools for patients with cardiovascular disease. Standardizing the device development process will make it easier for developers and will streamline the approval process. Ideally, this will help more patients access devices quicker.
The 23-item questionnaire collects patient-generated health data (PGHD) to assess how a cardiovascular health tool improves patient health and provides quality healthcare.
“By qualifying the tool under the FDA’s new, voluntary program, it will make it easier for product developers to rely on the outputs of this newly qualified tool as part of their development plans,” Gottlieb explained. “Innovators can trust in advance that the agency has already found the outputs of these measures to be reliable.”
“The development of more modern tools for measuring outcomes relevant to regulatory decisions is a key part of our efforts to promote innovation,” Gottlieb continued. “Such development tools can also help minimize the use of animal studies, reduce the duration of testing, or require fewer patients in a study by optimizing patient selection or improving on the ability to measure benefit and risk through the availability of measurements that are more sensitive for assessing these outcomes.”
Gottlieb also announced three guidance documents that will help improve patient access to potentially life-saving health technologies.
The “Breakthrough Devices Program” builds upon the Expedited Access Pathway (EAP) program via the 21st Century Cures Act and helps patients access devices that will stop or slow down progression of life-threatening or degenerative diseases. This is especially useful for devices for which there is no equivalent alternative.
The “Breakthrough Devices Program” draft proposal streamlines the authorization process and supports better and more frequent communication between device developers and FDA.
The final two guidance documents pertain to 510(k)s, which relate to premarket notification documents. This will essentially make it clearer to device developers when they need to consult with FDA about small, incremental changes to devices already on the market. Developers can make these safety changes on market devices quickly, improving patient access to important devices.
The final two guidance documents do not change FDA’s regulations, the agency maintained. Instead, they clarify regulations, making it easier and quicker for developers to work with FDA.
“This improved clarity will help lower the barriers to innovation and improve patient care by reducing unnecessary submissions to the FDA for changes that could not significantly affect device safety or effectiveness, so patients can benefit from upgraded products more quickly,” Gottlieb wrote.
These new policies are part of FDA’s overarching efforts to improve patient access to medical devices while protecting for patient safety. This is an admittedly hard line to walk, Gottlieb suggested. Over-regulation may hinder more patients from accessing life-saving tools, but under-regulation could land unsafe devices in the hands of patients and providers.
FDA has been working to improve patient access to medical devices and other treatments. The agency has recently implemented an expanded access program, which lets patients with terminal or life-threatening illnesses with no alternative treatment option access to experimental drugs.
The program has been largely successful, according to a 2017 Government Accountability Office (GAO) report. Between fiscal years 2012 and 2015, FDA approved 99 percent of the 5,800 expanded access requests it received.
Per program regulations, the FDA must respond to expanded access requests within a 30-day window. GAO reported that the FDA typically responded to these requests within hours, but in the case that responses did take longer, they have always fallen within the required timeframe.
FDA does have room for improvement with its expanded access program, specifically related to transparent outcomes data use. However, this program has been a boon for patients seeking alternative treatment options.
All of this is a part of FDA’s efforts for driving more patient-centric care. The agency is slated to announce further plans for patient access to medical devices, and will continue its patient-centric strategy through its Patient Engagement Advisory Committee.