The new article outlines the key points regarding patient preference information and its use in the context of the product lifecycle.
Table of content
The Food and Drug Administration (FDA or the Agency), the US regulating authority in the sphere of healthcare products, has published a guidance document dedicated to incorporating voluntary patient preference information over the Total Product Life Cycle (TPLC). The guidance provides a general overview of the applicable regulatory requirements, as well as additional clarifications and recommendations to be taken into consideration by medical device manufacturers and other parties involved in order to ensure compliance.
At the same time, provisions of the guidance are non-binding in their legal nature, nor are they intended to introduce new rules or impose new obligations. The authority explicitly states that an alternative approach could be applied, provided such an approach is in line with the existing regulatory framework and has been agreed with the authority in advance.
Introduction
First of all, the FDA recognizes the significant value that patient experiences and perspectives bring to its decision-making process regarding medical devices. Patients living with a disease or condition often develop unique insights into the benefits and risks associated with medical devices.
Their lived experiences can contribute crucially to the FDA’s benefit-risk evaluation of specific devices. The FDA believes patients’ views and preferences should play a role in shaping the Agency’s assessments and encourages the inclusion of patient input in the evaluation process.
As a reflection of this, the FDA acknowledges and utilizes patient experience data, as described in Section 569C of the Federal Food, Drug, and Cosmetic Act (FD&C Act), amended by the 21st Century Cures Act. This data informs the FDA about patients’ experiences with a disease or condition, and helps guide industry in incorporating such insights into the device development and evaluation process.
In particular, “patient preference information” (PPI) is a subset of patient experience data that focuses on patients’ preferences concerning specific outcomes and treatments. This document outlines recommendations for incorporating voluntary PPI in FDA staff decision-making and aims to provide clarity on the Agency’s expectations regarding PPI.
It details the importance of patient perspectives in assessing the benefit-risk profile of certain devices and offers recommendations to industry stakeholders for collecting and submitting this type of data for regulatory consideration.
Regulatory Background
In 2016, the FDA released guidance on the voluntary submission and use of PPI in device applications, including Premarket Approval Applications (PMAs), Humanitarian Device Exemption (HDE) applications, and De Novo classification requests. This initiative was part of the FDA’s response to the Food and Drug Administration Safety and Innovation Act (FDASIA), which mandates the Agency to solicit patient views during the development process of medical products.
Since the release of the 2016 guidance, the landscape surrounding PPI has evolved significantly. Industry-sponsored PPI studies have increasingly been submitted to the FDA for consideration in benefit-risk assessments.
The FDA has also engaged in collaborations with scientists and other stakeholders to incorporate PPI into clinical trial designs and regulatory decision-making. The Agency has co-hosted numerous events to advance the scientific and practical applications of PPI.
In light of these developments, the FDA’s benefit-risk framework now extends beyond the premarket review phase and encompasses the entire product life cycle. This includes the submission of Investigational Device Exemption (IDE) applications, 510(k) submissions, and more.
The draft guidance proposes revisions to the 2016 guidance to reflect this broader scope and provides updated recommendations based on the Agency’s expanded experience with PPI
Scope
The scope of this guidance is focused on the voluntary use of PPI in decision-making related to medical devices. PPI can be considered at all stages of a product’s life cycle, provided it meets applicable legal standards.
In certain cases, patient preferences may be particularly useful in evaluating the benefit-risk profile of a device, especially when the decisions patients must make regarding their treatment options are preference-sensitive.
Preference-sensitive decisions arise under several conditions: (1) when multiple treatment options exist and none is clearly superior for all patients, (2) when the supporting evidence for one option over others is uncertain or variable across patient populations, and (3) when patients’ preferences regarding the benefits and risks of treatment differ significantly from healthcare professionals’ views.
The FDA encourages sponsors and other interested parties to submit PPI when it can inform the Agency’s decision-making process. This guidance outlines several key objectives, including encouraging the submission of PPI, recommending qualities for patient preference studies, offering practical advice for collecting and submitting PPI, and providing guidance on how PPI can be included in decision summaries and device labeling.
Practical Recommendations for PPI
This guidance provides several practical recommendations for incorporating PPI in FDA decision-making processes. The recommendations are designed to help sponsors and other stakeholders conduct and submit high-quality patient preference studies, which may be considered valid scientific evidence.
PPI can play a critical role in various aspects of the FDA’s benefit-risk assessments. For instance, it can identify the most important benefits and risks from a patient’s perspective, assess the relative importance of different attributes of benefit and risk, and clarify how patients think about the trade-offs involved.
Additionally, PPI can help capture the heterogeneity of patient preferences across different subpopulations, offering valuable insights into subgroup considerations during benefit-risk assessments. While PPI provides meaningful insights, it is important to note that this guidance does not change any regulatory standards for marketing authorization or premarket reviews.
The inclusion of PPI remains voluntary and does not impose any new responsibilities on device sponsors. Rather, it serves as a recommendation for the voluntary collection of patient preference data that can be considered alongside clinical, non-clinical, and real-world evidence.
Patient Preference Information Across the Product Lifecycle
As explained by the Authority, the knowledge gained from PPI studies can be applied throughout a product’s life cycle. It may be useful during the review of IDE applications, PMAs, HDE applications, De Novo classification requests, and 510(k) submissions.
Furthermore, PPI can inform sponsors during the early stages of device development, including design, nonclinical testing, and the investigational and pre-submission phases. Sponsors and other stakeholders, such as patient groups and academic researchers, are encouraged to engage with the FDA early in the process of designing patient preference studies.
Early interactions with the FDA can help ensure that studies are designed effectively and aligned with regulatory expectations. The Agency is open to feedback and willing to work with stakeholders to refine study designs and optimize the quality of the data collected.
Conclusion
In summary, the FDA values the perspectives of patients and recognizes the importance of patient preference information in evaluating the benefit-risk profiles of medical devices. This guidance builds on the Agency’s efforts to incorporate PPI into regulatory decision-making and encourages stakeholders to submit patient preference data voluntarily. The recommendations outlined in this document are intended to facilitate the collection and submission of PPI, enhancing the FDA’s ability to make well-informed decisions that reflect patients’ needs and preferences across the total product life cycle.
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