A group of researchers representing devicemakers, academic institutions and the FDA has published a list of factors device developers should consider when collecting patient preference information (PPI) to help design trials.

Writing in Therapeutic Innovation & Regulatory Science, the group listed five topics critical to leveraging PPI based on research done by the Medical Device Innovation Consortium: determining regulatory interest, selecting novel endpoints, aligning patient preference study attributes and traditional trial endpoints, statistical considerations, and the appropriateness of surveyed populations.

The researchers stress the importance of selecting the most appropriate attributes related to safety and effectiveness when developing a patient preference study. It’s highly critical that the attributes in a patient preference study are outcomes, characteristics or qualities that matter and make sense to patients, they say.

“Endpoints are most useful as attributes in a patient preference study when they can be translated into attributes that are understandable and meaningful to patients,” they wrote. “Once attributes that matter to patients have been identified through patient preference studies or other approaches, researchers can work with their clinical trial teams and regulators to appropriately incorporate them as endpoints in the clinical trial.”

Yet while an attribute deemed highly important to patients (such as survival or pain) and affected by the trial device could be used as a primary or secondary endpoint when appropriate, existing endpoints may not always map to attributes prioritized by patients, they noted.

Also critical is ensuring that the PPI gathered reflects the intended population of the trial device. To this end, the participants of a patient preference study need to be able to accurately provide details about their condition, a task that can be made complicated by different disease subtypes, stages and other aspects.

One approach is to engage patients referred by a physician or those whose electronic medical records include the specific diagnosis the trial device is meant to treat, but this can be expensive and time-consuming. As an alternative, patient preference studies can also gather data from patients who self-report their condition.

“In these cases, researchers may want to look for secondary data (e.g., the channel through which a patient was contacted, such as a patient organization network) and supporting information from participants (e.g., information about their symptoms or treatments that may be unique to the relevant condition) that can be used to increase confidence about the diagnosis,” they wrote. “Depending on the condition … self-report may be a justifiable approach to identifying participants.”

The researchers, including FDA Deputy Director of the Office of Strategic Partnerships and Technology Michelle Tarver, also advise sponsors to be keenly aware of the FDA’s strong support for gathering and using PPI in device development programs. The agency is very open to communicating directly with researchers on PPI initiatives, they said.

This was signaled in a big way at the beginning of the year, when the agency published final guidances on using patient-reported outcomes and patient advisers to enhance the design and conduct of device trials (CenterWatch Weekly, Jan. 31).

In addition, the agency this summer released further draft guidance on the topic, publishing a 52-page guidance on developing fit-for-purpose, patient-focused outcome measurements in device trials (CenterWatch Weekly, July 11).

Access the full paper here: https://bit.ly/3ASOBcV.