Considering Patient Preference Information in FDA Submissions

Incorporating voluntary patient preference information throughout the total product lifecycle

Patient preference may have more of an effect on Food and Drug Administration (FDA) medical device submissions in the future. In March, FDA finalized its guidance for incorporating voluntary patient preference information (PPI) into various medical device submissions. This guidance provides recommendations about when and how to collect and submit PPI to FDA. While the agency does not require PPI, it now considers PPI as part of the risk-benefit human factors validation submissions, where additional data, repeat testing, or redesign may be requested. 

This finalized guidance signals FDA's expectation that sponsors will increasingly know when and how patient preferences matter; as such, they may need to justify why they did or did not collect PPI.

What does the FDA guidance say about PPI?

PPI is defined as "qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions." PPI should measure which attributes are important to patients, how important they are, and/or what tradeoffs patients may be willing to make among them.

The finalized guidance document expands the scope of PPI use, formalizing its application beyond traditional pathways such as premarket approval (PMA) and humanitarian device exemption (HDE). It now explicitly includes the consideration of PPI in 510(k) submissions, De Novo requests, and postmarket compliance and enforcement decisions. This expansion signals FDA's commitment to integrating patient perspectives more broadly across the entire medical device regulatory lifecycle.

In addition to expanding scope, the guidance provides greater methodological specificity. It clarifies recommendations for how to structure study designs to effectively incorporate patient engagement, detailing the stages of study development and implementation where such involvement may yield the greatest value. By establishing clearer expectations for when and how to involve patients in research, Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle aims to strengthen the quality and relevance of PPI studies submitted to FDA.

Finally, the guidance offers strategic advice to stakeholders and emphasizes the importance of PPI in the postmarket phase. It includes recommendations on when sponsors should consider initiating meetings with FDA to discuss their PPI plans, supporting alignment early in the process. Moreover, it highlights how patient input can play a critical role in postmarket surveillance and regulatory decision-making once a device is commercially available, reinforcing a continuous focus on patient-centric evaluation throughout the product's lifecycle.

When may it be useful to collect and submit PPI?

FDA offers guidance to medical device manufacturers about when it may be useful to collect and submit PPI. The guidance recommends PPI for devices that directly involve patients and where patient-perceived tradeoffs are central to evaluating benefits and risks. This includes devices with a direct patient interface; those expected to provide meaningful health, appearance, or health-related quality-of-life benefits; life-saving but higher-risk devices; products intended to address unmet medical needs or rare diseases/conditions; devices offering alternative benefits compared with marketed options; devices using novel technology; and devices for which key clinical endpoints rely on subjective patient experience.

According to the guidance, FDA staff may also find PPI particularly useful when they need to better understand the real-world impact of a disease/condition and available treatment options on patients and caregivers, or when patients may value a device's benefits and risks differently than clinicians or caregivers do. PPI can help clarify population-level differences in perspectives across demographic groups, disease stages, or disease/condition phenotypes. 

PPI in the real world

The new guidance features several examples of PPI playing an important role in FDA submissions, including a study sponsored by the Center for Devices and Radiological Health (CDRH) to evaluate patient preference regarding benefits and risk tolerance for weight-loss device treatments for obesity. Over 500 U.S. adults representative of obese patients participated. Stratified by body mass index, the study used validated preference-elicitation methods to segment patients by risk tolerance and identify those willing to accept surgical risks for substantial weight loss. Conducted under recognized research standards, it sought to minimize cognitive bias and ensure patient-centered communication. Findings indicated many patients would accept notable risks for meaningful weight reduction, offering data to improve clinical trial design and support patient-centered regulatory decisions. 

What it means for medical device manufacturers

Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle provides several points of consideration by manufacturers. Specifically, the FDA encourages manufacturers to:

• Approach gathering PPI as an iterative process across the total product lifecycle.
• Consider users and subgroups of users who may interact with the device and consider their preferences, even during the early stages of design. 
• Define attributes expected to be key in decision-making processes when collecting PPI. 
• Take a robust scientific approach to collecting and analyzing PPI, using quantitative analysis and statistical models that provide estimates of the parameters of interest to help reviewers understand the sources of uncertainty that will be considered in PPI survey results.
• Include results of PPI surveys on labeling in a manner that users and health professionals can easily understand, including device benefits and risks.

Additionally, FDA may also assess questions asked during the PPI collection process to confirm they avoid cognitive biases that could affect results and the labeling provided by manufacturers.