Let’s Listen to Patients: FDA Releases Final Guidance on Patient Engagement in the Design and Conduct of Medical Device Clinical Studies

March 1, 2022By Philip Won & James E. Valentine & Larry J. Bauer, Senior Regulatory Drug Expert

Our readers probably know that we value and advocate for patient engagement as an essential component in the development of medical products (see our firm’s commitment here).  The COVID-19 pandemic cannot keep us from actively facilitating Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings with the Food and Drug Administration (FDA) (see here) along with a legacy of patient engagement in pre-pandemic times (see our previous coverage on patient engagement activities here, here, here, here, here, here, and here).  While FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have been in the limelight for championing approaches to patient engagement for drug development, the Center for Devices and Radiological Health (CDRH) has more quietly been plugging away at their own draft guidance on Patient Engagement in the Design and Conduct of Medical Device Clinical Investigations in September 2019 (see our previous coverage on it here).

On January 26, 2022, CDRH, along with CBER, released a final guidance document Patient Engagement in the Design and Conduct of Medical Device Clinical Studies (“Guidance”), which is the culmination of many years of efforts including FDA’s Patient Engagement Advisory Committee (PEAC) meetings in 2017 and 2018, and the Clinical Trials Transformation Initiative (CTTI) workshop in 2019.  The Agency’s goal is demonstrated in its definition of patient engagement:

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

To this end, this Guidance is intended 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;
  • highlight the benefits of engaging with patient advisors early in the medical device development process;
  • illustrate which patient engagement activities are generally not considered by FDA to constitute research or an activity subject to FDA’s regulations, including regulations regarding institutional review boards (IRBs); and
  • address common questions and misconceptions about collecting and submitting to FDA patient engagement information regarding the design and conduct of a medical device clinical study.”

What would be the potential benefits if companies prospectively design medical device clinical studies with input from diverse patient advisors?  There are several examples that the Agency provided in the Guidance such as (1) streamlined data collection resulting in better quality data, (2) more relevant data on outcomes that matter to patients, and (3) fewer costly and time-consuming protocol revisions.  The Agency also acknowledges that patient engagement may be beneficial across the total product lifecycle, which is beyond the scope of the design and conduct of clinical studies.  This Guidance embodies the Agency’s belief that:

“Successful adoption of legally marketed medical devices increasingly depends on patient acceptance of that technology and patients being more engaged in the healthcare process, along with demonstrated public health benefits.”

The Guidance provides practical advice for device manufacturers to utilize patient engagement in clinical studies.  The Agency envisions a range of activities that may enhance the design and conduct of clinical studies through engagement with “patient advisors” – individuals who have experience living with a disease or condition, but who are not study/research participants themselves or caregivers of study/research participants.  This includes:

  • Improving the informed consent document to ensure patients understand the information presented for the clinical study;
  • Obtaining input on flexible options for follow-up visits and data collection techniques to reduce unnecessary burden on study/research participants;
  • Working with patient advisors as needed during an ongoing study to discuss barriers to recruitment or other issues such as causes of study delays or challenges not anticipated before the study;
  • Discussing views on which potential endpoints are meaningful in the treatment of the specific disease/condition;
  • Informing the concepts that should be captured by patient reported outcome (PRO) measures in the clinical study to better reflect outcomes that are important to patients; and
  • Informing the design of patient preference studies that may be used to inform the development of clinical studies or to help understand the benefit-risk tradeoffs among patients for the proposed treatment or multiple treatment options used for the disease/condition.

The Agency highlights that sponsors should gather input from patient advisors during the early planning phases of the clinical study so that their input can be incorporated while the study plan is being developed.  FDA not only endorses this for use by investigational device developers, but to be provided to the Agency to inform their own thinking.  For example, if clinical studies require submission of an investigational device exemption (IDE) application, the Agency encourages patient advisors’ input be included in the final protocols and informed consent documents in the IDE application for FDA review.  For the actual adoption of inputs from patient advisors, we hope that the Agency provides practical guidance on how those inputs could be incorporated in the IDE application or subsequent premarket submissions.

The Agency encourages sponsors to engage in early interactions with the relevant FDA office/division to obtain feedback upon appropriate design and any applicable regulatory requirements when considering incorporating input from patient advisors in the design or conduct of medical device clinical study.  For additional information on planning patient engagement, please see the CDRH’s Patient Science and Engagement Program.  A sponsor can use the Q-Submission Program to obtain FDA feedback on its patient engagement plan or patient-centered study design.

Finally, mark your calendars for the upcoming March 22, 2022 FDA webinar about this Guidance.  The FDA webinar information can be found here.  Hearing from patients at the outset can help lead to better clinical trial designs, expedited patient recruitment, and ultimately greater acceptance of new medical devices!