The U.S. Food and Drug Administration (FDA) has issued two final guidance notices that are intended to give patients and advocates a larger voice in how medical devices are manufactured, tested and monitored. The FDA released the guidances on January 26.
- Patient Engagement in the Design and Conduct of Medical Device Clinical Studies: Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders
- Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation: Guidance for Industry and Food and Drug Administration Staff, And Other Stakeholders,
The Patient Engagement in the Design and Conduct of Medical Device Clinical Studies guidance was the first to be published by the FDA. The full patient engagement guidance, published in the Federal Register, focused on patients sharing their experiences.
This guidance was developed by the FDA’s Patient Engagement Advisory Committee (PEAC) who recommend a framework in order to “clarify how patient advisors can engage in the clinical investigation process.”
This PEAC framework has been in development since October 11, 2017, when the subject of increasing voluntary patient engagement became a focus of the committee.
The primary goal of this guidance is to “Help sponsors understand how they can voluntarily use patient engagement to elicit experience, perspectives, and other relevant information from patient advisors . . . to improve the design and conduct of medical device clinical studies.”
The guidance also seeks to address common barriers to understanding how to engage with the FDA, including clarifying common questions and misconceptions about collecting and submitting patient engagement information in the design and methods of a medical device study to the FDA.
By listening to patients, the FDA states that devices can be made safer and more effective from the very beginning of the design process. This proactive approach to medical device design heavily emphasizes how patient feedback can catch red flags in the medical device’s use very early on.
The FDA also notes that although patient feedback is helpful across the lifecycle of a device, this patient engagement guidance was tailored to focus on how patients can help shape the medical device design process through feedback and experiences. The second guidance was a broader topic that was able to provide ways for patient engagement to help manufacturers over the lifetime of the device.
The Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation guidance focuses on how patient experiences can be converted into hard data that manufacturers can use in order to make long-term decisions about their devices.
The FDA describes Patient-reported outcome (PRO) instruments that allow for “the systematic collection of patient perspectives as valid scientific evidence to support the regulatory and healthcare decision-making process.”
According to the FDA guidance, a PRO instrument can be used to:
- Inform a patient's eligibility for inclusion within a study
- Capture safety or effectiveness outcomes
- Serve as primary or secondary measurement of a clinical study’s success
- Serve as a component of a composite measurement of a study’s success
When it comes to the use of PRO instruments to measure the results of a study, the FDA takes a flexible, “fit-for-purpose” approach to determine whether a PRO instrument’s measurements are properly answering the questions of a medical device trial study. The agency has come out in strong support of PRO instruments’ use in clinical studies, stating, “FDA encourages the collection, analysis, and integration of patient perspectives in the development, evaluation, and surveillance of medical devices.”
With these recently issued guidances combined, the FDA has given manufacturers a new, specific framework through which they can incorporate patient feedback at all stages of development for medical devices. This guidance also gives health advocates and patients a new avenue by which they can make their voices heard.