The significance of patient-reported outcomes (PROs) in real-world evidence (RWE) studies is growing. As regulatory agencies like the FDA focus more on PROs impact on drug development and approvals, clinical trial teams must navigate the complexities of timing, methods, and data quality.
Here we summarize the insights shared during a recent webinar hosted by Castor’s CEO, Derk Arts, featuring industry experts Ari Gnanasakthy (Principal Scientist, RTI Health Solutions) and Willie Muehlhausen (Co-CEO, Safira Clinical Research Ltd), where they discussed a new research paper entitled Critical Comments by Food and Drug Administration Reviewers on Patient-Reported Outcomes in Food and Drug Administration Regulatory Submissions (2018-2021).
FDA’s Expectations for PRO Data in RWE
A key takeaway is how critical the FDA’s recent guidance on PROs is for shaping both regulatory decisions and drug development. Ari Gnanasakthy emphasized how PROs reflect the patient experience, particularly in RWE settings, where traditional clinical endpoints may miss key treatment impacts.
The FDA’s guidance highlights the importance of when and how PRO data is collected, stressing that timing directly impacts data reliability.
“We’ve often seen that poor timing in PRO collection skews the real-world applicability of the data.” – Willie Muehlhausen
To ensure compliance, trial sponsors must align PRO methodologies with regulatory expectations early on, incorporating PROs in both pre- and post-marketing phases.
Timing and Methods: Getting it Right
Collecting PROs at the right time and using the right methods is crucial for maintaining data integrity. Timing isn’t just about capturing experiences at set intervals—it’s about ensuring the data mirrors real-world treatment conditions.
Ari elaborated: “If we collect PROs too early or too late, we risk either underestimating or overestimating the patient burden. FDA expects us to align with the patient’s journey.” Muehlhausen noted that digital tools like ePRO systems enable more dynamic data capture, which aligns with regulatory expectations.
The panel also stressed the importance of continually evaluating self-reported data for accuracy, requiring structured PRO methodologies, including careful frequency of assessments and question framing.
ePRO Fatigue: A Real Threat to Data Quality
ePRO fatigue is a significant challenge in PRO collection. Muehlhausen explained how the rise of digital PRO collection brings the risk of overwhelming patients with too many questionnaires, leading to lower compliance and subpar data.
To combat this, the experts recommended patient-centered design from the outset. “ePRO systems should be intuitive and designed with the patient in mind,” Muehlhausen said. Simplicity and ease of use are critical to keeping patients engaged, with automated reminders and user-friendly interfaces playing a key role in maintaining data consistency.
Tolerability Data: Differentiate Your Treatment in Competitive Markets
Beyond efficacy data, tolerability data is gaining importance. Gnanasakthy highlighted that the FDA is focusing more on how well patients endure treatment, especially for chronic conditions where long-term adherence is critical.
“The FDA’s focus on tolerability reflects a broader trend toward understanding the holistic patient experience. This isn’t just about ‘does the treatment work,’ but ‘can the patient live with it long-term?’” – Ari Gnanasakthy
Including tolerability data in RWE studies can help sponsors differentiate their products, especially in competitive markets with multiple treatment options.
Practical Solutions to Common Pitfalls in PRO Collection
The panel shared strategies to overcome common pitfalls in PRO collection. One key point was the need for agile study designs that allow for adjustments based on early PRO data trends. Gnanasakthy emphasized that “rigid study designs often fail to account for real-world variability in patient experiences.”
Other solutions included embedding PRO collection into patients’ daily routines to reduce disruption and fatigue. Muehlhausen stated, “We need to think about the patient’s lifestyle when designing PRO questionnaires. If the process fits into their day, we’re more likely to get high-quality, consistent data.”
The panel also recommended bring-your-own-device (BYOD) strategies, allowing patients to use their personal devices for PRO assessments. This increases convenience and lowers the barrier to participation.
FDA Guidance: A Blueprint for the Future
As the FDA refines its expectations for PRO collection, it’s clear that RWE will play an increasingly important role in regulatory decisions. The insights shared during the webinar provide a blueprint for aligning PRO strategies with FDA expectations while focusing on patient experience.
The experts agreed that the future of PRO data collection lies in seamlessly integrating technology with patient-centric design. Castor’s CEO, Derk Arts, closed the session by stressing the importance of ongoing collaboration between sponsors, patients, and regulators: “We must continue to innovate and adapt, keeping the patient at the heart of clinical trials.”
Conclusion: Innovate Your PRO Strategy with Castor
To stay ahead of the curve and ensure compliance with FDA guidelines, trial sponsors must adopt innovative PRO collection methods that prioritize patient engagement and data quality. As the industry continues to evolve, Castor offers cutting-edge solutions that integrate seamlessly into your clinical trials, ensuring robust data collection and real-world relevance.