The US Food and Drug Administration (“FDA”) recently published a draft guidance, entitled Patient Engagement in the Design and Conduct of Medical Device Clinical Investigations: Draft Guidance, providing suggestions to medical device sponsors on improving the design and conduct of medical device clinical investigations. Specifically, the draft guidance is intended to help sponsors understand how to use patient engagement to elicit experience and perspective, and to use that to improve study design, as well as identifying which patient engagement activities are generally not considered to be subject to FDA regulations, and addressing questions about collecting and submitting this patient engagement information to the FDA.
The draft guidance addresses the following four questions:
- What approaches might sponsors use to engage patient advisors (defined by FDA as those who have dealt with or are dealing with a particular condition but are not involved in clinical trials) to inform the design and conduct of device clinical investigations?
Here, the FDA recommends identifying patient advisors early in the clinical investigation planning process. Patient advisors should be “educated about clinical investigations, the various approaches to managing the disease/ condition of interest, and how a device may work.” Specific patient engagement activities that may enhance clinical investigation design and conduct include, for example, working with patient advisors to improve informed consent documents, obtaining input on flexible options for follow-up visits and data collection techniques, and discussing views on which potential endpoints are clinically meaningful.
- When can input be gathered from patient advisors and incorporated into the clinical investigation?
Patient advisors should be involved during the early planning phases so that their input can be incorporated into the study protocol while it is being developed. Such input should generally be incorporated prior to the submission of documents for FDA review. For ongoing studies, patient advisors can be used to help troubleshoot problems and propose solutions.
- What are the roles of institutional review boards (“IRBs”) and other institutional groups in patient engagement?
The FDA notes that “[b]ecause patient engagement activities with patient advisors primarily involve interaction in a consultative or advisory capacity, FDA does not generally consider these types of activities to constitute research or an activity subject to FDA’s regulation on their own.” Therefore, IRB requirements would generally not apply.
- How can a sponsor receive feedback on its patient engagement plan or patient-centered study design from FDA?
The FDA is open to discussing patient engagement approaches through an informational meeting and will provide feedback on strategies and plans.
The FDA notes that it believes “appropriate patient engagement may lead to improved efficiency and quality in the design and conduct of medical device clinical investigations,” and further that it may lead to “greater uptake of results … when making treatment decisions …, ultimately leading to earlier US patient access to beneficial medical devices.”