USFDA Guidance: Incorporating Voluntary Patient Preference Information Throughout the Product Life Cycle

The USFDA Center for Devices and Radiological Health (CDRH) and the Center for Biologics Evaluation and Research (CBER) have issued a draft guidance titled “Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle.” This document outlines when and how to gather and submit patient preference information (PPI) throughout the entire product lifecycle. The aim is to incorporate voluntary PPI into the decision-making processes for medical devices and biologics.

The guidance is mainly directed at device manufacturers and biologics developers, encouraging them to incorporate Patient Preference Information (PPI) throughout the Total Product Life Cycle (TPLC), from development to post-market surveillance.

“Patient experience data” refers to information about patients’ experiences with a disease or condition. The FDA promotes the development and evaluation of devices that take into account patient experience data, including patient preferences regarding outcomes and treatments. This guidance specifically focuses on Patient Preference Information (PPI).

PPI is defined as qualitative or quantitative evaluations of how patients perceive the desirability or acceptability of different outcomes or attributes associated with various health interventions.

This guidance offers recommendations on how voluntary Patient Preference Information (PPI) can be utilized by FDA staff in their decision-making processes. The main objectives of this guidance are:USFDA

• To encourage sponsors or other interested parties to submit available PPI to the FDA to assist in decision-making.

  

• To outline the recommended qualities of patient preference studies that can produce valid scientific evidence.
• To provide practical advice on collecting and submitting PPI to the FDA.
• To discuss how the FDA includes PPI in its decision summaries and to offer recommendations for incorporating such information into device labeling.

Patient Preference Information (PPI) can be particularly valuable for assessing the benefit-risk profile of a device when patient choices are influenced by personal preferences. Such decisions are deemed preference-sensitive under the following conditions:

1. There are multiple treatment or diagnostic options available, none of which is universally superior for every patient.
2. The evidence favoring one option over others is either ambiguous or varies among different patient groups.
3. There are significant differences between patients’ perceptions of benefits and acceptable risks compared to those of healthcare professionals.

PPI can enhance the FDA’s benefit-risk evaluations by:

1. Highlighting the key benefits and risks from the patients’ viewpoint.
2. Gauging the relative significance of these benefits and risks to patients, including their preferences for trade-offs.
3. Revealing variations in patient preferences, particularly across different subgroups.

Manufacturers are advised to engage with the FDA during the design of Patient Preference Information (PPI) studies and to share their findings. This collaboration helps to ensure that the studies meet regulatory standards.

For comprehensive details on integrating patient input into FDA decision-making and guidelines for conducting Patient Preference Studies, refer to the official FDA guidance documents:

• Incorporating Voluntary Patient Preference Information Over the Total Product Life Cycle (PDF)
• FDA Guidance Webpage: Incorporating Voluntary Patient Preference Information Over the Total Product Life Cycle.