Patient Engagement in Device Clinical Trials Guidance

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02/15/2022

Photo by National Cancer Institute on Unsplash

The US Food and Drug Administration (FDA) released a guidance, “Patient Engagement in the Design and Conduct of Medical Device Clinical Studies.” The purpose is to guide industry to consider patient and caregiver perspectives on living with a particular disease or condition and how medical devices impact their lives, as well as perspectives on how a clinical trial may impact a study participant. The guidance is part of the FDA Patient Engagement Advisory Committee (PEAC) initiative to increase patient engagement in medical device clinical trials. The topic was discussed in a public workshop through a collaboration with the Clinical Trials Transformation Initiative (CTTI) with comments generated incorporated into the final guidance. The objective of patient engagement is that patients must be willing and able to use a medical device in order for it to have a practical benefit, so understanding any potential barriers prior to conducting a clinical trial would be beneficial. Engaging patient advisors early in the process can reduce challenges to recruitment and retention and avoid over burdening participants while maintaining a more meaningful approach to study conduct.

Background:

• Patient engagement is defined as “intentional, meaningful interactions with patients that provide opportunities for mutual learning, and effective collaborations.”

• Patient advisors are “individuals who have experience living with a disease or condition, and can serve in an advisory or consultative capacity to improve clinical investigation design and conduct, but who are not study/research participants themselves or caregivers of study/research participants.”

• Patient advisors: 1) may provide valuable information for medical device clinical trial design, 2) may recommend improved participant experience, and 3) may improve the relevance of the clinical trial.

• To avoid a conflict of interest, patient advisors should not be enrolled in the study for which they advised.

Questions and Answers about Patient Engagement:

• Question 1: “What approaches might sponsors use to engage patient advisors to inform the design and conduct of medical device clinical studies?”

  • Answer: Sponsors should be clear in their study plan which activities are intended for study participants and which are for patient advisors. Patient advisors who are educated about clinical trials, their disease or condition, and how the medical device works would be more beneficial and influential. It is suggested for sponsors to train patient advisors to strengthen their ability to meaningfully contribute to engagement activities.

  • Some engagement activities that are suggested include:

    • Discussing what endpoints are meaningful to the patient

    • Ensuring the consent form is understandable

    • Understanding patient perspective on benefit-risk for the treatment option or informing the design of patient preference trials

    • Discussing potential burdens for recruitment, data collection, and follow-up. Discussions can include possibilities for increased flexibility in study visits and data collection.

    • Informing which data measure meaningful outcomes that should be captured by patient-reported outcome (PRO)

• Question 2: “When can input be gathered from patient advisors and incorporated into the clinical study?”

  • Answer: Input from patient advisors should be taken into consideration during the early planning of a trial. This ensures a patient-centric approach is built into the study design.

  • Any patient input needs to be included before the final protocol and informed consent are submitted to the IRB for review, and to the FDA as part of an investigational device exemption (IDE) application.

  • If a trial is having challenges with recruitment or retention, it is recommended to involve patient advisors and site staff to assess and propose possible solutions.

  • There may also be a benefit to involving patient advisors post-study to inform future trials.

• Question 3: “What are the roles of IRBs and other institutional groups in patient engagement?”

  • Answer: Because communication with a patient advisor is not the same as the interaction with a research subject, the FDA’s research regulations for IRB review to ensure subject safety and protection do not apply.

  • When engaging with patient advisors, experiences and perspectives may be a part of the discussion. If there is concern over any privacy issues, the FDA recommends consulting IRBs and Health Insurance Portability and Accountability Act (HIPAA) Privacy Boards for clarification.

• Question 4: “How can a sponsor receive feedback on its patient engagement plan or patient centered study design from FDA?”

  • Answer: The sponsor may receive feedback from the FDA about their patient engagement approaches through the optional Q-Submission Program.

  • Additional information on how to request feedback is available on the CDRH Patient Science and Engagement Program website.

Sponsors should document patient engagement activities used to develop the study plan in their marketing application.

By engaging with patient advisors to understand their perspectives on meaningful medical devices and potential burdens or challenges in the study plan, such as overly restrictive inclusion/exclusion criteria, sponsors can build quality into their study design. A patient-centric, more meaningful, less burdensome design can increase enrollment and compliance, thus resulting in high-quality data. Engaging patient advisors can lead to a medical device with an established practical benefit that fits the needs of the patients who have the condition.

- The Clinical Pathways Team

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