CDRH Discusses Patient Preference Data in Clinical Trials
CDRH says it is expecting more collection of data on patient preference in device clinical trials. Asked a question during a recent teleconference (see transcript) on the agency’s final guidance on “Factors to Consider When Making Benefit-Risk Determinations for Medical Device Investigational Device Exemptions,” CDRH policy analyst Karen Ulisney said that if a medical device product area “lends itself to collecting more patient preference information, then we would expect that you would consider including that in your [IDE] application. If there are specific patient-reported outcome tools that may be appropriate to the particular patient population we would highly encourage you to include that in the study design and include that as a factor in considering the benefit risk determination.”
The guidance is intended to provide greater clarity on the principal factors that FDA considers when assessing the benefits and risks of IDE applications for human clinical studies. The agency believes that by improving clarity more medical device clinical trials will be conducted in the U.S. It also discusses how risk-benefit uncertainty may be offset by a variety of risk mitigation measures which can ensure appropriate patient and research participant protections in investigational research settings.
CDRH clinical trials director Owen Ferris added that “if there are data that help us understand the benefit risk equation for whether to approve an IDE or not — that some patients would accept a certain benefit risk profile that other patients might not — that would be helpful information if it’s reliable data that we can use as part of our decision making process. It’s also the case that trials themselves can benefit from gathering patient preference information as part of the trial design so that that information can be used to inform future decisions around that product. So a trial could be designed to gather some patient preference information, some patient recorded outcomes, information about how patients experienced the benefit or the risks of a device. And that information can be extremely helpful in informing the decisions we make later about whether that device should be on the market.”
Ferris also said that patients can be very helpful in determining the right end points for a trial. “If we're saying that this is a particular success criterion for what determines study success, well if that information is informed by patient input that’s even better,” he said. “And so there are many ways in which patients can and should be brought more into the conversation. This guidance in particular focuses on the decision around approving an IDE and if there’s patient information that helps us make that benefit risk determination as to whether that IDE should go forward. But I think that is just one segment of the whole scope of where patient information can and should be increased and it’s something that we as an agency are trying to more towards.”