The U.S. Food and Drug Administration (FDA) is setting a new standard for patient-centred care with its recently updated draft guidance on Patient Preference Information (PPI). This updated guidance significantly expands the role of PPI, extending its application beyond the premarket phase to cover the total product life cycle (TPLC) of medical interventions. This marks a shift in the FDA’s regulatory approach, ensuring that patient insights are considered not just before a product is approved, but throughout its entire journey—from development and design to post-market monitoring. Stakeholders are invited to provide feedback on this draft guidance before the 5th of November 2024.
History of PPI in FDA Decision-Making
The FDA first formally incorporated PPI into its regulatory decision-making framework with its 2016 guidance. This early framework encouraged the voluntary submission of PPI in applications such as Premarket Approval, De Novo requests, and Humanitarian Device Exemptions. The intention was to use patient perspectives, particularly around treatment outcomes and risk tolerance, to enhance benefit-risk assessments. By doing so, the FDA acknowledged that patients offer unique insights into which benefits they value most and the level of risk they are willing to accept.
The 2024 update broadens this framework by integrating PPI across the entire TPLC. This means that patient preferences will now inform not only the initial design and approval stages but also ongoing post-market surveillance, device design adjustments, and clinical trial endpoints. This evolution underscores the FDA’s belief that patient-centred healthcare is driven by understanding what truly matters to patients—whether that’s improved quality of life, the willingness to face certain risks, or specific treatment preferences.
Incorporating PPI Across the TPLC
By incorporating PPI throughout the product life cycle, the FDA ensures that patient perspectives inform key regulatory milestones, enhancing both the safety and effectiveness of medical interventions. The benefits of which can be found in our article: What are the benefits of preference research? Regardless at which stage a product is at there is likely some application of PPI that could support product development.
Overcoming Challenges in PPI Integration
While integrating PPI into regulatory decisions is a major step forward, it comes with several challenges:
One significant hurdle is ensuring that PPI studies capture the diversity of the U.S. population. For the data to be meaningful, study populations must be large enough and diverse enough to reflect the full range of patient experiences, preferences, and conditions. Otherwise, there is a risk of bias that could influence the regulatory process.
Another challenge is designing PPI studies that avoid cognitive biases. Studies must be carefully structured so that patients clearly understand the risks and benefits they are being asked to evaluate. Selecting the right patient preference elicitation methodologies—such as discrete choice experiments or best-worst scaling—and thoroughly piloting these methods with patients are essential steps in ensuring reliable data. Take a read of our articles on patient preference methodologies:
Finally, conducting PPI studies requires time and resources. These studies can be complex, often requiring significant investment. The FDA encourages early engagement between industry sponsors and the specialist research agencies to align on study design, methodology, and objectives, which can streamline the process and improve data quality.
The FDA as a Global Leader in PPI Integration
Compared to other regulatory markets, such as the European Union (EU), the FDA has taken a leading role in integrating PPI into its regulatory decision-making. While the EU’s Medical Device Regulation (MDR) emphasizes patient outcomes, the FDA’s approach is more comprehensive, embedding PPI across the entire product life cycle. This means that patient preferences are not only considered at the initial stages of product development but also during post-market surveillance and product monitoring. The U.S. market is therefore seen as a global leader in patient-centred regulation, as the FDA ensures that medical interventions are designed and evaluated based on real-world patient experiences and values.
Conclusion: Shaping the Future of Patient-centred Regulation
The FDA’s expanded use of PPI is transforming the landscape of medical intervention development and regulation. By embedding patient preferences across the entire product life cycle, the agency is fostering a more personalized and responsive regulatory framework. Although challenges remain in collecting representative data and ensuring study designs avoid bias, the FDA’s proactive approach is setting a new precedent for patient-centred healthcare innovation. As the FDA continues to lead globally in this area, its commitment to integrating patient voices will drive more meaningful and effective healthcare outcomes, ensuring that treatments and devices align with the real needs of the people who use them.
At DHT.health we have expertise in preference research. Our team members have previously been involved in research for the IMI PREFER initiative.
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Reference:
Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle. Draft Guidance for Industry, Food and Drug Administration Staff, and Other Interested Parties. September 2024, Link
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