FDA updates patient preference guidance to span the product life cycle

The US Food and Drug Administration (FDA) on Thursday issued a draft guidance that would significantly revise its previous guidance on using voluntary patient preference information (PPI) for medical devices, shifting its focus from the premarket phase to incorporating PPI across the total product life cycle.
 
The updates are based on lessons it has learned from using PPI data in regulatory decisions since the final guidance was published.
 
When finalized, the guidance will supersede the agency’s 2016 guidance on the inclusion of PPI in premarket approval (PMA) applications, humanitarian device exemption applications, or de novo requests. The 2016 guidance was part of the agency’s mandate to develop strategies for using voluntary PPI during product development and in regulatory decisions. FDA said the changes in the new draft guidance were made based on its experience with a growing number of industry-sponsored PPI studies, better understanding of PPI trial designs, and other factors.
 
“FDA is issuing this draft guidance to propose revisions to the 2016 guidance to reflect the current scope of FDA’s benefit-risk paradigm, which may under appropriate circumstances include PPI, and to provide additional considerations and practical recommendations based on additional experience evaluating patient preferences regarding devices,” said FDA.
 
FDA also noted the draft guidance would fulfill certain provisions of the Medical Device User Fee Amendments (MDUFA V) deal negotiated with the medtech industry. Under MDUFA V, the agency agreed to expand its capacity and increase resources to respond to premarket submissions that include PPI data and update its 2016 guidance with pragmatic insights and address common questions surrounding PPI.
 
While the 2016 guidance has a section that asks when and how might FDA consider PPI during premarket reviews, the new guidance details situations where the agency used such information to support its review decisions.
 
It provides several examples, including a situation in which the Center for Devices and Radiological Health (CDRH) sponsored a PPI study to evaluate how much risk patients are willing to tolerate against the potential benefits of an obesity weight loss device. The agency noted that it used a large enough sample size and demographic characteristics representative of the US population. It provides other details that sponsors could factor in designing PPI studies.
 
The guidance also includes examples the use of PPI studies to determine whether to expand a device's indication, update its labeling, and establish its performance threshold.
 
In the proposed draft guidance, FDA also adds more detail on when and how it may consider PPI in premarket submissions, reiterating that it will consider PPI during all stages of the device's total product life cycle (TPLC). The agency noted that for IDEs it will consider not only the risk-benefit to study patients but also the potential societal benefits from the knowledge gained from the study.
 
"In the context of a clinical study, patient preferences may vary in which outcomes matter most to a particular patient, the amount of risk they would be willing to accept in exchange for a certain amount of benefit, their preferred modality of treatment/diagnostic procedure (often devices are one option to be considered in a clinical care path which may include medication or surgical procedures), as well as the value they assign to the potential societal benefits of the research itself, in advancing potential medical options for patients in the future,” said FDA.
 
Similarly, FDA said it may look at data from a PPI study to make a regulatory decision for a 510(k) device even if it hasn’t met its typical risk-benefit requirements.
 
“There may be situations in which some patients and caregivers would prefer to have access to the device despite that the device is not in compliance with FDA requirements,” the agency said. “When making decisions involving administrative, enforcement, and other actions, FDA intends to consider, among other things, patient impact, whether patients and caregivers adequately understand related benefits and risks, and information that may be available regarding patient preferences for the availability of nonconforming or non-compliant devices.”
 
The draft guidance also details important factors when designing a PPI study, getting FDA Feedback on PPI study plans and providing results, and different methods for collecting PPI data. The appendix also includes a new section on determining the right study method depending on the device and its objective.
 
Stakeholders can comment on the draft guidance on www.regulations.gov under docket no. FDA-2015-D-1580 until 5 November. The agency is also hosting a webinar on 15 October to discuss the proposed guidance.
 
Draft guidance