FDA tries to spur patient shaping of clinical trial design with draft guidance

Dive Brief:

FDA is aiming to better incorporate patient views into clinical trial design, as laid out in a draft guidance released Monday.
Regulators are advocating that sponsors identify patient advisors and work with them to improve informed consent and make studies less burdensome for participants.
The agency is accepting feedback on the "Patient Engagement in the Design and Conduct of Medical Device Clinical Investigations" document for the next 60 days.

Dive Insight:

FDA thinks companies that fail to incorporate the patient voice into the designs of their medical device clinical trials risk running suboptimal studies. In a statement published to accompany the guidance, acting FDA Commissioner Ned Sharpless said trials that lack patient input may fail to gather data on important outcomes, be overly burdensome for subjects and, as a consequence, enroll slowly.

"We value patient experience and insights about living with a disease, like diabetes or end-stage renal disease, as well as any impact a medical device — such as a glucose monitor or hemodialysis system—has had in a patient’s diagnosis, treatment and management of that disease," Sharpless said in the statement. "Patient experiences and insight can help us understand the benefits most important to patients and what risks patients may or may not be willing to tolerate."

The Patient Engagement Advisory Committee pushed these issues up the agenda at FDA from its first meeting in 2017, leading to the publication of draft guidance on patient engagement this week.

In the draft, FDA discusses how sponsors can best engage patients. FDA said sponsor companies are best served by identifying patient advisors with expertise in clinical trials, how a disease is managed and why the device being tested may improve on existing options. The agency is encouraging sponsors to work with organizations that train patient advisors to get the most from their contributors.

Patient advisors can inform a range of aspects of clinical trial design. In the guidance, the agency discusses how to work with patient advisors to make trials less burdensome; for example, by allowing subjects to visit study sites on weekends or using technologies to eliminate the need for some face-to-face interactions altogether.

FDA is advising sponsors to gather the views of patient advisors on such topics early in the process of designing a clinical trial, which can enable patient advisors to influence the protocol. Another option put forward by FDA is to enlist patient advisors to identify ways to improve ongoing studies struggling to recruit or retain subjects.

The agency said that sponsors wanting feedback on their planned use of input from patient advisors can send questions to FDA via its Q-Submission Program, which regulators use to field specific queries related to device filings.